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O. Box 331|Westbury|New York|11590|United States", "orphan_designation": { "original_text": "Treatment of Huntington's Disease", "umls": "C0020179", "parsed_text": "Huntington's Disease" } }, { "pubchem_cid": 9962735, "pubchem_sid": [ "136343285", "163639023", "254788202", "318029880", "319341249", "341140596", "341843053", "347737334", "347827969", "383859683", "438472612", "3680", "313077440", "319295328", "319382831", "348016842", "348275188", "405514769", "440061425", "8143934", "50625719", "57392606", "348282000", "385782543", "386619299", "126600207", "135263274", "164235288", "318692856", "342551539", "364207342", "404571817", "404573485" ], "inchikey": "QNTNKSLOFHEFPK-UPTCCGCDSA-N", "generic_name": "ubiquinol", "designated_date": "2004-04-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Gel-Tec, Division of Tishcon Corp.|30 New York Avenue|P. O. Box 331|Westbury|New York|11590|United States", "orphan_designation": { "original_text": "Treatment of Huntington's Disease", "umls": "C0020179", "parsed_text": "Huntington's Disease" } }, { "pubchem_cid": 197033, "pubchem_sid": [ "50034601", "126667050", "135174889", "224311642", "319198566", "319447901", "340503287", "341171113", "347828086", "349007744", "375581269", "381375857", "135626655", "252160153", "252215282", "317223813", "135329100" ], "inchikey": "JNODQFNWMXFMEV-UHFFFAOYSA-N", "generic_name": "dimebon", "designated_date": "2009-05-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Medivation, Inc.|201 Spear Street|San Francisco|California|94105|United States", "orphan_designation": { "original_text": "Treatment of Huntington's Disease.", "umls": "C0020179", "parsed_text": "Huntington's Disease" } }, { "pubchem_cid": 23697158, "pubchem_sid": [ "12015373", "135203220", "160681713", "224362604", "315739927", 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"Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "AOP Orphan Pharmaceuticals AG|91/llf Wilhelminenstra\u07e5|Wien|Wien|Austria", "orphan_designation": { "original_text": "Treatment of Huntington's disease", "umls": "C0020179", "parsed_text": "Huntington's Disease" } }, { "pubchem_cid": 6018, "pubchem_sid": [ "13350", "442479", "444004", "7980763", "10528302", "46506426", "49890235", "51230337", "56311658", "103928969", "104234203", "126658298", "126687256", "134222085", "144205356", "160967820", "163565040", "165238101", "170464675", "170500523", "178101536", "185996914", "196111337", "223399152", "224190819", "241102161", "250070083", "251893367", "252218966", "252559579", "254761331", "274087011", "310280666", "312345209", "316505858", "319064017", "319483732", "335964608", "340503622", "341102540", "341143339", "348284015", "349877216", "349983746", "354329944", "355036900", "355046980", "355429006", "374032971", "375577778", "375780570", 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"designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Laxdale Ltd.|Kings Park House, Laurelhill Business Park|Polmaise Road|Stirling|United Kingdom", "orphan_designation": { "original_text": "Treatment of Huntington's disease.", "umls": "C0020179", "parsed_text": "Huntington's Disease" } }, { "pubchem_sid": 162221917, "generic_name": "monosialotetrahexosylganglioside", "designated_date": "2018-08-07", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Qilu Pharmaceutical Co., Ltd.|243 Gong Ye Bei Road Jinan|Shandong|China", "orphan_designation": { "original_text": "Treatment of Huntingtons Disease", "umls": "C0020179", "parsed_text": "huntingtons disease" } }, { "pubchem_sid": 404719522, "generic_name": "Tominersen", "designated_date": "2015-12-29", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genentech, Inc.|1 DNA Way|South San Francisco|California|94080|United States", "orphan_designation": { "original_text": "Treatment of Huntington's disease", "umls": "C0020179", "parsed_text": "Huntington's Disease" } }, { "pubchem_sid": 402425700, "generic_name": "Tominersen", "designated_date": "2015-12-29", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genentech, Inc.|1 DNA Way|South San Francisco|California|94080|United States", "orphan_designation": { "original_text": "Treatment of Huntington's disease", "umls": "C0020179", "parsed_text": "Huntington's Disease" } } ] }, { "_id": "UMLS:C3542021", "fda_orphan_drug": [ { "pubchem_cid": 182232, "pubchem_sid": [ "11916", "611229", "103359466", "104025594", "135160710", "171572012", "274886242", "312232849", "319189673", "319884313", "322078602", "341194549", "378134545", "385679100", "386515665", "404760250", "415867362", "438792813", "439433489", "99233487", "254756175", "254791380", "348292973", "375666764", "441078034" ], "inchikey": "PFTAWBLQPZVEMU-ZFWWWQNUSA-N", "generic_name": "(+)-Epicatechin", "designated_date": "2020-04-06", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Epirium Bio Inc.|9171 Towne Centre Dr.,|Suite 270|San Diego|California|92122|United States", "orphan_designation": { "original_text": "Treatment of Duchenne and Becker muscular dystrophy", "umls": "C3542021", "parsed_text": "Duchenne and Becker Muscular Dystrophy" } }, { "pubchem_cid": 9904, "pubchem_sid": [ "9463", "7980080", "14718104", "48416309", "49965900", "50531284", "53788175", "85148374", "85300761", "92714065", "103202464", "103980273", "126624424", "126655692", "126688604", "134974495", "144089075", "144206718", "144206750", "144208410", "162222852", "163686430", "170466348", "178103531", "196108514", "210281245", "223385874", "224778327", "241086115", "250133434", "252159077", "252405880", "274190679", "310277448", 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St_xDE8B__xDD2C_oppsv\u49e5n|H\u49e5rsten-Liljeholmen|Stockholms l\u4b8d|Sweden", "orphan_designation": { "original_text": "Treatment of Duchenne and Becker Muscular Dystrophy", "umls": "C3542021", "parsed_text": "Duchenne and Becker Muscular Dystrophy" } }, { "pubchem_cid": 71587904, "pubchem_sid": [ "163643060", "315704063", "319413224", "385749595", "386586242", "404770113" ], "inchikey": "JOITUBWXBFATOR-HKBOAZHASA-N", "generic_name": "L-aminocarnityl-succinyl-leucyl-argininal-diethylacetal", "designated_date": "2006-01-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "CepTor Corporation|200 International Circle|Hunt Valley|Maryland|21030|United States", "orphan_designation": { "original_text": "Treatment of Duchenne and Becker muscular dystrophy", "umls": "C3542021", "parsed_text": "Duchenne and Becker Muscular Dystrophy" } } ] }, { "_id": "MONDO:0010383", "fda_orphan_drug": [ { "pubchem_cid": 9926832, "pubchem_sid": 223933254, "inchikey": "ZFPZEYHRWGMJCV-ZHALLVOQSA-N", "generic_name": "(-)-(3aR,4S,7aR)-4-Hydroxy-4-m-tolylethynyl-octahydro-indole-1-carboxylic acid methyl ester", "designated_date": "2011-10-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Novartis Pharmaceuticals Corp.|One Health Plaza|East Hanover|New Jersey|7936|United States", "orphan_designation": { "original_text": "Treatment of Fragile X syndrome", "umls": "C0016667", "parsed_text": "Fragile X Syndrome" } }, { "pubchem_cid": 22869857, "pubchem_sid": [ "319208225", "341722628" ], "inchikey": "HWSLMIXAPNOWGU-AWEZNQCLSA-N", "generic_name": "(3S)-(+)-(5-chloro-2-methoxyphenyl)-1,3-dihydro-3-fluoro-6-(trifluoromethyl)-2H-indol-2-one", "designated_date": "2015-12-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Centre National de la Recherche Scientifique (CNRS)|3 Rue Michel-Ange|Paris|France", "orphan_designation": { "original_text": "Treatment of Fragile X Syndrome.", "umls": "C0016667", "parsed_text": "Fragile X Syndrome" } }, { "pubchem_cid": 57411069, "pubchem_sid": 224706142, "inchikey": "JYHHQTRVJFMEGR-UHFFFAOYSA-N", "generic_name": "5,5-dimethyl-3-[2-(7-methylspiro[2H-benzofuran-3,1'-cyclopropane]-4-yl)oxypyrimidin-5-yl]imidazolidine-2,4-dione", "designated_date": "2017-06-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Autifony Therapeutics Limited|Gunnels Wood Road|Stevenage|England|United Kingdom", "orphan_designation": { "original_text": "Treatment of fragile X syndrome", "umls": "C0016667", "parsed_text": "Fragile X Syndrome" } }, { "pubchem_cid": 71158, "pubchem_sid": [ "46506657", "50021914", "56464352", "124955684", "134224179", "135027346", "160964004", "178103683", "250136754", "275739181", "312228300", "315680238", "316935568", "318160076", "319111350", "348283306", "349987190", "363592937", 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9864881, "pubchem_sid": [ "249748714", "313370987", "313390214", "313390341", "313513294", "318464630", "319340099", "319556295", "341187651", "342615156", "347260224", "347713305", "347827716", "347966319", "374093546", "375566681", "375675460", "376032014", "382370879", "383418273", "384416325", "384569453", "385646533", "386482917", "404638786", "405559762", "433835388", "434122195", "434250970", "438455067", "438504575", "439542126", "440062366", "440711126", "441017307", "313094237", "340494517", "340504622", "349338264", "364008431", "384283404", "386330316" ], "inchikey": "AFLFKFHDSCQHOL-IZZDOVSWSA-N", "generic_name": "elafibranor", "designated_date": "2019-07-25", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genfit SA|Parc Eurasant\u9b20885 Avenue Eug\u8ba5 Avin\u994d|Loos|France", "orphan_designation": { "original_text": "Treatment of Primary Biliary Cholangitis", "umls": "C0008312", "parsed_text": "Primary 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O. Box 2000, RY 33-212|Rahway|New Jersey|7065|United States", "orphan_designation": { "original_text": "Treatment of multiple myeloma", "umls": "C0026764", "parsed_text": "Multiple Myeloma" } }, { "pubchem_cid": 216326, "pubchem_sid": [ "12015317", "46505725", "50067858", "50484855", "53786789", "91148448", "103222228", "126591352", "126667069", "134964412", "135134887", "135600354", "135610831", "135683667", "136374703", "144206045", "152258939", "160647784", "160963826", "162184835", "163116827", "164194989", "170465201", "174478244", "178103903", "204370207", "223393899", "223762183", "251915646", "251916996", "251971155", "252110265", "252160457", "252165691", "252215688", "254760266", "255345986", "272830542", "310275735", "312228170", "312616225", "313062285", "313081285", "315682133", "316490020", "316967097", "318162929", "319209266", "319552138", "322090437", "329795811", "340462709", "340547198", "341100977", "341141245", "341839826", "346420003", "347760383", "348273293", 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myeloma", "marketing_approval_date": "2006-05-25", "exclusivity_end_date": "2013-05-25", "sponsor": "Celgene Corporation|P.O. Box 4914|7 Powder Horn Srive|Warren|New Jersey|7059|United States", "orphan_designation": { "original_text": "Treatment of multiple myeloma", "umls": "C0026764", "parsed_text": "Multiple Myeloma" } }, { "pubchem_sid": 434122177, "generic_name": "belantamab mafodotin-blmf", "trade_name": "BLENREP", "designated_date": "2017-06-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "for the treatment of adults with relapsed or refractory multiple myeloma who have received at least 4 prior therapies, including an anti-CD38 monoclonal antibody, a proteasome inhibitor, and an immunomodulatory agent", "marketing_approval_date": "2020-08-05", "exclusivity_end_date": "2027-08-05", "exclusivity_protected_indication": "for the treatment of adults with relapsed or refractory multiple myeloma who have received at least 4 prior therapies, including an anti-CD38 monoclonal antibody, a proteasome inhibitor, and an immunomodulatory agent", "sponsor": "GlaxoSmithKline Intellectual Property Development Ltd.|980 GREAT WEST ROAD|BRENTFORD|MIDDLESEX|TW89GS|United Kingdom", "orphan_designation": { "original_text": "Treatment of multiple myeloma", "umls": "C0026764", "parsed_text": "Multiple Myeloma" } }, { "pubchem_sid": 405226462, "generic_name": "belantamab mafodotin-blmf", "trade_name": "BLENREP", "designated_date": "2017-06-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "for the treatment of adults with relapsed or refractory multiple myeloma who have received at least 4 prior therapies, including an anti-CD38 monoclonal antibody, a proteasome inhibitor, and an immunomodulatory agent", "marketing_approval_date": "2020-08-05", "exclusivity_end_date": "2027-08-05", "exclusivity_protected_indication": "for the treatment of adults with relapsed or refractory multiple myeloma who have received at least 4 prior therapies, including an anti-CD38 monoclonal antibody, a proteasome inhibitor, and an immunomodulatory agent", "sponsor": "GlaxoSmithKline Intellectual Property Development Ltd.|980 GREAT WEST ROAD|BRENTFORD|MIDDLESEX|TW89GS|United Kingdom", "orphan_designation": { "original_text": "Treatment of multiple myeloma", "umls": "C0026764", "parsed_text": "Multiple Myeloma" } }, { "pubchem_sid": 404719842, "generic_name": "belantamab mafodotin-blmf", "trade_name": "BLENREP", "designated_date": "2017-06-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "for the treatment of adults with relapsed or refractory multiple myeloma who have received at least 4 prior therapies, including an anti-CD38 monoclonal antibody, a proteasome inhibitor, and an immunomodulatory agent", "marketing_approval_date": "2020-08-05", "exclusivity_end_date": "2027-08-05", "exclusivity_protected_indication": "for the treatment of adults with relapsed or refractory multiple myeloma who have received at least 4 prior therapies, including an anti-CD38 monoclonal antibody, a proteasome inhibitor, and an 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Box 776|Spring House|Pennsylvania|19477|United States", "orphan_designation": { "original_text": "Treatment of multiple myeloma", "umls": "C0026764", "parsed_text": "Multiple Myeloma" } }, { "pubchem_sid": 135263755, "generic_name": "siltuximab", "designated_date": "2005-10-11", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Janssen Research & Development, LLC|1400 McKean Road|P.O. Box 776|Spring House|Pennsylvania|19477|United States", "orphan_designation": { "original_text": "Treatment of multiple myeloma", "umls": "C0026764", "parsed_text": "Multiple Myeloma" } }, { "pubchem_sid": 404719648, "generic_name": "idecabtagene vicleucel", "trade_name": "Abecma", "designated_date": "2016-05-11", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody.", "marketing_approval_date": "2021-03-26", "sponsor": "Celgene Corporation|86 Morris Avenue|Summit|New Jersey|7901|United States", "orphan_designation": { "original_text": "Treatment of multiple myeloma", "umls": "C0026764", "parsed_text": "Multiple Myeloma" } }, { "pubchem_sid": 387061105, "generic_name": "pembrolizumab", "designated_date": "2016-02-17", "designation_status": 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O. 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Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of pulmonary tuberculosis.", "umls": "C0041327", "parsed_text": "Pulmonary tuberculosis" } }, { "pubchem_cid": 137235501, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-RDKOBYJASA-N", "generic_name": "Rifapentine", "trade_name": "Priftin", "designated_date": "1995-06-09", "designation_status": "Designated/Approved", "marketing_approval_date": "1998-06-22", "exclusivity_end_date": "2005-06-22", "sponsor": "Hoechst Marion Roussel|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of pulmonary tuberculosis.", "umls": "C0041327", "parsed_text": "Pulmonary tuberculosis" } }, { "pubchem_cid": 137091916, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-LBJWSYCFSA-N", "generic_name": "Rifapentine", "trade_name": "Priftin", "designated_date": "1995-06-09", "designation_status": "Designated/Approved", "marketing_approval_date": "1998-06-22", "exclusivity_end_date": "2005-06-22", "sponsor": "Hoechst Marion Roussel|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of pulmonary tuberculosis.", "umls": "C0041327", "parsed_text": "Pulmonary tuberculosis" } }, { "pubchem_cid": 137030254, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-USLNORDZSA-N", "generic_name": "Rifapentine", "trade_name": "Priftin", "designated_date": "1995-06-09", "designation_status": "Designated/Approved", "marketing_approval_date": "1998-06-22", "exclusivity_end_date": "2005-06-22", "sponsor": "Hoechst Marion Roussel|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of pulmonary tuberculosis.", "umls": "C0041327", "parsed_text": "Pulmonary tuberculosis" } }, { "pubchem_cid": 135887820, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-QFWJEWEWSA-N", "generic_name": "Rifapentine", "trade_name": "Priftin", "designated_date": "1995-06-09", "designation_status": "Designated/Approved", "marketing_approval_date": "1998-06-22", "exclusivity_end_date": "2005-06-22", "sponsor": "Hoechst Marion Roussel|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of pulmonary tuberculosis.", "umls": "C0041327", "parsed_text": "Pulmonary tuberculosis" } }, { "pubchem_cid": 136122663, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-FHDUCCPFSA-N", "generic_name": "Rifapentine", "trade_name": "Priftin", "designated_date": "1995-06-09", "designation_status": "Designated/Approved", "marketing_approval_date": "1998-06-22", "exclusivity_end_date": "2005-06-22", "sponsor": "Hoechst Marion Roussel|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of pulmonary tuberculosis.", "umls": "C0041327", "parsed_text": "Pulmonary tuberculosis" } }, { "pubchem_cid": 135932821, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-METIDKAPSA-N", "generic_name": "Rifapentine", "trade_name": "Priftin", "designated_date": "1995-06-09", "designation_status": "Designated/Approved", "marketing_approval_date": "1998-06-22", "exclusivity_end_date": "2005-06-22", "sponsor": "Hoechst Marion Roussel|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of pulmonary tuberculosis.", "umls": "C0041327", "parsed_text": "Pulmonary tuberculosis" } }, { "pubchem_cid": 135925739, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-PWXWUWFUSA-N", "generic_name": "Rifapentine", "trade_name": "Priftin", "designated_date": "1995-06-09", "designation_status": "Designated/Approved", "marketing_approval_date": "1998-06-22", "exclusivity_end_date": "2005-06-22", "sponsor": "Hoechst Marion Roussel|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of pulmonary tuberculosis.", "umls": "C0041327", "parsed_text": "Pulmonary tuberculosis" } }, { "pubchem_cid": 135749824, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-HJTBMZGUSA-N", "generic_name": "Rifapentine", "trade_name": "Priftin", "designated_date": "1995-06-09", "designation_status": "Designated/Approved", "marketing_approval_date": "1998-06-22", "exclusivity_end_date": "2005-06-22", "sponsor": "Hoechst Marion Roussel|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of pulmonary tuberculosis.", "umls": "C0041327", "parsed_text": "Pulmonary tuberculosis" } }, { "pubchem_cid": 136246610, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-BGZYVKEHSA-N", "generic_name": "Rifapentine", "trade_name": "Priftin", "designated_date": "1995-06-09", "designation_status": "Designated/Approved", "marketing_approval_date": "1998-06-22", "exclusivity_end_date": "2005-06-22", "sponsor": "Hoechst Marion Roussel|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of pulmonary tuberculosis.", "umls": "C0041327", "parsed_text": "Pulmonary tuberculosis" } }, { "pubchem_cid": 135968625, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-LYDPARFQSA-N", "generic_name": "Rifapentine", "trade_name": "Priftin", "designated_date": "1995-06-09", "designation_status": "Designated/Approved", "marketing_approval_date": "1998-06-22", "exclusivity_end_date": "2005-06-22", "sponsor": "Hoechst Marion Roussel|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of pulmonary tuberculosis.", "umls": "C0041327", "parsed_text": "Pulmonary tuberculosis" } }, { "pubchem_cid": 136238508, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-NAFQJNKLSA-N", "generic_name": "Rifapentine", "trade_name": "Priftin", "designated_date": "1995-06-09", "designation_status": "Designated/Approved", "marketing_approval_date": "1998-06-22", "exclusivity_end_date": "2005-06-22", "sponsor": "Hoechst Marion Roussel|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of pulmonary tuberculosis.", "umls": "C0041327", "parsed_text": "Pulmonary tuberculosis" } }, { "pubchem_cid": 136073066, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-QEDJKGLXSA-N", "generic_name": "Rifapentine", "trade_name": "Priftin", "designated_date": "1995-06-09", "designation_status": "Designated/Approved", "marketing_approval_date": "1998-06-22", "exclusivity_end_date": "2005-06-22", "sponsor": "Hoechst Marion Roussel|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of pulmonary tuberculosis.", "umls": "C0041327", "parsed_text": "Pulmonary tuberculosis" } }, { "pubchem_cid": 135659016, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-PRHSTWAZSA-N", "generic_name": "Rifapentine", "trade_name": "Priftin", "designated_date": "1995-06-09", "designation_status": "Designated/Approved", "marketing_approval_date": "1998-06-22", "exclusivity_end_date": "2005-06-22", "sponsor": "Hoechst Marion Roussel|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of pulmonary tuberculosis.", "umls": "C0041327", "parsed_text": "Pulmonary tuberculosis" } }, { "pubchem_cid": 135403821, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-GZAUEHORSA-N", "generic_name": "Rifapentine", "trade_name": "Priftin", "designated_date": "1995-06-09", "designation_status": "Designated/Approved", "marketing_approval_date": "1998-06-22", "exclusivity_end_date": "2005-06-22", "sponsor": "Hoechst Marion Roussel|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of pulmonary tuberculosis.", "umls": "C0041327", "parsed_text": "Pulmonary tuberculosis" } }, { "pubchem_cid": 135431094, "pubchem_sid": [ "603306", "50064322", "75991563", "103536525", "126682304", "135270008", "175426909", "252356475", "254765255", "341853850", "347761037", "348685253", "385640623", "386634879", "404982750", "405021458", "426117527", "433958824", "434305013", "438488047", "319331748", "349995263", "438580343", "75991562", "75991565", "126624292", "316898584", "341237105", "405447794", "434123879", "439994458", "75991564", "162223574", "318039514", "365292305", "375201963", "375676632", "387239722" ], "inchikey": "IXSVOCGZBUJEPI-HTQYORAHSA-N", "generic_name": "Rifalazil", "designated_date": "1999-04-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "PathoGenesis Corporation|201 Elliott Avenue West|Suite 150|Seattle|Washington|98119|United States", "orphan_designation": { "original_text": "Treatment of pulmonary tuberculosis.", "umls": "C0041327", "parsed_text": "Pulmonary tuberculosis" } } ] }, { "_id": "MONDO:0019087", "fda_orphan_drug": [ { "pubchem_cid": 124081779, "pubchem_sid": [ "376161678", "434250533", "438453701" ], "inchikey": "OLDWOVJAXRWCGT-FBHGDYMESA-N", "generic_name": "(R)-6-(2-fluorophenyl)-N-(3-(2-((2-methoxyethyl)amino)ethyl)phenyl)-5,6-dihydrobenzo[h]quinazolin-2-amine dihydrochloride", "designated_date": "2015-11-19", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Basilea Pharmaceutical International Ltd.|Grenzacherstrasse 487|P.O. Box|Basel|Switzerland", "orphan_designation": { "original_text": "Treatment of cholangiocarcinoma.", "umls": "C0206698", "parsed_text": "Cholangiocarcinoma" } }, { "pubchem_cid": 53235510, "pubchem_sid": [ "152258531", "160647365", "163642798", "196409720", "223366206", "242459086", "252160439", "312616444", "319393309", "340088428", "347828222", "355040419", "375586013", "377015756", "384568707", "384572677", "385647133", "386483530", "387247480", "419562004", "438469455", "375646074", "405507520", "440054176" ], "inchikey": "QADPYRIHXKWUSV-UHFFFAOYSA-N", "generic_name": "Infigratinib", "designated_date": "2019-09-11", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "QED Therapeutics|8000 Marina Boulevard|Suite 400|Brisbane|California|94005|United States", "orphan_designation": { "original_text": "Treatment of cholangiocarcinoma", "umls": "C0206698", "parsed_text": "Cholangiocarcinoma" } }, { "pubchem_cid": 135565128, 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"approved_labeled_indication": "Indicated for maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy", "marketing_approval_date": "2017-03-27", "exclusivity_end_date": "2024-03-27", "exclusivity_protected_indication": "Indicated for maintenance treatment of adult patients with recurrent epithelial ovarian cancer who are in a complete or partial response to platinum-based chemotherapy", "sponsor": "GlaxoSmithKline, LLC.|1000 Winter St.|Suite 3300|Waltham|Massachusetts|2451|United States", "orphan_designation": { "original_text": "Treatment of ovarian cancer", "umls": "C4721610", "parsed_text": "Ovarian Cancer" } }, { "pubchem_cid": 158780, "pubchem_sid": [ "135114357", "160682136", "385758894", "386595557", "404622000", "349977020", "434302045", "438484040" ], "inchikey": "GDLPAGOVHZLZEK-JBUFHSOLSA-L", "generic_name": "oglufanide disodium", "designated_date": "2001-09-24", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Implicit Bioscience Pty Ltd|P. O. Box 2072|Toowong Business Center|Brisbane|Queensland|Australia", "orphan_designation": { "original_text": "Treatment of ovarian cancer", "umls": "C4721610", "parsed_text": "Ovarian Cancer" } }, { "pubchem_cid": 23725625, "pubchem_sid": [ "85197660", "103605183", "126626885", "126644879", "126664211", "126666996", "131407195", "134222742", "134964396", "135261055", "135697767", "135708109", "136377786", "136895641", "152258100", "160646939", "160810829", "162058030", "162172208", "163088771", "187051821", "187072934", "223375126", "223736293", "251971206", "252159146", "252160170", "252215948", "254759126", "254761805", "255385074", "273884861", "275893966", "310278320", "312616324", "316580561", "316900182", "318163909", "319379284", "319436166", "319463087", "319530703", "319552093", "340506476", "340522971", "340548349", "341143310", "346435859", "347735953", "347827823", "349892423", "349988538", "350079491", "350080568", "350095305", "355040777", "371389352", "374073206", "375577677", "375581723", "375757262", "376033298", "376191636", "378130213", "381316845", "382371665", "384570915", "384572353", "385637718", "386244507", "386631899", "387020473", "387088344", "387250273", "402419171", "426257378", "433796082", "438507039", "440806628", "440999262", "441104665", "170559095", "402287500", "99218744", "164231742", "204362527", "221672931", "349485330", "364206739", "419498287" ], "inchikey": "FDLYAMZZIXQODN-UHFFFAOYSA-N", "generic_name": "olaparib", "trade_name": "Lynparza", "designated_date": "2013-10-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "Lynparza is indicated for the maintenance treatment of adult patients with deleterious or suspected deleterious germline or somatic BRCA-mutated (gBRCAm or sBRCAm) advanced epithelial ovarian, fallopian tube or primary peritoneal cancer who are in complete or partial response to first-line platinum based chemotherapy. 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Box 8355|Wilmington|Delaware|19803|United States", "orphan_designation": { "original_text": "Treatment of ovarian cancer", "umls": "C4721610", "parsed_text": "Ovarian Cancer" } }, { "pubchem_cid": 23725625, "pubchem_sid": [ "85197660", "103605183", "126626885", "126644879", "126664211", "126666996", "131407195", "134222742", "134964396", "135261055", "135697767", "135708109", "136377786", "136895641", "152258100", "160646939", "160810829", "162058030", "162172208", "163088771", "187051821", "187072934", "223375126", "223736293", "251971206", "252159146", "252160170", "252215948", "254759126", "254761805", "255385074", "273884861", "275893966", "310278320", "312616324", "316580561", "316900182", "318163909", "319379284", "319436166", "319463087", "319530703", "319552093", "340506476", "340522971", "340548349", "341143310", "346435859", "347735953", "347827823", "349892423", "349988538", "350079491", "350080568", "350095305", "355040777", "371389352", "374073206", "375577677", "375581723", "375757262", "376033298", "376191636", "378130213", "381316845", "382371665", "384570915", "384572353", "385637718", "386244507", "386631899", "387020473", "387088344", "387250273", "402419171", "426257378", "433796082", "438507039", "440806628", "440999262", "441104665", "170559095", "402287500", "99218744", "164231742", "204362527", "221672931", "349485330", "364206739", "419498287" ], "inchikey": "FDLYAMZZIXQODN-UHFFFAOYSA-N", "generic_name": "olaparib", "trade_name": "Lynparza", "designated_date": "2013-10-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "Maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer, who are in a complete or partial response to platinum-based chemotherapy.", "marketing_approval_date": "2017-08-17", "exclusivity_end_date": "2024-08-17", "exclusivity_protected_indication": "Maintenance treatment of adult patients with recurrent epithelial ovarian cancer, who are in a complete or partial response to platinum-based chemotherapy.", "sponsor": "AstraZeneca Pharmaceuticals LP|1800 Concord Pike|P. 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Box 8355|Wilmington|Delaware|19803|United States", "orphan_designation": { "original_text": "Treatment of ovarian cancer", "umls": "C4721610", "parsed_text": "Ovarian Cancer" } }, { "pubchem_cid": 44147881, "pubchem_sid": 341411255, "inchikey": "HSUDWURBWSUCOB-IBEPEKEJSA-N", "generic_name": "Protaxel", "designated_date": "2003-05-21", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Biophysica, Inc.|2159 Avenida de la Playa|Suite B|La Jolla|California|92037|United States", "orphan_designation": { "original_text": "Treatment of ovarian cancer", "umls": "C4721610", "parsed_text": "Ovarian Cancer" } }, { "pubchem_cid": 6918736, "pubchem_sid": [ "85345035", "103956529", "123100264", "135268707", "196409610", "223366164", "252480712", "255344259", "315768285", "318153631", "319332977", "319552695", "340580296", "347828442", "349145989", "349989045", "355200504", "374243553", "375590759", "378132064", "382162881", "385644678", "386481007", "404622261", "419596551", "433865271", "438447942", "439399426", "439478217", "439575551", "440703372", "275174301", "317223505", "341109255", "341189821", "348273539", "375577206", "375583145", "341101386", "405493361", "440040019", "210274754", "210280387", "322095628", "387244248", "440805428" ], "inchikey": "OWBFCJROIKNMGD-BQYQJAHWSA-N", "generic_name": "rigosertib", "designated_date": "2012-03-16", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Onconova Therapeutics, Inc.|375 Pheasant Run|Newton|Pennsylvania|18940|United States", "orphan_designation": { "original_text": "Treatment of ovarian cancer", "umls": "C4721610", "parsed_text": "Ovarian Cancer" } }, { "pubchem_cid": 9931954, "pubchem_sid": [ "85334460", "103769588", "162108736", "170474818", "194944026", "210274713", "210280347", "223366069", "223471430", "254760581", "255511898", "313090884", "315742943", "319340914", "319552790", "335988518", "341191031", "347828593", "348355010", "348947434", "349976023", "350079479", "355039660", "373898099", "375397402", "375590790", "376026891", "376142821", "378132134", "381120351", "381340392", "382370917", "384416344", "384568643", "384572593", "385640676", "386634932", "387099764", "387218156", "403228967", "405559537", "433865315", "439483764", "441057114", "162202720", "251971427", "252160225", "402381605", "221672886", "341106425" ], "inchikey": "HMABYWSNWIZPAG-UHFFFAOYSA-N", "generic_name": "rucaparib", "trade_name": "Rubraca", "designated_date": "2012-07-31", "designation_status": "Designated/Approved", "approved_labeled_indication": "As monotherapy for the treatment of patients with deleterious BRCA mutation (germline and/or somatic) associated advanced ovarian cancer who have been treated with two or more chemotherapies", "marketing_approval_date": "2016-12-19", "exclusivity_end_date": "2023-12-19", "exclusivity_protected_indication": "As monotherapy for the treatment of patients with deleterious BRCA mutation (germline and/or somatic) associated advanced ovarian cancer who have been treated with two or more chemotherapies", "sponsor": "Clovis Oncology, Inc.|5500 Flatiron Parkway|Suite 100|Boulder|Colorado|80301|United States", "orphan_designation": { "original_text": "Treatment of ovarian cancer", "umls": "C4721610", "parsed_text": "Ovarian Cancer" } }, { "pubchem_cid": 25091037, "pubchem_sid": [ "103770537", "126645764", "135119090", "319382177", "404615726", "434314910", "438492330", "439639868", "53787705", "135003636", "318162666", "440790630", "144205806", "315681992", "537734", "50070153", "316967941", "316967942", "348652723" ], "inchikey": "ZBVJFYPGLGEMIN-UHFFFAOYSA-N", "generic_name": "Triptorelin pamoate", "designated_date": "1990-08-10", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Debio R.A. 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O. Box 915|Whippany|New Jersey|7981|United States", "orphan_designation": { "original_text": "Treatment of systemic sclerosis", "umls": "C0036421", "parsed_text": "Systemic sclerosis" } }, { "pubchem_cid": 443951, "pubchem_sid": [ "12013758", "103272403", "103928787", "134223771", "134999860", "135651246", "144204835", "162226496", "224890546", "275456739", "310280640", "315681487", "318162401", "319223379", "329735779", "341155028", "349976308", "354272745", "385638429", "386632623", "387110135", "404770946", "419499563", "439367308", "439462902", "440062905", "319063509", "387268530", "438430676", "50060722", "319277385", "374236159" ], "inchikey": "JOAHPSVPXZTVEP-YXJHDRRASA-N", "generic_name": "terguride", "designated_date": "2013-05-17", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Medac GmbH|TheaterstraBe 6+|Wedel|Germany", "orphan_designation": { "original_text": "Treatment of systemic sclerosis", "umls": 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Lab|Milwaukee|Wisconsin|53233|United States", "orphan_designation": { "original_text": "Treatment of Systemic sclerosis", "umls": "C0036421", "parsed_text": "Systemic sclerosis" } }, { "pubchem_cid": 90420193, "pubchem_sid": [ "336446947", "336895202", "347831709", "347832120", "404772830", "406942915", "438645245" ], "inchikey": "REQQVBGILUTQNN-UHFFFAOYSA-N", "generic_name": "Ziritaxestat", "designated_date": "2020-01-27", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Galapagos NV|Generaal De Wittelaan|L11 A3|Mechelen|Belgium", "orphan_designation": { "original_text": "Treatment of systemic sclerosis", "umls": "C0036421", "parsed_text": "Systemic sclerosis" } }, { "pubchem_cid": 135423438, "pubchem_sid": [ "99431587", "103654502", "135267499", "152258852", "160647702", "178102559", "223471427", "249738349", "249807126", "251970985", "252090009", "255344081", "277286638", "310265007", "312616403", "318163907", 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O. Box 368|Ridgefield|Connecticut|6877|United States", "orphan_designation": { "original_text": "Treatment of systemic sclerosis (including the associated interstitial lung disease).", "umls": [ "C0036421", "C0206062" ], "parsed_text": [ "Systemic sclerosis", "Interstitial lung disease" ] } }, { "pubchem_cid": 5281613, "pubchem_sid": [ "12225", "623490", "29204087", "51194415", "87225346", "103531615", "126604530", "126671702", "134976843", "136367628", "136946451", "143490287", "143503044", "144204945", "152243492", "162037858", "162177436", "170466258", "172440052", "211535301", "223398359", "223439556", "223818765", "241090040", "250133959", "252159237", "252215293", "252359169", "252401625", "254761153", "274294572", "277309131", "310264956", "310272200", "312248420", "312694916", "315444733", "315681543", "316541322", "316972285", "318162797", "318166511", "319295975", "319447902", "329891073", "340520088", "341142697", "341832773", "346439740", "347756852", "347913079", "349096042", 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Indication", "sponsor": "Primus Pharmaceuticals, Inc.|7373 North Scottsdale Road|Suite B-200|Scottsdale|Alaska|85253|United States", "orphan_designation": { "original_text": "Treatment of systemic sclerosis", "umls": "C0036421", "parsed_text": "Systemic sclerosis" } }, { "pubchem_cid": 456390, "pubchem_sid": [ "78308738", "126600263", "136343340", "241097630", "252215534", "252393108", "277330375", "310274428", "313390293", "313390777", "315683447", "319220460", "319557571", "333497452", "341105543", "341138225", "341849655", "349124007", "354330598", "355036997", "374911450", "375201725", "378135279", "381366317", "383425740", "387121203", "404615874", "419499034", "433837096", "434315259", "436399811", "438493901", "441125423", "78308737", "126624129", "126655403", "176249982", "194161780", "196111405", "224826828", "312230239", "318042417", "318242142", "319104188", "340492606", "348287117", "371666427", "374386102", "375590811", "381118863", "382162480", "387121204", "415731178", 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Box 1005|425 Privet Road|Horsham|Pennsylvania|19044|United States", "orphan_designation": { "original_text": "Treatment of amyotrophic lateral sclerosis", "umls": "C0002736", "parsed_text": "Amyotrophic Lateral Sclerosis" } }, { "pubchem_cid": 3547, "pubchem_sid": [ "841200", "49961274", "50871527", "53790340", "92720222", "99444633", "103210437", "135023837", "136023843", "144076157", "160969216", "165237684", "178101871", "196106390", "210274676", "210280309", "224208489", "251916811", "251918050", "252215416", "252478843", "274603730", "312693366", "315683732", "316539207", "316966837", "318161072", "318238919", "319062384", "319476448", "319555411", "329894421", "341143145", "341852927", "348519453", "349977751", "350095398", "354284092", "355039692", "375296391", "375582474", "382152821", "385789643", "386626789", "387061033", "387128275", "419476730", "433862456", "434251812", "438456181", "439577715", "440802661", "441004996", "26758860", "163687984", "223485076", "252359270", 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Box 776|Spring hOuse|Pennsylvania|19477|United States", "orphan_designation": { "original_text": "Treatment of diffuse large B-cell lymphoma.", "umls": "C1332201", "parsed_text": "Diffuse Large B-Cell Lymphoma" } }, { "pubchem_sid": 381128081, "generic_name": "tisagenlecleucel-T", "trade_name": "Kymriah", "designated_date": "2015-02-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "KYMRIAH is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of Adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, high grade B-cell lymphoma and DLBCL arising from follicular lymphoma", "marketing_approval_date": "2018-05-01", "exclusivity_end_date": "2025-05-01", "exclusivity_protected_indication": "For treatment of adult patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma 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"pubchem_sid": 160687707, "generic_name": "Naratuximab emtansine", "designated_date": "2016-05-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Debiopharm International S.A.|Forum \"apres-demain\"|Chemin Messidor 5-7, CP 5911|Switzerland", "orphan_designation": { "original_text": "Treatment of diffuse large B-cell lymphoma", "umls": "C1332201", "parsed_text": "Diffuse Large B-Cell Lymphoma" } }, { "pubchem_sid": 347911301, "generic_name": "polatuzumab vedotin-piiq", "trade_name": "POLIVY", "designated_date": "2016-12-12", "designation_status": "Designated/Approved", "approved_labeled_indication": "POLIVY in combination with bendamustine and a rituximab product is indicated for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), not otherwise specified, after at least two prior therapies.", "marketing_approval_date": "2019-06-10", "exclusivity_end_date": "2026-06-10", 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lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (including DLBCL arising from indolent lymphoma), high-grade B cell lymphoma, primary mediastinal large B-cell lymphoma, and follicular lymphoma grade 3B", "marketing_approval_date": "2021-02-05", "exclusivity_end_date": "2028-02-05", "exclusivity_protected_indication": "treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy; treatment of adult patients with relapsed or refractory primary mediastinal large B-cell lymphoma after two or more lines of systemic therapy; and treatment of adult patients with relapsed or refractory follicular lymphoma grade 3B after two or more lines of systemic therapy", "sponsor": "Juno Therapeutics, Inc.|400 Dexter Avenue North|Suite 1200|Seattle|Washington|98109|United States", "orphan_designation": { "original_text": "Treatment of diffuse large B-cell lymphoma", "umls": 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B-Cell Lymphoma" } }, { "pubchem_sid": 381127365, "generic_name": "tafasitamab", "trade_name": "MONJUVI", "designated_date": "2014-12-01", "designation_status": "Designated/Approved", "approved_labeled_indication": "MONJUVI is indicated in combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT)", "marketing_approval_date": "2020-07-31", "exclusivity_end_date": "2027-07-31", "exclusivity_protected_indication": "indicated in combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT)", "sponsor": "MorphoSys AG|Semmelweisstrasse 7|Plannegg|Germany", 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Limitations of Use: COTELLIC is not indicated for treatment of patients with wild-type BRAF melanoma.", "marketing_approval_date": "2015-11-10", "exclusivity_end_date": "2022-11-10", "exclusivity_protected_indication": "For the treatment of patients with unresectable or metastatic melanoma with BRAF V600E or V600K mutation, in combination with vemurafenib. COTELLIC is not indicated for treatment of patients with wild-type BRAF melanoma", "sponsor": "Genentech, Inc.|1 DNA Way, MS 241A|South San Francisco|California|94080|United States", "orphan_designation": { "original_text": "Treatment of stage IIb, IIc, III, and IV melanoma with BRAFV600 mutation", "umls": [ "C0025202", "C4553416" ], "parsed_text": [ "melanoma", "IIC" ] } }, { "pubchem_cid": 10309114, "pubchem_sid": [ "224290135", "341721337", "349104806" ], "inchikey": "YZOQZEXYFLXNKA-UHFFFAOYSA-N", "generic_name": "N-[4-(4-amino-2-ethyl-1H-imidazo[4,5-c]quinolin-1-yl)butyl]methanesulfonamide", "designated_date": "2004-08-13", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Pfizer, Inc.|50 Pequot|6025-A3106|New London|Connecticut|6320|United States", "orphan_designation": { "original_text": "Treatment of stages IIB-IV melanoma", "umls": "C0025202", "parsed_text": "melanoma" } }, { "pubchem_cid": 118984458, 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Drive|Berkeley Heights|New Jersey|7922|United States", "orphan_designation": { "original_text": "Treatment of advanced malignant melanoma (Stages II,III, IV).", "umls": "C0025202", "parsed_text": "Malignant Melanoma" } }, { "pubchem_cid": 135564890, "pubchem_sid": [ "210275286", "210280935", "249565901", "249748718", "252451738", "252664617", "254759159", "255343590", "255495891", "312468219", "315369704", "321902353", "340503727", "340575709", "341187334", "347828080", "354329958", "355039305", "355173654", "365289515", "374089746", "375577065", "376033574", "378134682", "382154110", "383418302", "384569216", "385099620", "385651035", "386487485", "387061136", "433840039", "434254321", "436380783", "440062373", "440812851", "365265692", "374243533", "319439542", "332853702" ], "inchikey": "FBKMWOJEPMPVTQ-UHFFFAOYSA-N", "generic_name": "epacadostat", "designated_date": "2016-02-11", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Incyte Corporation|1801 Augustine Cut-Off|Wilmington|Delaware|19803|United States", "orphan_designation": { "original_text": "Treatment of stage IIB-IV melanoma.", "umls": "C0025202", "parsed_text": "melanoma" } }, { "pubchem_cid": 42611257, "pubchem_sid": [ "104245715", "123055406", "125240992", "131408691", "135267495", "135611115", "136367377", "152258377", "160647214", "163345507", "163390276", "175427131", "178102516", "223377738", "224732010", "249737055", "251916530", "251917877", "251971373", "252160309", "252553680", "255417511", "275027213", "312242875", "312616271", "312811253", "313077323", "315798888", "316497749", "316903952", "318163480", "319383307", "319552219", "329981134", "335962941", "340557354", "341142605", "347746699", "349995632", "355040540", "355045913", "373259044", "375561949", "375577201", "375782231", "376191703", "376229550", "381347952", "384568548", "384572464", "385785116", "386393063", "386622043", "387095544", "402419969", "406671415", 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"inchikey": "KSERXGMCDHOLSS-LJQANCHMSA-N", "generic_name": "Ulixertinib", "designated_date": "2013-06-24", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "BioMed Valley Discoveries, Inc.|4435 Main Street|Suite 550|Kansas City|Missouri|64111|United States", "orphan_designation": { "original_text": "Treatment of Stage IIb through Stage IV BRAF mutant melanoma", "umls": "C0025202", "parsed_text": "melanoma" } }, { "pubchem_cid": 405012, "pubchem_sid": [ "103707643", "135267136", "223090896", "249565906", "254803594", "312322666", "312811278", "317223253", "319220474", "319556279", "341483331", "347828994", "348551318", "355038078", "375981434", "376241469", "378132499", "384568951", "384573066", "385652666", "386489121", "433986451", "434316031", "438493658", "440062490", "335987348", "375590302", "375758485", "387258902" ], "inchikey": "ZADWXFSZEAPBJS-SNVBAGLBSA-N", "generic_name": "indoximod", "designated_date": "2017-10-26", 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O. 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O. 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Box 8355|1800 Concord Pike|Wilmington|Delaware|19803|United States", "orphan_designation": { "original_text": "Treatment of epidermal growth factor receptor mutation-positive non-small cell lung cancer", "umls": [ "C1414313", "C3539721" ], "parsed_text": [ "EPIDERMAL GROWTH FACTOR RECEPTOR", "Non-Small Cell Lung Cancer" ] } }, { "pubchem_cid": 134436906, "pubchem_sid": [ "381148416", "381148595", "381609315", "381744999", "386331670", "387059723", "406867341", "430057539", "433865382", "434310743", "438489574", "438645327", "438790728", "439507330", "440200949", "440702368", "440798555", "438433354", "438433356", "440792428" ], "inchikey": "XIIOFHFUYBLOLW-UHFFFAOYSA-N", "generic_name": "selpercatinib", "trade_name": "RETEVMO", "designated_date": "2019-10-30", "designation_status": "Designated/Approved", "approved_labeled_indication": "RETEVMO is indicated for the treatment of adult patients with metastatic RET fusion-positive non-small cell lung cancer (NSCLC)", 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Box 4099|Richmond|California|94804|United States", "orphan_designation": { "original_text": "Treatment of chronic lymphocytic leukemia (CLL), including refractory CLL.", "umls": [ "C0023434", "C0278791" ], "parsed_text": [ "Chronic Lymphocytic Leukemia", "Refractory CLL" ] } }, { "pubchem_cid": 24821094, "pubchem_sid": [ "135268986", "136940589", "136961336", "152258295", "160647134", "163679284", "165245631", "171572071", "178103494", "184611713", "224184328", "251971042", "252088605", "252160516", "254762145", "274856561", "310264995", "312616233", "312808358", "315666679", "316579164", "316895237", "318163839", "319380131", "319467196", "329891104", "336298027", "340503210", "340521902", "340557335", "341143338", "346400508", "349983592", "350079530", "350095230", "354326201", "374060348", "375675186", "378130332", "381930724", "384439412", "385790189", "386627347", "405045803", "406521656", "419509191", "434314965", "436380298", "438493198", "438509205", "439576951", "439656277", "440799881", "441002856", "441117731", "135727459", "176250273", "252543441", "254759213", "255386749", "319530702", "319882825", "332832563", "335987367", "354269615", "354329136", "375577284", "375582916", "375758466", "402287765", "440794864", "221673009", "223471421" ], "inchikey": "XYFPWWZEPKGCCK-GOSISDBHSA-N", "generic_name": "ibrutinib", "trade_name": "Imbruvica", "designated_date": "2012-04-06", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of patients with chronic lymphocytic leukemia (CLL) who have received at least one prior therapy", "marketing_approval_date": "2014-02-12", "exclusivity_end_date": "2021-02-12", "exclusivity_protected_indication": "Treatment of patients with chronic lymphocytic leukemia (CLL) who have received at least one prior therapy", "sponsor": "Pharmacyclics, LLC|995 E. Arques Avenue|Sunnyvale|California|94085|United States", "orphan_designation": { "original_text": "Treatment of chronic lymphocytic leukemia (CLL)", "umls": "C0023434", "parsed_text": "Chronic Lymphocytic Leukemia" } }, { "pubchem_cid": 118984458, "pubchem_sid": [ "53788769", "135296278", "319431432", "349979899", "428438211", "160671608", "160693157", "252166481", "404771330" ], "inchikey": "MIMNFCVQODTQDP-NDLVEFNKSA-N", "generic_name": "oblimersen", "designated_date": "2001-08-28", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genta, Inc.|200 Connell Drive|Berkeley Heights|New Jersey|7922|United States", "orphan_designation": { "original_text": "Treatment of chronic lymphocytic leukemia", "umls": "C0023434", "parsed_text": "Chronic Lymphocytic Leukemia" } }, { "pubchem_cid": 118984457, "pubchem_sid": [ "53788769", "135296278", "319431432", "349979899", "428438211", "160671608", "160693157", "252166481", "404771330" ], "inchikey": "IBXPAFBDJCXCDW-MHFPCNPESA-A", "generic_name": "oblimersen", "designated_date": "2001-08-28", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genta, Inc.|200 Connell Drive|Berkeley Heights|New Jersey|7922|United States", "orphan_designation": { "original_text": "Treatment of chronic lymphocytic leukemia", "umls": "C0023434", "parsed_text": "Chronic Lymphocytic Leukemia" } }, { "pubchem_cid": 59473233, "pubchem_sid": [ "175427696", "210275460", "210281108", "242638282", "249565580", "254759173", "255344044", "317223533", "319406912", "341558333", "347828420", "355039225", "373946850", "375577521", "384569053", "385651032", "386487482", "387060288", "434315235", "438493409", "440801805", "441107038" ], "inchikey": "XSMSNFMDVXXHGJ-UHFFFAOYSA-N", "generic_name": "entospletinib", "designated_date": "2014-04-14", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Kronos Bio, Inc.|1300 S. El Camino Real, Suite 300|San Mateo|California|94402|United States", "orphan_designation": { "original_text": "Treatment of chronic lymphocytic leukemia", "umls": "C0023434", "parsed_text": "Chronic Lymphocytic Leukemia" } }, { "pubchem_cid": 24821094, "pubchem_sid": [ "135268986", "136940589", "136961336", "152258295", "160647134", "163679284", "165245631", "171572071", "178103494", "184611713", "224184328", "251971042", "252088605", "252160516", "254762145", "274856561", "310264995", "312616233", "312808358", "315666679", "316579164", "316895237", "318163839", "319380131", "319467196", "329891104", "336298027", "340503210", "340521902", "340557335", "341143338", "346400508", "349983592", "350079530", "350095230", "354326201", "374060348", "375675186", "378130332", "381930724", "384439412", "385790189", "386627347", "405045803", "406521656", "419509191", "434314965", "436380298", "438493198", "438509205", "439576951", "439656277", "440799881", "441002856", "441117731", "135727459", "176250273", "252543441", "254759213", "255386749", "319530702", "319882825", "332832563", "335987367", "354269615", "354329136", "375577284", "375582916", "375758466", "402287765", "440794864", "221673009", "223471421" ], "inchikey": "XYFPWWZEPKGCCK-GOSISDBHSA-N", "generic_name": "ibrutinib", "trade_name": "IMBRUVICA", "designated_date": "2013-05-30", "designation_status": "Designated/Approved", "approved_labeled_indication": "Indicated for the treatment of patients with chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) and indicated for the treatment of patients with chronic lymphocytic leukemia (CLL)/small lymphocytic leukemia (SLL) with 17p deletion.", "marketing_approval_date": "2016-05-06", "exclusivity_end_date": "2023-05-06", "exclusivity_protected_indication": "For treatment of patients with small lymphocytic lymphoma (SLL)", "sponsor": "Pharmacyclics, LLC|995 E. Arques Avenue|Sunnyvale|California|94085|United States", "orphan_designation": { "original_text": "Treatment of small lymphocytic lymphoma", "umls": "C0023434", "parsed_text": "Small Lymphocytic Lymphoma" } }, { "pubchem_cid": 11625818, "pubchem_sid": [ "152258425", "160647261", "160778577", "163315619", "163352092", "163642790", "178103352", "223447422", "223471406", "249814532", "252088602", "252160790", "255343101", "255344072", "310264996", "312616234", "318163887", "319346791", "340567050", "341143280", "349988420", "350079569", "350095238", "355040500", "375577257", "375581350", "375646674", "376032529", "381351798", "384568574", "384572495", "385790056", "386627209", "402420173", "413664031", "433794854", "439574388", "439655793", "440802644", "440814879", "348350157", "377019309", "387233919", "405429520", "439976198", "375589932", "419592782" ], "inchikey": "IFSDAJWBUCMOAH-HNNXBMFYSA-N", "generic_name": "idelalisib", "trade_name": "Zydelig", "designated_date": "2013-10-15", "designation_status": "Designated/Approved", "approved_labeled_indication": "Zydelig is indicated, in combination with rituximab, for the treatment of patients with relapsed chronic lymphocytic leukemia (CLL) for whom rituximab alone would be considered appropriate therapy due to other co-morbidities; and Zydelig is indicated for the treatment of patients with relapsed small lymphocytic lymphoma (SLL) who have received at least two prior systemic therapies.", "marketing_approval_date": "2014-07-23", "exclusivity_end_date": "2021-07-23", "exclusivity_protected_indication": "(1) Relapsed chronic lymphocytic leukemia (CLL), in combination with rituximab, in patients for whom rituximab alone would be considered appropriate therapy due to other co-morbidities; and (2) Relapsed small lymphocytic lymphoma (SLL) in patients who have received at least two prior systemic therapies.", "sponsor": "Gilead Sciences, Inc.|199 E. Blaine St|Seattle|Washington|98102|United States", "orphan_designation": { "original_text": "Treatment of chronic lymphocytic leukemia and small lymphocytic lymphoma", "umls": [ "C0023434", "C0855095" ], "parsed_text": [ "Chronic Lymphocytic Leukemia", "Small Lymphocytic Lymphoma" ] } }, { "pubchem_cid": 122362431, "pubchem_sid": [ "48428179", "160708953", "319362407", "319447948", "336446047", "347713579", "347730488", "355040620", "436380206", "440703750", "162189538", "223401577", "319392679", "340503106", "371458039", "381938423", "406855326", "433844818", "165238038", "319413636", "385734854", "386571477", "434315357", "438493716", "26683815", "126666972", "251970946", "317223349", "319363145", "319407839", "346441064", "375581496", "126657609", "135266802", "196111308", "254761869", "313078258", "319402477", "341857720", "433987162", "434122147", "440807240", "318037860", "375895442" ], "inchikey": "ZVAGBRFUYHSUHA-XEKNBVSESA-N", "generic_name": "obatoclax mesylate", "designated_date": "2004-10-08", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Teva Branded Pharmaceutical Products R&D, Inc.|41 Moores Road|P. O. Box 4011|Frazer|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of chronic lymphocytic leukemia", "umls": "C0023434", "parsed_text": "Chronic Lymphocytic Leukemia" } }, { "pubchem_cid": 56843157, "pubchem_sid": [ "48428179", "160708953", "319362407", "319447948", "336446047", "347713579", "347730488", "355040620", "436380206", "440703750", "162189538", "223401577", "319392679", "340503106", "371458039", "381938423", "406855326", "433844818", "165238038", "319413636", "385734854", "386571477", "434315357", "438493716", "26683815", "126666972", "251970946", "317223349", "319363145", "319407839", "346441064", "375581496", "126657609", "135266802", "196111308", "254761869", "313078258", "319402477", "341857720", "433987162", "434122147", "440807240", "318037860", "375895442" ], "inchikey": "ZVAGBRFUYHSUHA-QUIKYCSHSA-N", "generic_name": "obatoclax mesylate", "designated_date": "2004-10-08", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Teva Branded Pharmaceutical Products R&D, Inc.|41 Moores Road|P. O. Box 4011|Frazer|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of chronic lymphocytic leukemia", "umls": "C0023434", "parsed_text": "Chronic Lymphocytic Leukemia" } }, { "pubchem_cid": 70700268, "pubchem_sid": [ "48428179", "160708953", "319362407", "319447948", "336446047", "347713579", "347730488", "355040620", "436380206", "440703750", "162189538", "223401577", "319392679", "340503106", "371458039", "381938423", "406855326", "433844818", "165238038", "319413636", "385734854", "386571477", "434315357", "438493716", "26683815", "126666972", "251970946", "317223349", "319363145", "319407839", "346441064", "375581496", "126657609", "135266802", "196111308", "254761869", "313078258", "319402477", "341857720", "433987162", "434122147", "440807240", "318037860", "375895442" ], "inchikey": "ZVAGBRFUYHSUHA-UHFFFAOYSA-N", "generic_name": "obatoclax mesylate", "designated_date": "2004-10-08", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Teva Branded Pharmaceutical Products R&D, Inc.|41 Moores Road|P. O. Box 4011|Frazer|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of chronic lymphocytic leukemia", "umls": "C0023434", "parsed_text": "Chronic Lymphocytic Leukemia" } }, { "pubchem_cid": 16727411, "pubchem_sid": [ "48428179", "160708953", "319362407", "319447948", "336446047", "347713579", "347730488", "355040620", "436380206", "440703750", "162189538", "223401577", "319392679", "340503106", "371458039", "381938423", "406855326", "433844818", "165238038", "319413636", "385734854", "386571477", "434315357", "438493716", "26683815", "126666972", "251970946", "317223349", "319363145", "319407839", "346441064", "375581496", "126657609", "135266802", "196111308", "254761869", "313078258", "319402477", "341857720", "433987162", "434122147", "440807240", "318037860", "375895442" ], "inchikey": "ZVAGBRFUYHSUHA-CFWFSBERSA-N", "generic_name": "obatoclax mesylate", "designated_date": "2004-10-08", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Teva Branded Pharmaceutical Products R&D, Inc.|41 Moores Road|P. O. Box 4011|Frazer|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of chronic lymphocytic leukemia", "umls": "C0023434", "parsed_text": "Chronic Lymphocytic Leukemia" } }, { "pubchem_cid": 71816154, "pubchem_sid": [ "48428179", "160708953", "319362407", "319447948", "336446047", "347713579", "347730488", "355040620", "436380206", "440703750", "162189538", "223401577", "319392679", "340503106", "371458039", "381938423", "406855326", "433844818", "165238038", "319413636", "385734854", "386571477", "434315357", "438493716", "26683815", "126666972", "251970946", "317223349", "319363145", "319407839", "346441064", "375581496", "126657609", "135266802", "196111308", "254761869", "313078258", "319402477", "341857720", "433987162", "434122147", "440807240", "318037860", "375895442" ], "inchikey": "ZVAGBRFUYHSUHA-RKMKAUCSSA-N", "generic_name": "obatoclax mesylate", "designated_date": "2004-10-08", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Teva Branded Pharmaceutical Products R&D, Inc.|41 Moores Road|P. O. Box 4011|Frazer|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of chronic lymphocytic leukemia", "umls": "C0023434", "parsed_text": "Chronic Lymphocytic Leukemia" } }, { "pubchem_cid": 46930996, "pubchem_sid": [ "48428179", "160708953", "319362407", "319447948", "336446047", "347713579", "347730488", "355040620", "436380206", "440703750", "162189538", "223401577", "319392679", "340503106", "371458039", "381938423", "406855326", "433844818", "165238038", "319413636", "385734854", "386571477", "434315357", "438493716", "26683815", "126666972", "251970946", "317223349", "319363145", "319407839", "346441064", "375581496", "126657609", "135266802", "196111308", "254761869", "313078258", "319402477", "341857720", "433987162", "434122147", "440807240", "318037860", "375895442" ], "inchikey": "ZVAGBRFUYHSUHA-VLJPQPJMSA-N", "generic_name": "obatoclax mesylate", "designated_date": "2004-10-08", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Teva Branded Pharmaceutical Products R&D, Inc.|41 Moores Road|P. O. Box 4011|Frazer|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of chronic lymphocytic leukemia", "umls": "C0023434", "parsed_text": "Chronic Lymphocytic Leukemia" } }, { "pubchem_cid": 16681698, "pubchem_sid": [ "48428179", "160708953", "319362407", "319447948", "336446047", "347713579", "347730488", "355040620", "436380206", "440703750", "162189538", "223401577", "319392679", "340503106", "371458039", "381938423", "406855326", "433844818", "165238038", "319413636", "385734854", "386571477", "434315357", "438493716", "26683815", "126666972", "251970946", "317223349", "319363145", "319407839", "346441064", "375581496", "126657609", "135266802", "196111308", "254761869", "313078258", "319402477", "341857720", "433987162", "434122147", "440807240", "318037860", "375895442" ], "inchikey": "ZVAGBRFUYHSUHA-LZOXOEDVSA-N", "generic_name": "obatoclax mesylate", "designated_date": "2004-10-08", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Teva Branded Pharmaceutical Products R&D, Inc.|41 Moores Road|P. O. Box 4011|Frazer|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of chronic lymphocytic leukemia", "umls": "C0023434", "parsed_text": "Chronic Lymphocytic Leukemia" } }, { "pubchem_cid": 24821094, "pubchem_sid": [ "135268986", "136940589", "136961336", "152258295", "160647134", "163679284", "165245631", "171572071", "178103494", "184611713", "224184328", "251971042", "252088605", "252160516", "254762145", "274856561", "310264995", "312616233", "312808358", "315666679", "316579164", "316895237", "318163839", "319380131", "319467196", "329891104", "336298027", "340503210", "340521902", "340557335", "341143338", "346400508", "349983592", "350079530", "350095230", "354326201", "374060348", "375675186", "378130332", "381930724", "384439412", "385790189", "386627347", "405045803", "406521656", "419509191", "434314965", "436380298", "438493198", "438509205", "439576951", "439656277", "440799881", "441002856", "441117731", "135727459", "176250273", "252543441", "254759213", "255386749", "319530702", "319882825", "332832563", "335987367", "354269615", "354329136", "375577284", "375582916", "375758466", "402287765", "440794864", "221673009", "223471421" ], "inchikey": "XYFPWWZEPKGCCK-GOSISDBHSA-N", "generic_name": "ibrutinib", "trade_name": "Imbruvica", "designated_date": "2012-04-06", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of patients with chronic lymphocytic leukemia (CLL) who have received at least one prior therapy and treatment of patients with chronic lymphocytic leukemia (CLL) with 17p deletion", "marketing_approval_date": "2014-07-28", "exclusivity_end_date": "2021-07-28", "exclusivity_protected_indication": "Treatment of chronic lymphocytic leukemia with 17p deletion who have not received at least one prior therapy", "sponsor": "Pharmacyclics, LLC|995 E. Arques Avenue|Sunnyvale|California|94085|United States", "orphan_designation": { "original_text": "Treatment of chronic lymphocytic leukemia (CLL)", "umls": "C0023434", "parsed_text": "Chronic Lymphocytic Leukemia" } }, { "pubchem_cid": 24821094, "pubchem_sid": [ "135268986", "136940589", "136961336", "152258295", "160647134", "163679284", "165245631", "171572071", "178103494", "184611713", "224184328", "251971042", "252088605", "252160516", "254762145", "274856561", "310264995", "312616233", "312808358", "315666679", "316579164", "316895237", "318163839", "319380131", "319467196", "329891104", "336298027", "340503210", "340521902", "340557335", "341143338", "346400508", "349983592", "350079530", "350095230", "354326201", "374060348", "375675186", "378130332", "381930724", "384439412", "385790189", "386627347", "405045803", "406521656", "419509191", "434314965", "436380298", "438493198", "438509205", "439576951", "439656277", "440799881", "441002856", "441117731", "135727459", "176250273", "252543441", "254759213", "255386749", "319530702", "319882825", "332832563", "335987367", "354269615", "354329136", "375577284", "375582916", "375758466", "402287765", "440794864", "221673009", "223471421" ], "inchikey": "XYFPWWZEPKGCCK-GOSISDBHSA-N", "generic_name": "ibrutinib", "trade_name": "Imbruvica", "designated_date": "2012-04-06", "designation_status": "Designated/Approved", "approved_labeled_indication": "Indicated for the treatment of patients with chronic lymphocytic leukemia", "marketing_approval_date": "2016-03-04", "exclusivity_end_date": "2023-03-04", "exclusivity_protected_indication": "Indicated for the treatment of patients with chronic lymphocytic leukemia without 17p deletion who have not received at least one prior therapy (first line therapy).", "sponsor": "Pharmacyclics, LLC|995 E. Arques Avenue|Sunnyvale|California|94085|United States", "orphan_designation": { "original_text": "Treatment of chronic lymphocytic leukemia (CLL)", "umls": "C0023434", "parsed_text": "Chronic Lymphocytic Leukemia" } }, { "pubchem_cid": 11625818, "pubchem_sid": [ "152258425", "160647261", "160778577", "163315619", "163352092", "163642790", "178103352", "223447422", "223471406", "249814532", "252088602", "252160790", "255343101", "255344072", "310264996", "312616234", "318163887", "319346791", "340567050", "341143280", "349988420", "350079569", "350095238", "355040500", "375577257", "375581350", "375646674", "376032529", "381351798", "384568574", "384572495", "385790056", "386627209", "402420173", "413664031", "433794854", "439574388", "439655793", "440802644", "440814879", "348350157", "377019309", "387233919", "405429520", "439976198", "375589932", "419592782" ], "inchikey": "IFSDAJWBUCMOAH-HNNXBMFYSA-N", "generic_name": "idelalisib", "designated_date": "2011-08-25", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Gilead Sciences, Inc.|199 E. Blaine St.|Seattle|Washington|98102|United States", "orphan_designation": { "original_text": "Treatment of chronic lymphocytic leukemia", "umls": "C0023434", "parsed_text": "Chronic Lymphocytic Leukemia" } }, { "pubchem_cid": 20279, "pubchem_sid": [ "610461", "7978957", "46386544", "46504588", "48415788", "49865077", "50446026", "57309572", "87323981", "92713835", "99218181", "99431527", "103602769", "126624559", "126655830", "134984314", "136342484", "144204385", "144210913", "160963590", "162175079", "178101501", "204377291", "210274895", "210280530", "211535115", "223380222", "224247735", "241085789", "242082983", "251885699", "251915935", "251917283", "252158753", "252215169", "252347497", "252619821", "254760927", "274134026", "310270378", "312227998", "312694695", "313080496", "315673485", "316966522", "318151990", "318160647", "319076602", "319447889", "319553934", "329857026", "329977410", "340521602", "340546980", "341137036", "341139685", "341832128", "347728173", "347912502", "348282950", "348397188", "349053600", "349979485", "354266678", "355039566", "355047636", "363662545", "363907320", "374031390", "375204717", "375583144", "375636898", "376044850", "377309807", "382160971", "384568011", "384571752", "385784288", "386621165", "387140656", "404623361", "419554287", "433795555", "434249230", "436380392", "438452774", "438474082", "440234228", "440790996", "441049356", "441113473", "405818", "50446025", "126663837", "136374294", "222524101", "340503315", "346441239", "381931435", "387196488", "387209568", "406855832", "26758460", "163688376", "318049042", "160871152", "162258727", "50446027", "50446028", "50446029", "126599404", "252215168", "255369934", "318695226", "441079440" ], "inchikey": "PTOAARAWEBMLNO-KVQBGUIXSA-N", "generic_name": "cladribine", "designated_date": "1990-12-31", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Janssen Research & Development, LLC|920 Route 202 South|Raritan|New Jersey|8869|United States", "orphan_designation": { "original_text": "Treatment of chronic lymphocytic leukemia", "umls": "C0023434", "parsed_text": "Chronic Lymphocytic Leukemia" } }, { "pubchem_cid": 24821094, "pubchem_sid": [ "135268986", "136940589", "136961336", "152258295", "160647134", "163679284", "165245631", "171572071", "178103494", "184611713", "224184328", "251971042", "252088605", "252160516", "254762145", "274856561", "310264995", "312616233", "312808358", "315666679", "316579164", "316895237", "318163839", "319380131", "319467196", "329891104", "336298027", "340503210", "340521902", "340557335", "341143338", "346400508", "349983592", "350079530", "350095230", "354326201", "374060348", "375675186", "378130332", "381930724", "384439412", "385790189", "386627347", "405045803", "406521656", "419509191", "434314965", "436380298", "438493198", "438509205", "439576951", "439656277", "440799881", "441002856", "441117731", "135727459", "176250273", "252543441", "254759213", "255386749", "319530702", "319882825", "332832563", "335987367", "354269615", "354329136", "375577284", "375582916", "375758466", "402287765", "440794864", "221673009", "223471421" ], "inchikey": "XYFPWWZEPKGCCK-GOSISDBHSA-N", "generic_name": "ibrutinib", "designated_date": "2012-03-27", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Pharmacyclics, LLC|995 E. Arques Avenue|Sunnyvale|California|94085|United States", "orphan_designation": { "original_text": "Treatment of chronic lymphocytic leukemia (CLL).", "umls": "C0023434", "parsed_text": "Chronic Lymphocytic Leukemia" } }, { "pubchem_cid": 49846579, "pubchem_sid": [ "85332829", "163620947", "163643283", "163686269", "194147759", "252160763", "252166530", "252819663", "312616251", "318163982", "319392910", "341097094", "341188650", "347827991", "350079438", "354272751", "355039871", "355362240", "374072973", "375577083", "378132095", "385646633", "386483019", "387120282", "387232944", "402288047", "440801145" ], "inchikey": "LQBVNQSMGBZMKD-UHFFFAOYSA-N", "generic_name": "venetoclax", "trade_name": "VENCLEXTA", "designated_date": "2012-09-20", "designation_status": "Designated/Approved", "approved_labeled_indication": "VENCLEXTA (venetoclax) indicated for the treatment of patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), with or without 17p deletion, who have received at least one prior therapy.", "marketing_approval_date": "2018-06-08", "exclusivity_end_date": "2025-06-08", "exclusivity_protected_indication": "For treatment of patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), without 17p deletion, who have received at least one prior therapy.", "sponsor": "AbbVie, Inc|1 North Waukegan Road|Dept. PA72, Bldg. AP30|North Chicago|Illinois|60064|United States", "orphan_designation": { "original_text": "Treatment of chronic lymphocytic leukemia", "umls": "C0023434", "parsed_text": "Chronic Lymphocytic Leukemia" } }, { "pubchem_cid": 49846579, "pubchem_sid": [ "85332829", "163620947", "163643283", "163686269", "194147759", "252160763", "252166530", "252819663", "312616251", "318163982", "319392910", "341097094", "341188650", "347827991", "350079438", "354272751", "355039871", "355362240", "374072973", "375577083", "378132095", "385646633", "386483019", "387120282", "387232944", "402288047", "440801145" ], "inchikey": "LQBVNQSMGBZMKD-UHFFFAOYSA-N", "generic_name": "venetoclax", "trade_name": "VENCLEXTA", "designated_date": "2012-09-20", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of patients with chronic lymphocytic leukemia (CLL) with 17p deletion, as detected by an FDA approved test, who have received at least one prior therapy", "marketing_approval_date": "2016-04-11", "exclusivity_end_date": "2023-04-11", "exclusivity_protected_indication": "Treatment of patients with chronic lymphocytic leukemia (CLL) with 17p deletion, as detected by an FDA approved test, who have received at least one prior therapy", "sponsor": "AbbVie, Inc|1 North Waukegan Road|Dept. PA72, Bldg. AP30|North Chicago|Illinois|60064|United States", "orphan_designation": { "original_text": "Treatment of chronic lymphocytic leukemia", "umls": "C0023434", "parsed_text": "Chronic Lymphocytic Leukemia" } }, { "pubchem_cid": 667528, "pubchem_sid": [ "135192627", "274105068", "319243621", "384571276" ], "inchikey": "NNYBQONXHNTVIJ-QGZVFWFLSA-N", "generic_name": "R-etodolac", "designated_date": "2002-12-09", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Cephalon, Inc.|41 Moores Road|Frazer|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of chronic lymphocytic leukemia", "umls": "C0023434", "parsed_text": "Chronic Lymphocytic Leukemia" } }, { "pubchem_cid": 49846579, "pubchem_sid": [ "85332829", "163620947", "163643283", "163686269", "194147759", "252160763", "252166530", "252819663", "312616251", "318163982", "319392910", "341097094", "341188650", "347827991", "350079438", "354272751", "355039871", "355362240", "374072973", "375577083", "378132095", "385646633", "386483019", "387120282", "387232944", "402288047", "440801145" ], "inchikey": "LQBVNQSMGBZMKD-UHFFFAOYSA-N", "generic_name": "venetoclax", "trade_name": "VENCLEXTA", "designated_date": "2012-09-20", "designation_status": "Designated/Approved", "approved_labeled_indication": "VENCLEXTA is indicated for the treatment of adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).", "marketing_approval_date": "2019-05-15", "exclusivity_end_date": "2026-05-15", "exclusivity_protected_indication": "Treatment of previously untreated adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL)", "sponsor": "AbbVie, Inc|1 North Waukegan Road|Dept. 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"C0024301", "C0024419", "C0242647", "C0349632", "C0855139", "C1955690" ], "parsed_text": [ "follicular lymphoma", "lymphoplasmacytic lymphoma", "Mucosa-associated lymphoma", "Splenic marginal zone lymphoma", "Nodal Marginal Zone Lymphoma", "Extranodal marginal zone B-cell lymphoma" ] } }, { "pubchem_cid": 24821094, "pubchem_sid": [ "135268986", "136940589", "136961336", "152258295", "160647134", "163679284", "165245631", "171572071", "178103494", "184611713", "224184328", "251971042", "252088605", "252160516", "254762145", "274856561", "310264995", "312616233", "312808358", "315666679", "316579164", "316895237", "318163839", "319380131", "319467196", "329891104", "336298027", "340503210", "340521902", "340557335", "341143338", "346400508", "349983592", "350079530", "350095230", "354326201", "374060348", "375675186", "378130332", "381930724", "384439412", "385790189", "386627347", "405045803", "406521656", "419509191", "434314965", "436380298", "438493198", "438509205", "439576951", "439656277", "440799881", "441002856", "441117731", "135727459", "176250273", "252543441", "254759213", "255386749", "319530702", "319882825", "332832563", "335987367", "354269615", "354329136", "375577284", "375582916", "375758466", "402287765", "440794864", "221673009", "223471421" ], "inchikey": "XYFPWWZEPKGCCK-GOSISDBHSA-N", "generic_name": "ibrutinib", "trade_name": "Imbruvica", "designated_date": "2013-10-15", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of patients with Waldenstrom's macroglobulinemia", "marketing_approval_date": "2015-01-29", "exclusivity_end_date": "2022-01-29", "exclusivity_protected_indication": "Treatment of patients with Waldenstrom's macroglobulinemia", "sponsor": "Pharmacyclics, LLC|995 E. Arques Avenue|Sunnyvale|California|94085|United States", "orphan_designation": { "original_text": "Treatment of Waldenstrom's macroglobulinemia", "umls": "C0024419", "parsed_text": "Waldenstrom's Macroglobulinemia" } }, { "pubchem_cid": 6442177, "pubchem_sid": [ "12014881", "46505248", "50044230", "56311446", "56312241", "103987301", "104178855", "135156085", "136929859", "143493384", "144206063", "152236848", "152258132", "160645716", "160646971", "162189189", "174527790", "178102512", "203355779", "224627271", "254760057", "255345989", "312244639", "312616346", "315678225", "318161060", "319552082", "336851372", "341141968", "341852299", "342465019", "354270236", "374015776", "384431653", "385787399", "386624465", "405068250", "419533960", "433982778", "438477198", "439656880", "53788326", "126644878", "126662810", "251915630", "251916980", "251971409", "272722577", "283537548", "313053725", "313080931", "316538449", "319321081", "319434589", "319446374", "349988787", "355040747", "381745152", "438518916", "439390629", "440791123", "162224766", "184816442", "211535430", "252090889", "319185451", "319393691", "341101126", "347241527", "348349940", "354283816", "375577172", "383424876", "387099540", "387099541", "387099543", "428401474", "433836861", "252215408", "313076696", "313086632", "319423877", "335978376", "375446828", "375767367", "376146480", "384568320", "384572403", "126627402", "160964828", "162262965", "316899720", "319363762", "319423164", "347743018", "348283957", "422710143", "428402521", "433981240", "434127016", "48430111", "51071324", "162011845", "162250622", "242060298", "252451493", "273007467", "277424818", "316966825", "319361597", "319373039", "319379163", "319392681", "319397956", "319399231", "319407864", "319407905", "319408884", "319409126", "319409350", "319422960", "319423130", "319430973", "319431561", "340503263", "348273726", "364196435", "375228315", "375563204", "375636396", "406696943", "428402226", "434122881", "438635435", "441074816" ], "inchikey": "HKVAMNSJSFKALM-GKUWKFKPSA-N", "generic_name": "everolimus", "designated_date": "2010-09-16", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Novartis Pharmaceuticals Corp.|One Health Plaza, 315/5450A|East Hanover|New Jersey|7936|United States", "orphan_designation": { "original_text": "Treatment of Waldenstrom macroglobulinemia (also known as lymphoplasmacytic lymphoma)", "umls": [ "C0024419", "C0334633" ], "parsed_text": [ "Waldenstrom Macroglobulinemia", "Lymphoplasmacytic Lymphoma" ] } }, { "pubchem_cid": 135565884, "pubchem_sid": [ "348351323", "355203029", "355203141", "363894155", "374328055", "375561452", "377020097", "377360318", "377602297", "381973893", "404607980", "404772479", "406529429", "406866248", "433866630", "433909683", "434314706", "438494641", "438790653", "440796275", "440820038", "404647162" ], "inchikey": "RNOAOAWBMHREKO-QFIPXVFZSA-N", "generic_name": "Zanubrutinib", "designated_date": "2016-06-29", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "BeiGene USA, Inc.|2955 Campus Drive|Suite 200|San Mateo|California|94403|United States", "orphan_designation": { "original_text": "Treatment of Waldenstrom's macroglobulinemia", "umls": "C0024419", "parsed_text": "Waldenstrom's Macroglobulinemia" } }, { "pubchem_cid": 25067547, "pubchem_sid": [ "136350027", "160692475", "162108668", "163347631", "210274759", "210280392", "252472838", "252827397", "254762142", "255344186", "277425499", "317223220", "319382165", "319552889", "329976724", "341190026", "347713536", "347746845", "347828311", "348273480", "349482789", "354270283", "355039637", "375590575", "378132092", "384568748", "384572731", "385650872", "386487322", "439576227", "440681573", "440796054", "441063502", "363748265", "405512232", "440058888", "332853970", "349976380", "375578098", "440703144" ], "inchikey": "SWZXEVABPLUDIO-WSZYKNRRSA-N", "generic_name": "oprozomib", "designated_date": "2014-08-25", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Onyx Therapeutics, Inc.|249 E. Grand Avenue|South San Francisco|California|94080|United States", "orphan_designation": { "original_text": "Treatment of Waldenstrom's macroglobulinemia", "umls": "C0024419", "parsed_text": "Waldenstrom's Macroglobulinemia" } }, { "pubchem_cid": 11625818, "pubchem_sid": [ "152258425", "160647261", "160778577", "163315619", "163352092", "163642790", "178103352", "223447422", "223471406", "249814532", "252088602", "252160790", "255343101", "255344072", "310264996", "312616234", "318163887", "319346791", "340567050", "341143280", "349988420", "350079569", "350095238", "355040500", "375577257", "375581350", "375646674", "376032529", "381351798", "384568574", "384572495", "385790056", "386627209", "402420173", "413664031", "433794854", "439574388", "439655793", "440802644", "440814879", "348350157", "377019309", "387233919", "405429520", "439976198", "375589932", "419592782" ], "inchikey": "IFSDAJWBUCMOAH-HNNXBMFYSA-N", "generic_name": "idelalisib", "designated_date": "2013-09-26", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Gilead Sciences, Inc.|199 E. Blaine St|Seattle|Washington|98102|United States", "orphan_designation": { "original_text": "Treatment of lymphoplasmacytic lymphoma with or without Walenstrom's macroglobulinemia", "umls": [ "C0024419", "C0334633" ], "parsed_text": [ "lymphoplasmacytic lymphoma", "Lymphoplasmacytic Lymphoma" ] } } ] }, { "_id": "UMLS:C1853926", "fda_orphan_drug": [ { "pubchem_cid": 14017587, "pubchem_sid": [ "134973567", "144205940", "160692729", "178101361", "315684541", "347828146", "355036894", "387112653", "605414", "319223490", "348274337", "434132560", "8145622", "175443104", "319221644", "103400157", "250135112", "318692710", "319223476", "319391027", "319407457", "319407925", "319429313" ], "inchikey": "KBGAYAKRZNYFFG-BOHATCBPSA-N", "generic_name": "aceneuramic acid", "designated_date": "2011-09-23", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Ultragenyx Pharmaceutical, Inc.|60 Leveroni Court|Suite 200|Novato|California|94949|United States", "orphan_designation": { "original_text": "Treatment of hereditary inclusion body myopathy", "umls": "C1853926", "parsed_text": "Hereditary inclusion body myopathy" } } ] }, { "_id": "UMLS:C0572025", "fda_orphan_drug": [ { "pubchem_cid": 12035, "pubchem_sid": [ "8144902", "12013391", "48413148", "48422870", "49856254", "53790587", "56310757", "56311342", "56311343", "56311724", "56314540", "92714687", "99443235", "126623942", "126655263", "134978062", "136348796", "160967850", "174478612", "184818743", "196106914", "223741118", "249926182", "275228731", "318160103", "319070271", "321939574", "342398324", "347912801", "348275440", "349974913", "387141259", "404834430", "433777488", "409483", "85083256", "124994937", "125321798", "315683346", "341139678", "386304089", "387141261", "127268235", "127268236", "223710691" ], "inchikey": "PWKSKIMOESPYIA-BYPYZUCNSA-N", "generic_name": "Acetylcysteine", "designated_date": "1987-08-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Intravenous treatment of patients presenting with moderate to severe acetaminophen overdose.", "umls": "C0572025", "parsed_text": "Acetaminophen overdose" } }, { "pubchem_cid": 12035, "pubchem_sid": [ "8144902", "12013391", "48413148", "48422870", "49856254", "53790587", "56310757", "56311342", "56311343", "56311724", "56314540", "92714687", "99443235", "126623942", "126655263", "134978062", "136348796", "160967850", "174478612", "184818743", "196106914", "223741118", "249926182", "275228731", "318160103", "319070271", "321939574", "342398324", "347912801", "348275440", "349974913", "387141259", "404834430", "433777488", "409483", "85083256", "124994937", "125321798", "315683346", "341139678", "386304089", "387141261", "127268235", "127268236", "223710691" ], "inchikey": "PWKSKIMOESPYIA-BYPYZUCNSA-N", "generic_name": "acetylcysteine", "trade_name": "Acetadote", "designated_date": "2001-10-19", "designation_status": "Designated/Approved", "approved_labeled_indication": "For the use of Acetadote(registered TM) Injection, administered intravenously within 8 to 10 hours after ingestion of a potentially heptotoxic quantity of acetaminophen, to prevent or lessen hepatic injur", "marketing_approval_date": "2004-01-23", "exclusivity_end_date": "2011-01-23", "sponsor": "Cumberland Pharmaceuticals, Inc.|209 10th Street South|Suite 332|Nashville|Tennessee|37203|United States", "orphan_designation": { "original_text": "For the intravenous treatment of moderate to severe acetaminophen overdose", "umls": "C0572025", "parsed_text": "Acetaminophen overdose" } }, { "pubchem_cid": 896, "pubchem_sid": [ "2961", "4752", "105815", "410775", "798411", "841969", "7979886", "8144655", "8149639", "10321385", "17388930", "17389536", "26759121", "29204367", "46500483", "46509101", "46509793", "48416204", "48421927", "48423636", "48425270", "48425271", "49718192", "49747966", "49747967", "49855751", "50086687", "50105414", "50105416", "51951460", "53790088", "56310582", "56313759", "85087484", "87573071", "92298010", "92304139", "93166070", "103166463", "103913882", "117498614", "126632809", "126659037", "134972795", "135651518", "135697782", "136340363", "136367361", "143489736", "144203746", "144208725", "144213243", "152238569", "160810185", "160964399", "162170568", "163409810", "163564741", "163687728", "170465589", "172765315", "174549181", "185995573", "196109749", "198992727", "210279182", "210281505", "223377401", "224416925", "242059708", "249719786", "250042203", "252157348", "252306177", "252401243", "268735744", "273002888", "274190216", "274955123", "277309203", "310276097", "312226169", "312344912", "312695553", "312811195", "313081423", "315445488", "315673152", "316543344", "316897611", "316965872", "318152049", "318161539", "318692702", "319554558", "340074136", "340475143", "340509719", "340515021", "340516339", "340530802", "341139750", "341834004", "346427101", "347913722", "348273554", "348275256", "349280771", "349984554", "355049629", "363603067", "363901422", "368505455", "374016679", "375131258", "375666571", "375749969", "376193084", "376220001", "376227657", "377309456", "381337402", "381354891", "382164415", "384567881", "384571550", "385643487", "386191640", "386268896", "386393032", "386479790", "387022037", "387086053", "404425814", "404617308", "404754346", "419507128", "433774812", "433775326", "433777469", "433777470", "433777471", "433778009", "433778862", "433780693", "434299599", "438462485", "438473353", "438473354", "438517528", "438649698", "440065800", "51951461", "51951462", "349328807", "441076632" ], "inchikey": "DRLFMBDRBRZALE-UHFFFAOYSA-N", "generic_name": "melatonin", "designated_date": "2017-03-02", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "WORPHMED Srl|Via Malachia Marchesi de Taddei, 21|Milan|Lombardy|Italy", "orphan_designation": { "original_text": "Treatment of acute acetaminophen overdose", "umls": "C0572025", "parsed_text": "Acetaminophen overdose" } }, { "pubchem_cid": 3406, "pubchem_sid": [ "10039", "10534897", "46508566", "50029489", "50139274", "53788599", "103406649", "134986543", "136374275", "144076358", "160964546", "249944732", "254803962", "310273879", "312228720", "312323634", "312695596", "316396921", "318161167", "318238871", "319062298", "319556599", "341138428", "348283792", "348397689", "349296205", "349976863", "354284047", "355037895", "376199694", "376229587", "378166687", "384570715", "384571543", "385632821", "386264685", "386618406", "387148469", "387234097", "404754818", "406969750", "434132433", "434252190", "438456525", "440770185", "440791020", "57260551", "77722658", "92304249", "103060282", "419546229", "441080356" ], "inchikey": "RIKMMFOAQPJVMX-UHFFFAOYSA-N", "generic_name": "Fomepizole", "designated_date": "2021-03-02", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Denver Health - Rocky Mountain Poison & Drug Safety|777 Bannock St. MC 0180|Denver|Colorado|80204|United States", "orphan_designation": { "original_text": "Treatment of acetaminophen overdose", "umls": "C0572025", "parsed_text": "Acetaminophen overdose" } }, { "pubchem_sid": 404719767, "generic_name": "calmangafodipir", "designated_date": "2019-03-15", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "PledPharma AB|Grev Turegatan 11c|Stockholm|Sweden", "orphan_designation": { "original_text": "Treatment of acetaminophen overdose.", "umls": "C0572025", "parsed_text": "Acetaminophen overdose" } }, { "pubchem_sid": 381608948, "generic_name": "calmangafodipir", "designated_date": "2019-03-15", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "PledPharma AB|Grev Turegatan 11c|Stockholm|Sweden", "orphan_designation": { "original_text": "Treatment of acetaminophen overdose.", "umls": "C0572025", "parsed_text": "Acetaminophen overdose" } }, { "pubchem_sid": 134222734, "generic_name": "calmangafodipir", "designated_date": "2019-03-15", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "PledPharma AB|Grev Turegatan 11c|Stockholm|Sweden", "orphan_designation": { "original_text": "Treatment of acetaminophen overdose.", "umls": "C0572025", "parsed_text": "Acetaminophen overdose" } } ] }, { "_id": "UMLS:C0041296", "fda_orphan_drug": [ { "pubchem_cid": 5391612, "pubchem_sid": [ "50031719", "53003098", "134989922", "275413500", "319303819", "341237753", "385642726", "386479012", "411048794", "434139107", "438444555", "53003099", "144205530", "160687910", "164235730", "315681848", "53003097", "405449705", "439996369" ], "inchikey": "MDFXJBQEWLCGHP-MFOYZWKCSA-N", "generic_name": "Aconiazide", "designated_date": "1988-06-20", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Lincoln Diagnostics|P.O. Box 1128|Decatur|Illinois|62525|United States", "orphan_designation": { "original_text": "Treatment of tuberculosis.", "umls": "C0041296", "parsed_text": "Tuberculosis" } }, { "pubchem_cid": 4649, "pubchem_sid": [ "68777", "10531859", "26744216", "46505572", "48415551", "50045157", "53786895", "103340993", "124765999", "134972628", "160963581", "250013857", "312227989", "312695380", "315673280", "318161897", "319062987", "348282941", "349976181", "355120164", "385650419", "386486860", "387171782", "404613238", "434140923", "438448444", "438474464", "127324923", "127324924", "341150957", "348058691", "405477295", "440023954", "50071360", "57288744", "341860785" ], "inchikey": "WUBBRNOQWQTFEX-UHFFFAOYSA-N", "generic_name": "Aminosalicylic acid", "trade_name": "Paser Granules", "designated_date": "1992-02-19", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of tuberculosis", "marketing_approval_date": "1994-06-30", "exclusivity_end_date": "2001-06-30", "sponsor": "Jacobus Pharmaceutical Company|37 Cleveland Lane|P.O. Box 5290|Princeton|New Jersey|8540|United States", "orphan_designation": { "original_text": "Treatment of tuberculosis infections", "umls": "C0041296", "parsed_text": "Tuberculosis" } }, { "pubchem_cid": 165580, "pubchem_sid": [ "4090", "600745", "7889708", "7980252", "26707296", "26737833", "29195990", "29196150", "46505391", "48416383", "49693302", "50139565", "56313066", "87225335", "104234223", "123059634", "123059983", "127294526", "127294527", "127294528", "127294529", "134213896", "134213903", "134988691", "136349017", "136349597", "144186824", "152138149", "152138154", "152199472", "152199479", "160650099", "160650104", "160654719", "160964689", "163426273", "163426276", "163426451", "163621667", "186528375", "186528379", "223856902", "249807412", "252211031", "254815781", "254815785", "254815789", "254815793", "254815871", "275414335", "312228832", "315673994", "318161913", "319178215", "340127218", "340127222", "340127232", "341818230", "348283923", "348398276", "349889861", "349978795", "355050839", "355051843", "355156727", "363591531", "375573433", "375633765", "376219175", "376228649", "381339080", "382369514", "382370001", "382370004", "382370008", "383861753", "385785548", "386622510", "387160850", "429321039", "429321269", "433788270", "433788271", "433986357", "434303472", "438484962", "438622470", "440066623", "440066628", "440066632", "440066640", "440066644", "440066649", "440066652", "440066655", "440066660", "440066666", "440066677", "440066683", "440066882", "440066886", "440066969", "440067229", "440067260", "440068530", "440068536", "440068562", "440068566", "440704221", "440704245", "440704248", "440704256", "440704266", "440704292", "440786583", "440786672", "440786681", "440786690", "440786769", "440786877", "440786880", "440786908", "440786913", "440786919", "440790882", "85182178", "341825573", "381832735", "404766028", "50047958", "124766130", "249900544", "319079359", "405501466", "440048122", "85286193", "196107155", "319391947", "341825460", "135263911", "242081226", "252211030", "255511865", "341140988", "363603260", "386973149", "387119029", "440786878", "441075047" ], "inchikey": "UOZODPSAJZTQNH-LSWIJEOBSA-N", "generic_name": "Aminosidine", "designated_date": "1993-05-14", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Kanyok, Thomas P. Pharm.D.|University Of Illinois At Chicago College of Pharm|833 South Wood Street (m/c886) Rm. 176|Chicago|Illinois|60612|United States", "orphan_designation": { "original_text": "Treatment of tuberculosis.", "umls": "C0041296", "parsed_text": "Tuberculosis" } }, { "pubchem_cid": 5388906, "pubchem_sid": [ "3726039", "81044374", "103512862", "135262331", "160962911", "162220394", "175427143", "210275153", "210280792", "223471403", "251903327", "312245191", "312695738", "313071201", "318163806", "319303796", "319553105", "332832916", "340580285", "349350823", "355039397", "363907636", "364122776", "374095645", "376033458", "381329027", "381353742", "382165386", "383470764", "384443607", "385641668", "386477932", "404639802", "419501036", "433774167", "433774168", "433774173", "433774174", "434122231", "434142755", "434219419", "438451747", "439480510", "439575872", "440747214", "251971038", "347714031", "374243579", "378132408", "402287578", "405505091", "440051747", "172112135", "254761930", "319343172", "319346298", "319391295", "319438621", "341141918", "375583133", "375664536", "383409863", "251903326", "349995459", "441072047" ], "inchikey": "QUIJNHUBAXPXFS-XLJNKUFUSA-N", "generic_name": "bedaquiline", "trade_name": "SIRTURO", "designated_date": "2005-01-10", "designation_status": "Designated/Approved", "approved_labeled_indication": "SIRTURO is a diarylquinoline antimycobacterial drug indicated as part of combination therapy in adult and pediatric patients (5 years and older and weighing at least 15 kg) with pulmonary multi-drug resistant tuberculosis (MDR-TB). Reserve SIRTURO for use when an effective treatment regimen cannot otherwise be provided.", "marketing_approval_date": "2020-05-27", "exclusivity_end_date": "2027-05-27", "exclusivity_protected_indication": "As part of combination therapy in the treatment of pediatric patients 5 years and older to less than 12 years of age and weighing at least 15 kg with pulmonary multi-drug resistant tuberculosis (MDR-TB)", "sponsor": "Janssen Research & Development, LLC|920 Route 202 South|Raritan|New Jersey|8869|United States", "orphan_designation": { "original_text": "Treatment of active tuberculosis", "umls": "C0041296", "parsed_text": "Active Tuberculosis" } }, { "pubchem_cid": 5388906, "pubchem_sid": [ "3726039", "81044374", "103512862", "135262331", "160962911", "162220394", "175427143", "210275153", "210280792", "223471403", "251903327", "312245191", "312695738", "313071201", "318163806", "319303796", "319553105", "332832916", "340580285", "349350823", "355039397", "363907636", "364122776", "374095645", "376033458", "381329027", "381353742", "382165386", "383470764", "384443607", "385641668", "386477932", "404639802", "419501036", "433774167", "433774168", "433774173", "433774174", "434122231", "434142755", "434219419", "438451747", "439480510", "439575872", "440747214", "251971038", "347714031", "374243579", "378132408", "402287578", "405505091", "440051747", "172112135", "254761930", "319343172", "319346298", "319391295", "319438621", "341141918", "375583133", "375664536", "383409863", "251903326", "349995459", "441072047" ], "inchikey": "QUIJNHUBAXPXFS-XLJNKUFUSA-N", "generic_name": "bedaquiline", "trade_name": "SIRTURO", "designated_date": "2005-01-10", "designation_status": "Designated/Approved", "approved_labeled_indication": "Part of combination therapy in adults (greater than or equal to 18 years) with pulmonary multi-drug resistant tuberculosis (MDR-TB).", "marketing_approval_date": "2012-12-28", "exclusivity_end_date": "2019-12-28", "sponsor": "Janssen Research & Development, LLC|920 Route 202 South|Raritan|New Jersey|8869|United States", "orphan_designation": { "original_text": "Treatment of active tuberculosis", "umls": "C0041296", "parsed_text": "Active Tuberculosis" } }, { "pubchem_cid": 5388906, "pubchem_sid": [ "3726039", "81044374", "103512862", "135262331", "160962911", "162220394", "175427143", "210275153", "210280792", "223471403", "251903327", "312245191", "312695738", "313071201", "318163806", "319303796", "319553105", "332832916", "340580285", "349350823", "355039397", "363907636", "364122776", "374095645", "376033458", "381329027", "381353742", "382165386", "383470764", "384443607", "385641668", "386477932", "404639802", "419501036", "433774167", "433774168", "433774173", "433774174", "434122231", "434142755", "434219419", "438451747", "439480510", "439575872", "440747214", "251971038", "347714031", "374243579", "378132408", "402287578", "405505091", "440051747", "172112135", "254761930", "319343172", "319346298", "319391295", "319438621", "341141918", "375583133", "375664536", "383409863", "251903326", "349995459", "441072047" ], "inchikey": "QUIJNHUBAXPXFS-XLJNKUFUSA-N", "generic_name": "bedaquiline", "trade_name": "SIRTURO", "designated_date": "2005-01-10", "designation_status": "Designated/Approved", "approved_labeled_indication": "SIRTURO is a diarylquinoline antimycobacterial drug indicated as part of combination therapy in the treatment of adult and pediatric patients (12 to less than 18 years of age and weighing at least 30 kg) with pulmonary multi-drug resistant tuberculosis (MDR-TB). Reserve SIRTURO for use when an effective treatment regimen cannot otherwise be provided.", "marketing_approval_date": "2019-08-09", "exclusivity_end_date": "2026-08-09", "exclusivity_protected_indication": "As part of combination therapy in the treatment of pediatric patients (12 to less than 18 years of age and weighing at least 30 kg) with pulmonary multi-drug resistant tuberculosis (MDR-TB)", "sponsor": "Janssen Research & Development, LLC|920 Route 202 South|Raritan|New Jersey|8869|United States", "orphan_designation": { "original_text": "Treatment of active tuberculosis", "umls": "C0041296", "parsed_text": "Active Tuberculosis" } }, { "pubchem_cid": 101526, "pubchem_sid": [ "11528782", "12015073", "46386670", "53788827", "92719042", "103770718", "126621132", "126625643", "126656771", "126670268", "135058355", "135610161", "135684165", "135696603", "144204992", "162049225", "165235308", "170465209", "174527993", "187072315", "196111635", "203355668", "211534856", "223384006", "224399330", "249810600", "249833751", "251916442", "251917789", "252390106", "307080278", "310277321", "312694138", "315664924", "315681560", "318242326", "319134850", "322073862", "322093799", "329863993", "329979169", "340515980", "341137079", "341148480", "341839053", "342475676", "347760719", "347912706", "348858890", "354702894", "355039731", "363662621", "376190937", "376229688", "381388263", "383435823", "384416710", "385660856", "386497361", "387060956", "406627906", "433779894", "434132082", "438482577", "439462311", "440064549", "440818638", "441050242", "441115667", "160963566", "175438118", "313077394", "318161711", "375668612", "440798879", "136375558", "144075736", "252401549", "336297901", "346398775", "376226824", "10254", "252218682", "312654354", "315445592", "340523106", "375757128", "250203678", "268735035", "319435005", "368501109", "376068618", "384570900", "385872486", "441078715" ], "inchikey": "IDIIJJHBXUESQI-DFIJPDEKSA-N", "generic_name": "moxifloxacin hydrochloride", "designated_date": "2014-04-30", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bayer HealthCare Pharmaceuticals, Inc.|100 Bayer Boulevard|P. O. Box 915|Whippany|New Jersey|7981|United States", "orphan_designation": { "original_text": "Treatment of tuberculosis", "umls": "C0041296", "parsed_text": "Tuberculosis" } }, { "pubchem_cid": 2794, "pubchem_sid": [ "9132", "426821", "602744", "855829", "7978968", "10321645", "24714740", "24893114", "46386952", "46508174", "48415799", "49681815", "50004010", "50376246", "56314325", "93167165", "96079551", "103400985", "103914133", "104153270", "124766188", "126671524", "134223284", "134982031", "144075993", "144204067", "160857866", "172080532", "241038834", "249738327", "250130908", "252559282", "253661510", "254804139", "268735210", "275134025", "310270403", "312234670", "312323814", "312694194", "313077393", "315444469", "315661264", "315673496", "318154027", "318160670", "319061878", "319453250", "319556818", "329877105", "332868071", "335959191", "340516670", "340521318", "341140366", "341843591", "346441069", "347757410", "347914019", "348274027", "348283469", "348886799", "349992654", "350087295", "350096016", "354283943", "354326287", "363671221", "363740823", "364120288", "373774285", "374054399", "375293822", "375590147", "377309542", "377498661", "382158132", "384428451", "385784882", "386217795", "386621791", "387168585", "387168586", "387168587", "387195284", "387207892", "404616704", "405469198", "419503223", "419590435", "433779913", "433910201", "434132228", "434145169", "438453439", "440015859", "440064202", "440817885", "441113791", "223434544", "375677347", "438527532", "441071214" ], "inchikey": "WDQPAMHFFCXSNU-UHFFFAOYSA-N", "generic_name": "clofazimine", "designated_date": "2014-06-25", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Novartis Pharmaceuticals Corp.|One Health Plaza|Bldg 135/409|East Hanover|New Jersey|7936|United States", "orphan_designation": { "original_text": "Treatment of active tuberculosis", "umls": "C0041296", "parsed_text": "Active Tuberculosis" } }, { "pubchem_cid": 456199, "pubchem_sid": [ "103516892", "135143585", "175426953", "196409666", "210274726", "210280360", "317223528", "319225761", "341191451", "355039652", "375675290", "376033901", "378138520", "385638884", "386633080", "402420791", "434122232", "434304417", "438486948", "440776380", "405117841" ], "inchikey": "ZLHZLMOSPGACSZ-NSHDSACASA-N", "generic_name": "Pretomanid", "trade_name": "Pretomanid", "designated_date": "2007-07-05", "designation_status": "Designated/Approved", "approved_labeled_indication": "Pretomanid tablet is indicated, as part of a combination regimen with bedaquiline and linezolid for the treatment of adults with pulmonary extensively drug resistant (XDR) or treatment-intolerant or nonresponsive multidrug-resistant (MDR) tuberculosis (TB).", "marketing_approval_date": "2019-08-14", "exclusivity_end_date": "2026-08-14", "exclusivity_protected_indication": "As part of a combination regimen with bedaquiline and linezolid for the treatment of adults with pulmonary extensively drug resistant (XDR) or treatment-intolerant or nonresponsive multidrug-resistant (MDR) tuberculosis (TB).", "sponsor": "Global Alliance for TB Drug Development|40 Wall Street|24th Floor|New York|New York|10005|United States", "orphan_designation": { "original_text": "Treatment of tuberculosis", "umls": "C0041296", "parsed_text": "Tuberculosis" } }, { "pubchem_cid": 5426, "pubchem_sid": [ "10112", "111716", "482479", "596356", "7980770", "8150004", "10321467", "10524356", "11532929", "12013404", "46505665", "48416605", "48422030", "48424173", "49854363", "50104515", "53790759", "56314575", "57288570", "57288853", "78114245", "85089839", "92711690", "103168313", "103913961", "103964042", "104170098", "126630614", "126657153", "126670559", "134971183", "135697990", "144075631", "144203826", "144210897", "160964376", "163379272", "163620804", "163686129", "170465323", "172080723", "174560204", "176262144", "178103899", "196110038", "223739128", "241181800", "250053105", "251891499", "251912024", "251915098", "252122161", "252220266", "252357399", "252402585", "268734961", "274086531", "277309577", "310280840", "312694838", "312811257", "313075769", "313082358", "315673225", "318162415", "318241055", "319063529", "319461271", "319553051", "340516009", "340520354", "340562278", "341139130", "341835493", "346433002", "347913547", "348273553", "348283638", "349083706", "349993647", "350095871", "354284063", "354326253", "363662541", "363894758", "363906098", "374016662", "374190637", "375139760", "375563344", "375577695", "376192192", "376226696", "377309486", "381369564", "382161608", "384567913", "384571599", "385632692", "386266069", "386481966", "387065063", "387065628", "387158826", "387158827", "406855624", "419533589", "434132024", "434315458", "437412648", "438472722", "438493919", "438502714", "440064735", "440790619", "78114247", "93165950", "164194053", "385849682", "386033616", "78114248", "434123881", "78114246", "221673146", "318695808", "438632216", "441071215" ], "inchikey": "UEJJHQNACJXSKW-UHFFFAOYSA-N", "generic_name": "Thalidomide", "designated_date": "1993-01-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Celgene Corporation|P.O. Box 4914|7 Powder Horn Srive|Warren|New Jersey|7059|United States", "orphan_designation": { "original_text": "Treatment of the clinical manifestations of mycobacterial infection caused by Mycobacterium tuberculosis and non-tuberculous mycobacteria.", "umls": [ "C0026918", "C0041296", "C1265234" ], "parsed_text": [ "Mycobacterial infection", "Infection caused by Mycobacterium tuberculosis", "Non-Tuberculous Mycobacteria" ] } }, { "pubchem_cid": 145068, "pubchem_sid": [ "3009", "3815", "584758", "584760", "585304", "612541", "823106", "825225", "827786", "827830", "829083", "829196", "831905", "832739", "832741", "832746", "832749", "835684", "835951", "837432", "838043", "838516", "838735", "838738", "838741", "854516", "854518", "6435917", "6436465", "8009073", "8024763", "8025088", "10322712", "10502255", "11532572", "11532575", "11533089", "11538857", "11538865", "14716264", "14716267", "14716363", "14716369", "14716375", "24423725", "24770575", "26705869", "26707385", "26710838", "26719259", "46499904", "46506897", "48414109", "48421644", "53788122", "56310739", "56311104", "56312083", "56313031", "56313908", "57288490", "57288709", "77506068", "81061389", "81061737", "85256784", "85256791", "89449338", "92192291", "99453648", "99453650", "99453654", "99453656", "103770672", "104410496", "115001634", "121270567", "124349685", "124385040", "135653011", "136959423", "136959448", "144187008", "144220345", "152137752", "152138125", "152198992", "160649658", "160655020", "160657503", "160963781", "163726565", "163831348", "163831356", "177899904", "181085635", "181085637", "210024260", "210024520", "210024533", "249814630", "251972809", "252146112", "252300322", "252300324", "252300555", "252824468", "283537736", "312642117", "312811034", "313369172", "315492707", "315669507", "315669509", "315669512", "315669698", "315671871", "318161803", "318591033", "319163641", "329891624", "329891639", "341814076", "348276049", "348397827", "348397833", "348398212", "348398215", "348398218", "348398221", "348398226", "348498840", "349984329", "350101793", "355190794", "364015747", "364015753", "364015758", "364015763", "364015765", "364015781", "364015785", "364015789", "364015793", "364015797", "374364452", "374364458", "374364463", "374364468", "374881949", "374881952", "381170389", "381338239", "382368031", "382368302", "385157622", "387126461", "404847051", "405529307", "438791282", "439662433", "439662440", "439662449", "439785563", "439785568", "49857525", "134989382", "135660023", "176260048", "223759750", "363902269", "419559537", "584936", "822255", "6435750", "8025524", "46504217", "46504223", "46504228", "99453643", "104233937", "121360306", "144223651", "178100228", "249896950", "250231349", "382369553", "385270240" ], "inchikey": "MWUXSHHQAYIFBG-UHFFFAOYSA-N", "generic_name": "Nitric oxide", "designated_date": "2017-09-26", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Advanced Inhalation Therapies Ltd.|2 Ilan Ramon Street|Ness Ziona|Israel", "orphan_designation": { "original_text": "Treatment of non-tuberculosis mycobacterial (NTM) infections", "umls": [ "C0041296", "C1265234", "C3714514" ], "parsed_text": [ "Tuberculosis", "NTM", "Infections" ] } }, { "pubchem_cid": 145068, "pubchem_sid": [ "3009", "3815", "584758", "584760", "585304", "612541", "823106", "825225", "827786", "827830", "829083", "829196", "831905", "832739", "832741", "832746", "832749", "835684", "835951", "837432", "838043", "838516", "838735", "838738", "838741", "854516", "854518", "6435917", "6436465", "8009073", "8024763", "8025088", "10322712", "10502255", "11532572", "11532575", "11533089", "11538857", "11538865", "14716264", "14716267", "14716363", "14716369", "14716375", "24423725", "24770575", "26705869", "26707385", "26710838", "26719259", "46499904", "46506897", "48414109", "48421644", "53788122", "56310739", "56311104", "56312083", "56313031", "56313908", "57288490", 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Neuwelt, MD|3181 SW Sam Jackson Park Road|Portland|Oregon|97239|United States", "orphan_designation": { "original_text": "For use in MR imaging for the mangement of brain tumors", "umls": "C0006118", "parsed_text": "Brain Tumors" } }, { "pubchem_sid": 104234375, "generic_name": "ferumoxytol", "designated_date": "2011-04-29", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Edward A. Neuwelt, MD|3181 SW Sam Jackson Park Road|Portland|Oregon|97239|United States", "orphan_designation": { "original_text": "For use in MR imaging for the mangement of brain tumors", "umls": "C0006118", "parsed_text": "Brain Tumors" } }, { "pubchem_sid": 386330730, "generic_name": "ferumoxytol", "designated_date": "2011-04-29", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Edward A. 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Neuwelt, MD|3181 SW Sam Jackson Park Road|Portland|Oregon|97239|United States", "orphan_designation": { "original_text": "For use in MR imaging for the mangement of brain tumors", "umls": "C0006118", "parsed_text": "Brain Tumors" } }, { "pubchem_sid": 341169549, "generic_name": "ferumoxytol", "designated_date": "2011-04-29", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Edward A. Neuwelt, MD|3181 SW Sam Jackson Park Road|Portland|Oregon|97239|United States", "orphan_designation": { "original_text": "For use in MR imaging for the mangement of brain tumors", "umls": "C0006118", "parsed_text": "Brain Tumors" } }, { "pubchem_sid": 319392962, "generic_name": "ferumoxytol", "designated_date": "2011-04-29", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Edward A. Neuwelt, MD|3181 SW Sam Jackson Park Road|Portland|Oregon|97239|United States", "orphan_designation": { "original_text": "For use in MR imaging for the mangement of brain tumors", "umls": "C0006118", "parsed_text": "Brain Tumors" } }, { "pubchem_sid": 440790775, "generic_name": "ferumoxytol", "designated_date": "2011-04-29", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Edward A. Neuwelt, MD|3181 SW Sam Jackson Park Road|Portland|Oregon|97239|United States", "orphan_designation": { "original_text": "For use in MR imaging for the mangement of brain tumors", "umls": "C0006118", "parsed_text": "Brain Tumors" } }, { "pubchem_sid": 438456323, "generic_name": "ferumoxytol", "designated_date": "2011-04-29", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Edward A. Neuwelt, MD|3181 SW Sam Jackson Park Road|Portland|Oregon|97239|United States", "orphan_designation": { "original_text": "For use in MR imaging for the mangement of brain tumors", "umls": "C0006118", "parsed_text": "Brain Tumors" } }, { "pubchem_sid": 434252117, "generic_name": "ferumoxytol", "designated_date": "2011-04-29", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Edward A. Neuwelt, MD|3181 SW Sam Jackson Park Road|Portland|Oregon|97239|United States", "orphan_designation": { "original_text": "For use in MR imaging for the mangement of brain tumors", "umls": "C0006118", "parsed_text": "Brain Tumors" } }, { "pubchem_sid": 162064792, "generic_name": "ferumoxytol", "designated_date": "2011-04-29", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Edward A. Neuwelt, MD|3181 SW Sam Jackson Park Road|Portland|Oregon|97239|United States", "orphan_designation": { "original_text": "For use in MR imaging for the mangement of brain tumors", "umls": "C0006118", "parsed_text": "Brain Tumors" } }, { "pubchem_sid": 134979978, "generic_name": "ferumoxytol", "designated_date": "2011-04-29", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Edward A. Neuwelt, MD|3181 SW Sam Jackson Park Road|Portland|Oregon|97239|United States", "orphan_designation": { "original_text": "For use in MR imaging for the mangement of brain tumors", "umls": "C0006118", "parsed_text": "Brain Tumors" } } ] }, { "_id": "MONDO:0019945", "fda_orphan_drug": [ { "pubchem_cid": 16197727, "pubchem_sid": [ "135652322", "175426907", "349993943", "384566871", "385781765", "386618488", "404622388", "85335777", "103214129", "135112553", "319360833", "318163864", "319360678" ], "inchikey": "UAHFGYDRQSXQEB-LEBBXHLNSA-N", "generic_name": "afamelanotide", "designated_date": "2009-12-11", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Clinuvel, Inc.|30 Broad Street|Suites 1407 and 1408|New York|New York|10004|United States", "orphan_designation": { "original_text": "Treatment of solar urticaria", "umls": "C0263610", "parsed_text": "Solar urticaria" } } ] }, { "_id": "MONDO:0008218", "fda_orphan_drug": [ { "pubchem_cid": 16197727, "pubchem_sid": [ "135652322", "175426907", "349993943", "384566871", "385781765", "386618488", "404622388", "85335777", "103214129", "135112553", "319360833", "318163864", "319360678" ], "inchikey": "UAHFGYDRQSXQEB-LEBBXHLNSA-N", "generic_name": "afamelanotide", "designated_date": "2014-05-14", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Clinuvel, Inc.|40 Worth Street|10th Floor - Suite 1053|New York|New York|10013|United States", "orphan_designation": { "original_text": "Treatment of familial benign chronic pemphigus (Hailey-Hailey disease)", "umls": "C0085106", "parsed_text": "Familial Benign Chronic Pemphigus" } } ] }, { "_id": "UMLS:C0162530", "fda_orphan_drug": [ { "pubchem_cid": 16197727, "pubchem_sid": [ "135652322", "175426907", "349993943", "384566871", "385781765", "386618488", "404622388", "85335777", "103214129", "135112553", "319360833", "318163864", "319360678" ], "inchikey": "UAHFGYDRQSXQEB-LEBBXHLNSA-N", "generic_name": "afamelanotide", "trade_name": "SCENESSE", "designated_date": "2008-07-17", "designation_status": "Designated/Approved", "approved_labeled_indication": "SCENESSE is indicated to increase pain free light exposure in adult patients with a history of phototoxic reactions from erythropoietic protoporphyria (EPP)", "marketing_approval_date": "2019-10-08", "exclusivity_end_date": "2026-10-08", "exclusivity_protected_indication": "Indicated to increase pain free light exposure in adult patients with a history of phototoxic reactions from erythropoietic protoporphyria (EPP).", "sponsor": "Clinuvel Inc.|P. O. Box 94026|Menlo Park|California|94026|United States", "orphan_designation": { "original_text": "Treatment of erythropoietic porphyrias", "umls": "C0162530", "parsed_text": "Erythropoietic Porphyrias" } }, { "pubchem_cid": 2749, "pubchem_sid": [ "636498", "11528787", "46506333", "49832259", "53787046", "85643456", "93166238", "103493432", "124766195", "125082390", "126625781", "126656880", "135030204", "160964522", "162174789", "162831460", "170507189", "187072578", "192640214", "196107552", "210279601", "210281924", "223395623", "241049797", "250068259", "252218273", "252358870", "252644416", "254760621", "275323783", "310270251", "312228700", "312631054", "312694423", "313076797", "315683727", "316898222", "316966495", "318019886", "318154387", "318160618", "318166498", "318239775", "319061846", "319451993", "319555207", "322072603", "340521639", "341142562", "341832114", "346436111", "347713969", "348283767", "348397181", "348953529", "349981476", "350088726", "355038632", "363742098", "370626492", "375082692", "375574381", "375668215", "376191553", "376229693", "377309570", "381379884", "382159565", "384567974", "384571692", "385785756", "386622731", "387152333", "404616770", "405477969", "419493250", "434145954", "438452561", "438483038", "438617089", "438645017", "439458838", "440024628", "440816718", "11528724", "364179843", "441075804" ], "inchikey": "SCKYRAXSEDYPSA-UHFFFAOYSA-N", "generic_name": "Ciclopirox", "designated_date": "2018-04-17", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Atlas Molecular Pharma S.L.|Parque Tecnol\udb5e\ude63o de Bizkaia|Building 800|Derio|Spain", "orphan_designation": { "original_text": "Treatment of congenital erythropoietic porphyria", "umls": "C0162530", "parsed_text": "congenital erythropoietic porphyria" } } ] }, { "_id": "MONDO:0019142", "fda_orphan_drug": [ { "pubchem_cid": 16197727, "pubchem_sid": [ "135652322", "175426907", "349993943", "384566871", "385781765", "386618488", "404622388", "85335777", "103214129", "135112553", "319360833", "318163864", "319360678" ], "inchikey": "UAHFGYDRQSXQEB-LEBBXHLNSA-N", "generic_name": "afamelanotide", "designated_date": "2016-02-04", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Clinuvel Inc.|40 Worth Street|Suite 1053|New York|New York|10013|United States", "orphan_designation": { "original_text": "Treatment of cutaneous variants of porphyria (which includes treatment and prevention of cutaneous manifestations of disease)", "umls": "C0032708", "parsed_text": "Porphyria" } } ] }, { "_id": "UMLS:C0085136", "fda_orphan_drug": [ { "pubchem_cid": 10184653, "pubchem_sid": [ "57304398", "103768626", "104144352", "111978343", "134222879", "135267496", "162037384", "162202549", "164041641", "165245589", "170502166", "175427153", "178102295", "184611711", "223397630", "223938206", "249737078", "251971013", "252091143", "252160239", "252214676", "254760942", "275023591", "277300183", "312241967", "312616264", "312808608", "312811069", "316904614", "318047374", "318163560", "318168852", "319342204", "319478626", "319552062", "336909344", "340539706", "341142624", "347726122", "349360159", "349980003", "350079559", "354282190", "355143034", "355149224", "355215187", "374060019", "374185988", "375577118", "375665142", "375678716", "376021202", "376229615", "381317454", "381353309", "382161022", "383409324", "384439413", "384568423", "384572298", "385002860", "385787223", "386624284", "387160284", "402419976", "402420900", "403228970", "405071488", "406517797", "419563020", "433794904", "439646919", "440808165", "441017538", "313062288", "335977306", "335988503", "342460395", "354329207", "375757344", "375768428", "387160286", "387224966", "402287817", "440794243", "440794244", "252214675", "252543372", "405254249", "152258159", "160646996", "221673014", "312695750", "346404253", "354269559", "381317455" ], "inchikey": "ULXXDDBFHOBEHA-CWDCEQMOSA-N", "generic_name": "afatinib", "designated_date": "2014-06-04", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Boehringer Ingelheim Pharmaceuticals, Inc.|900 Ridgebury Road|P. O. Box 368|Ridgefield|Connecticut|6877|United States", "orphan_designation": { "original_text": "Treatment of malignant brain and central nervous system tumors", "umls": "C0085136", "parsed_text": "central nervous system tumors" } } ] }, { "_id": "MONDO:0005233", "fda_orphan_drug": [ { "pubchem_cid": 10184653, "pubchem_sid": [ "57304398", "103768626", "104144352", "111978343", "134222879", "135267496", "162037384", "162202549", "164041641", "165245589", "170502166", "175427153", "178102295", "184611711", "223397630", "223938206", "249737078", "251971013", "252091143", "252160239", "252214676", "254760942", "275023591", "277300183", "312241967", "312616264", "312808608", "312811069", "316904614", "318047374", "318163560", "318168852", "319342204", "319478626", "319552062", "336909344", "340539706", "341142624", "347726122", "349360159", "349980003", "350079559", "354282190", "355143034", "355149224", "355215187", "374060019", "374185988", "375577118", "375665142", "375678716", "376021202", "376229615", "381317454", "381353309", "382161022", "383409324", "384439413", "384568423", "384572298", "385002860", "385787223", "386624284", "387160284", "402419976", "402420900", "403228970", "405071488", "406517797", "419563020", "433794904", "439646919", "440808165", "441017538", "313062288", "335977306", "335988503", "342460395", "354329207", "375757344", "375768428", "387160286", "387224966", "402287817", "440794243", "440794244", "252214675", "252543372", "405254249", "152258159", "160646996", "221673014", "312695750", "346404253", "354269559", "381317455" ], "inchikey": "ULXXDDBFHOBEHA-CWDCEQMOSA-N", "generic_name": "afatinib", "trade_name": "Gilotrif", "designated_date": "2012-12-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "First-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 (L858R) substitution mutations as detected by an FDA-approved test.", "marketing_approval_date": "2013-07-12", "exclusivity_end_date": "2020-07-12", "exclusivity_protected_indication": "First-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 (L858R) substitution mutations as detected by an FDA-approved test.", "sponsor": "Boehringer Ingelheim Pharmaceuticals, Inc.|900 Ridgebury Road|P. O. Box 368|Ridgefield|Connecticut|6877|United States", "orphan_designation": { "original_text": "Treatment of epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC).", "umls": "C0007131", "parsed_text": "Non-Small Cell Lung Cancer" } }, { "pubchem_cid": 10184653, "pubchem_sid": [ "57304398", "103768626", "104144352", "111978343", "134222879", "135267496", "162037384", "162202549", "164041641", "165245589", "170502166", "175427153", "178102295", "184611711", "223397630", "223938206", "249737078", "251971013", "252091143", "252160239", "252214676", "254760942", "275023591", "277300183", "312241967", "312616264", "312808608", "312811069", "316904614", "318047374", "318163560", "318168852", "319342204", "319478626", "319552062", "336909344", "340539706", "341142624", "347726122", "349360159", "349980003", "350079559", "354282190", "355143034", "355149224", "355215187", "374060019", "374185988", "375577118", "375665142", "375678716", "376021202", "376229615", "381317454", "381353309", "382161022", "383409324", "384439413", "384568423", "384572298", "385002860", "385787223", "386624284", "387160284", "402419976", "402420900", "403228970", "405071488", "406517797", "419563020", "433794904", "439646919", "440808165", "441017538", "313062288", "335977306", "335988503", "342460395", "354329207", "375757344", "375768428", "387160286", "387224966", "402287817", "440794243", "440794244", "252214675", "252543372", "405254249", "152258159", "160646996", "221673014", "312695750", "346404253", "354269559", "381317455" ], "inchikey": "ULXXDDBFHOBEHA-CWDCEQMOSA-N", "generic_name": "afatinib", "trade_name": "Gilotrif", "designated_date": "2015-08-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of patients with metastatic, squamous, non-small cell lung cancer progressing after platinum-based chemotherapy", "marketing_approval_date": "2016-04-15", "exclusivity_end_date": "2023-04-15", "exclusivity_protected_indication": "Treatment of patients with metastatic, squamous, non-small cell lung cancer progressing after platinum-based chemotherapy", "sponsor": "Boehringer Ingelheim Pharmaceuticals, Inc.|900 Ridgebury Road|P. O. Box 368|Ridgefield|Connecticut|6877|United States", "orphan_designation": { "original_text": "Treatment of non-small cell lung cancer with squamous histology.", "umls": "C0007131", "parsed_text": "non-small cell lung cancer" } }, { "pubchem_cid": 10184653, "pubchem_sid": [ "57304398", "103768626", "104144352", "111978343", "134222879", "135267496", "162037384", "162202549", "164041641", "165245589", "170502166", "175427153", "178102295", "184611711", "223397630", "223938206", "249737078", "251971013", "252091143", "252160239", "252214676", "254760942", "275023591", "277300183", "312241967", "312616264", "312808608", "312811069", "316904614", "318047374", "318163560", "318168852", "319342204", "319478626", "319552062", "336909344", "340539706", "341142624", "347726122", "349360159", "349980003", "350079559", "354282190", "355143034", "355149224", "355215187", "374060019", "374185988", "375577118", "375665142", "375678716", "376021202", "376229615", "381317454", "381353309", "382161022", "383409324", "384439413", "384568423", "384572298", "385002860", "385787223", "386624284", "387160284", "402419976", "402420900", "403228970", "405071488", "406517797", "419563020", "433794904", "439646919", "440808165", "441017538", "313062288", "335977306", "335988503", "342460395", "354329207", "375757344", "375768428", "387160286", "387224966", "402287817", "440794243", "440794244", "252214675", "252543372", "405254249", "152258159", "160646996", "221673014", "312695750", "346404253", "354269559", "381317455" ], "inchikey": "ULXXDDBFHOBEHA-CWDCEQMOSA-N", "generic_name": "afatinib", "trade_name": "Gilotrif", "designated_date": "2012-12-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "GILOTRIF is indicated for the first-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have non-resistant epidermal growth factor receptor (EGFR) mutations as detected by an FDA-approved test.", "marketing_approval_date": "2018-01-12", "exclusivity_end_date": "2025-01-12", "exclusivity_protected_indication": "GILOTRIF is indicated for the first-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have non-resistant epidermal growth factor receptor (EGFR) mutations other than exon 19 deletions or exon 21 (L858R) substiution mutationas as detected by an FDA-approved test.", "sponsor": "Boehringer Ingelheim Pharmaceuticals, Inc.|900 Ridgebury Road|P. O. Box 368|Ridgefield|Connecticut|6877|United States", "orphan_designation": { "original_text": "Treatment of epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC).", "umls": "C0007131", "parsed_text": "Non-Small Cell Lung Cancer" } }, { "pubchem_cid": 68165256, "pubchem_sid": [ "251846332", "254803312", "255343362", "312322322", "312468161", "319407447", "319556529", "336446476", "340504511", "341102872", "341189488", "346429878", "346550515", "347714033", "347828538", "348357249", "348617011", "348981852", "354329277", "354330023", "363747217", "374094343", "374387522", "375567551", "376032027", "381323730", "381410280", "384569265", "385771605", "386608290", "387020434", "387069518", "387233937", "405040926", "405282122", "405429651", "405539836", "433860904", "436381539", "439976329", "440062228", "440794262", "440796079", "440801895", "441104664", "223366074", "385787143", "386624199", "332853700" ], "inchikey": "AILRADAXUVEEIR-UHFFFAOYSA-N", 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"Non-Small Cell Lung Cancer", "Non-Small Cell Lung Cancer" ] } }, { "pubchem_cid": 117909640, "pubchem_sid": 348351320, "inchikey": "IDRGFNPZDVBSSE-UHFFFAOYSA-N", "generic_name": "N-(3-(2-(2,3-difluoro-4-(4-(2-hydroxyethyl)piperazin-1-yl)phenylamino)quinazolin-8-yl)phenyl)acrylamide", "designated_date": "2017-09-05", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Checkpoint Therapeutics, Inc.|2 Gansevoort Street|9th Floor|New York|New York|10014|United States", "orphan_designation": { "original_text": "Treatment of epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC)", "umls": [ "C0007131", "C1414313", "C3539721" ], "parsed_text": [ "Non-Small Cell Lung Cancer", "EPIDERMAL GROWTH FACTOR RECEPTOR", "Non-Small Cell Lung Cancer" ] } }, { "pubchem_cid": 42611257, "pubchem_sid": [ "104245715", "123055406", "125240992", "131408691", "135267495", "135611115", "136367377", "152258377", 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Francisco|California|94080|United States", "orphan_designation": { "original_text": "Treatment of patients with non-small cell lung cancer (NSCLC) with BRAF V600 mutation", "umls": [ "C0007131", "C3539721" ], "parsed_text": [ "Non-Small Cell Lung Cancer", "Non-Small Cell Lung Cancer" ] } }, { "pubchem_cid": 129073603, "pubchem_sid": [ "375588015", "375589321", "375973221", "381608874", "402382159", "428799677", "433796916", "438433353", "438790727", "440200762", "440792424", "386331594", "438433351" ], "inchikey": "GBLBJPZSROAGMF-SIYOEGHHSA-N", "generic_name": "pralsetinib", "trade_name": "GAVRETO", "designated_date": "2018-04-11", "designation_status": "Designated/Approved", "approved_labeled_indication": "treatment of adult patients with metastatic RET fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test", "marketing_approval_date": "2020-09-04", "exclusivity_end_date": "2027-09-04", "exclusivity_protected_indication": "treatment of adult patients with metastatic RET fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test", "sponsor": "Blueprint Medicines Corporation|45 Sidney Street|Cambridge|Massachusetts|2139|United States", "orphan_designation": { "original_text": "Treatment of rearranged during transfection (RET)-rearranged non-small cell lung cancer (NSCLC), JAK1/2-positive NSCLC, or TRKC-positive NSCLC.", "umls": [ "C0007131", "C0694890", "C3539721" ], "parsed_text": [ "Non-Small Cell Lung Cancer", "rearranged during transfection", "Non-Small Cell Lung Cancer" ] } } ] }, { "_id": "MONDO:0005738", "fda_orphan_drug": [ { "pubchem_cid": 2082, "pubchem_sid": [ "4909", "129996", "603014", "855809", "7978654", "8144956", "8150054", "10321635", "11528637", "12014008", "46506472", "48415523", "49833437", "50282263", "53788484", "56312031", "56313225", "56313722", "57394905", "85083481", "87558956", "92710696", "93166773", "103544522", "103914123", "104098695", "104170113", "126631612", "126658047", 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"C0013502", "C0152069", "C4553297" ], "parsed_text": [ "Hydatid disease", "Alveolar echinococcosis", "Hydatid Disease" ] } } ] }, { "_id": "UMLS:C0152069", "fda_orphan_drug": [ { "pubchem_cid": 2082, "pubchem_sid": [ "4909", "129996", "603014", "855809", "7978654", "8144956", "8150054", "10321635", "11528637", "12014008", "46506472", "48415523", "49833437", "50282263", "53788484", "56312031", "56313225", "56313722", "57394905", "85083481", "87558956", "92710696", "93166773", "103544522", "103914123", "104098695", "104170113", "126631612", "126658047", "126671266", "135003573", "144075667", "144204210", "144212617", "160963863", "162176977", "170464897", "172080738", "174560151", "187072650", "196110442", "210279363", "210281686", "222558024", "223380582", "224266489", "241095709", "250069935", "251884402", "252359352", "252402873", "252453034", "254761236", "268734979", "275187539", "277309463", "310263885", "310265961", "312228199", "312695031", "313076999", "313080042", "315443943", 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bouts of increased seizure activity in selected refractory patients with epilepsy who are on stable regimens of anti-epileptic drugs and who require intermittent use of midazolam", "umls": [ "C0014544", "C1148454" ], "parsed_text": [ "Epilepsy", "seizure activity" ] } } ] }, { "_id": "MONDO:0015484", "fda_orphan_drug": [ { "pubchem_cid": 2082, "pubchem_sid": [ "4909", "129996", "603014", "855809", "7978654", "8144956", "8150054", "10321635", "11528637", "12014008", "46506472", "48415523", "49833437", "50282263", "53788484", "56312031", "56313225", "56313722", "57394905", "85083481", "87558956", "92710696", "93166773", "103544522", "103914123", "104098695", "104170113", "126631612", "126658047", "126671266", "135003573", "144075667", "144204210", "144212617", "160963863", "162176977", "170464897", "172080738", "174560151", "187072650", "196110442", "210279363", "210281686", "222558024", "223380582", "224266489", "241095709", "250069935", "251884402", "252359352", "252402873", 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associated with spinal cord injury", "umls": [ "C0027796", "C0037929" ], "parsed_text": [ "Neuropathic Pain", "Spinal Cord Injury" ] } }, { "pubchem_cid": 114869, "pubchem_sid": [ "11528627", "135073042", "162181264", "223399971", "251886934", "312695347", "313082406", "318153697", "319143352", "319461282", "347913733", "348397577", "349207584", "374059649", "375577734", "377309851", "381361539", "382163533", "434313639", "438491735", "160964042", "318162476" ], "inchikey": "ZWUKMNZJRDGCTQ-UHFFFAOYSA-N", "generic_name": "Tizanidine HCl", "designated_date": "1994-01-31", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Athena Neurosciences, Inc.|800 Gateway Boulevard|South San Francisco|California|94080|United States", "orphan_designation": { "original_text": "Treatment of spasticity associated with multiple sclerosis and spinal cord injury.", "umls": [ "C0026769", "C0026838", "C0037929" ], "parsed_text": [ "Multiple Sclerosis", "Spasticity", "Spinal Cord Injury" ] } }, { "pubchem_cid": 9844677, "pubchem_sid": 381609386, "inchikey": "DSBPAFWXMCKFIM-BSOLPCOYSA-N", "generic_name": "L-threonyl-L-prolyl-L-prolyl-L-threonine", "designated_date": "2005-04-26", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Nyxis Neurotherapies, Inc.|1801 Maple Avenue|Suite 4300|Evanston|Illinois|60201|United States", "orphan_designation": { "original_text": "Treatment of neuropathic pain associated with spinal cord injury", "umls": [ "C0027796", "C0037929" ], "parsed_text": [ "Neuropathic Pain", "Spinal Cord Injury" ] } }, { "pubchem_cid": 44602, "pubchem_sid": [ "135107526", "162255235", "319092574", "347828402", "123055284", "162255234", "348398423" ], "inchikey": "KPYSYYIEGFHWSV-QMMMGPOBSA-N", "generic_name": "L-baclofen", "designated_date": "1991-12-17", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Osmotica Pharmaceutical Corp.|1205 Culbreth Dr.|Suite 200|Wilmington|North Carolina|28405|United States", "orphan_designation": { "original_text": "Treatment of spasticity associated with spinal cord injury or multiple sclerosis.", "umls": [ "C0026769", "C0026838", "C0037929" ], "parsed_text": [ "Multiple Sclerosis", "Spasticity", "Spinal Cord Injury" ] } }, { "pubchem_cid": 44600, "pubchem_sid": [ "135107526", "162255235", "319092574", "347828402", "123055284", "162255234", "348398423" ], "inchikey": "KPYSYYIEGFHWSV-MRVPVSSYSA-N", "generic_name": "L-baclofen", "designated_date": "1991-12-17", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Osmotica Pharmaceutical Corp.|1205 Culbreth Dr.|Suite 200|Wilmington|North Carolina|28405|United States", "orphan_designation": { "original_text": "Treatment of spasticity associated with spinal cord injury or multiple sclerosis.", "umls": [ "C0026769", "C0026838", "C0037929" ], "parsed_text": [ "Multiple Sclerosis", "Spasticity", "Spinal Cord Injury" ] } } ] }, { "_id": "UMLS:C0522224", "fda_orphan_drug": [ { "pubchem_cid": 2083, "pubchem_sid": [ "597388", "7978655", "8149629", "24398148", "46505312", "48415524", "49857667", "53789009", "56413057", "92303250", "103195118", "126561471", "134991777", "135650977", "144075315", "144203817", "144207998", "160964337", "170465034", "223815455", "249897300", "252344808", "254757419", "310279840", "312694134", "313077408", "315672164", "318160139", "319061434", "322079874", "348275478", "349988839", "355037743", "375574610", "381388426", "384645270", "386266408", "387127577", "387127578", "404754531", "433779893", "434132284", "26719665", "49681717", "162037972", "310279841", "317223717", "347913191", "375578405", "375583312", "387061031", "136965309", "387127576", "347229524", "375652888" ], "inchikey": "NDAUXUAQIAJITI-UHFFFAOYSA-N", "generic_name": "Albuterol", "designated_date": "2002-03-12", 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"329877956", "340502925", "341188641", "347828371", "349974425", "355040526", "374075784", "375577260", "375581460", "375644255", "381334866", "381356862", "384568581", "384572508", "385640521", "386634775", "419592133", "433843394", "434315502", "436380033", "438493959", "440062971", "440681358", "440681359", "440703029", "440796953", "440798892", "441040073", "252216590", "254759136", "405501518", "440048174", "221672824", "223471422" ], "inchikey": "SXNJFOWDRLKDSF-XKHVUIRMSA-N", "generic_name": "Volasertib", "designated_date": "2020-10-08", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Oncoheroes Biosciences Inc.|43 Kent Street, #1|Brookline|Massachusetts|2445|United States", "orphan_designation": { "original_text": "treatment of soft tissue sarcoma", "umls": "C1261473", "parsed_text": "Soft tissue sarcoma" } }, { "pubchem_cid": 137704508, "pubchem_sid": [ "49684238", "135252141", "160681716", "162037389", "223366213", "254761315", "317223400", "318163824", "341173007", "375577124", "384571090", "384572809", "385645404", "386481759", "404770154", "440796027", "251916147", "251917495", "251971430", "319346450", "349990690", "355040169", "423212991", "163123673", "312654959", "319411487", "336349003", "342476450", "162224767", "428401475", "434315580", "438494097", "204360730", "223447400", "273912055", "319379127", "319382809", "319397924", "319407907", "319408885", "319413523", "319413976", "319416183", "319417053", "319423608", "319430515", "363741354", "364854391", "375581407", "375675360", "428558798" ], "inchikey": "BUROJSBIWGDYCN-MWBWAFHZSA-N", "generic_name": "ridaforolimus", "designated_date": "2005-08-12", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Merck Sharp & Dohme Corp.|Merck & Co., Inc. Subsidiary|P. Box 1000, UG-2C50|North Wales|Oregon|19454|United States", "orphan_designation": { "original_text": "Treatment of soft tissue sarcoma", "umls": "C1261473", "parsed_text": "Soft tissue sarcoma" } }, { "pubchem_cid": 137303705, "pubchem_sid": [ "49684238", "135252141", "160681716", "162037389", "223366213", "254761315", "317223400", "318163824", "341173007", "375577124", "384571090", "384572809", "385645404", "386481759", "404770154", "440796027", "251916147", "251917495", "251971430", "319346450", "349990690", "355040169", "423212991", "163123673", "312654959", "319411487", "336349003", "342476450", "162224767", "428401475", "434315580", "438494097", "204360730", "223447400", "273912055", "319379127", "319382809", "319397924", "319407907", "319408885", "319413523", "319413976", "319416183", "319417053", "319423608", "319430515", "363741354", "364854391", "375581407", "375675360", "428558798" ], "inchikey": "BUROJSBIWGDYCN-BPIPWEEESA-N", "generic_name": "ridaforolimus", "designated_date": "2005-08-12", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Merck Sharp & Dohme Corp.|Merck & Co., Inc. Subsidiary|P. Box 1000, UG-2C50|North Wales|Oregon|19454|United States", "orphan_designation": { "original_text": "Treatment of soft tissue sarcoma", "umls": "C1261473", "parsed_text": "Soft tissue sarcoma" } }, { "pubchem_cid": 133268486, "pubchem_sid": [ "49684238", "135252141", "160681716", "162037389", "223366213", "254761315", "317223400", "318163824", "341173007", "375577124", "384571090", "384572809", "385645404", "386481759", "404770154", "440796027", "251916147", "251917495", "251971430", "319346450", "349990690", "355040169", "423212991", "163123673", "312654959", "319411487", "336349003", "342476450", "162224767", "428401475", "434315580", "438494097", "204360730", "223447400", "273912055", "319379127", "319382809", "319397924", "319407907", "319408885", "319413523", "319413976", "319416183", "319417053", "319423608", "319430515", "363741354", "364854391", "375581407", "375675360", "428558798" ], "inchikey": "BUROJSBIWGDYCN-GAFNQWMWSA-N", "generic_name": "ridaforolimus", "designated_date": "2005-08-12", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Merck Sharp & Dohme Corp.|Merck & Co., Inc. Subsidiary|P. Box 1000, UG-2C50|North Wales|Oregon|19454|United States", "orphan_designation": { "original_text": "Treatment of soft tissue sarcoma", "umls": "C1261473", "parsed_text": "Soft tissue sarcoma" } }, { "pubchem_cid": 87423409, "pubchem_sid": [ "49684238", "135252141", "160681716", "162037389", "223366213", "254761315", "317223400", "318163824", "341173007", "375577124", "384571090", "384572809", "385645404", "386481759", "404770154", "440796027", "251916147", "251917495", "251971430", "319346450", "349990690", "355040169", "423212991", "163123673", "312654959", "319411487", "336349003", "342476450", "162224767", "428401475", "434315580", "438494097", "204360730", "223447400", "273912055", "319379127", "319382809", "319397924", "319407907", "319408885", "319413523", "319413976", "319416183", "319417053", "319423608", "319430515", "363741354", "364854391", "375581407", "375675360", "428558798" ], "inchikey": "BUROJSBIWGDYCN-AFTOUBNRSA-N", "generic_name": "ridaforolimus", "designated_date": "2005-08-12", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Merck Sharp & Dohme Corp.|Merck & Co., Inc. Subsidiary|P. Box 1000, UG-2C50|North Wales|Oregon|19454|United States", "orphan_designation": { "original_text": "Treatment of soft tissue sarcoma", "umls": "C1261473", "parsed_text": "Soft tissue sarcoma" } }, { "pubchem_cid": 86289926, "pubchem_sid": [ "49684238", "135252141", "160681716", "162037389", "223366213", "254761315", "317223400", "318163824", "341173007", "375577124", "384571090", "384572809", "385645404", "386481759", "404770154", "440796027", "251916147", "251917495", "251971430", "319346450", "349990690", "355040169", "423212991", "163123673", "312654959", "319411487", "336349003", "342476450", "162224767", "428401475", "434315580", "438494097", "204360730", "223447400", "273912055", "319379127", "319382809", "319397924", "319407907", "319408885", "319413523", "319413976", "319416183", "319417053", "319423608", "319430515", "363741354", "364854391", "375581407", "375675360", "428558798" ], "inchikey": "BUROJSBIWGDYCN-QSRQGROMSA-N", "generic_name": "ridaforolimus", "designated_date": "2005-08-12", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Merck Sharp & Dohme Corp.|Merck & Co., Inc. Subsidiary|P. Box 1000, UG-2C50|North Wales|Oregon|19454|United States", "orphan_designation": { "original_text": "Treatment of soft tissue sarcoma", "umls": "C1261473", "parsed_text": "Soft tissue sarcoma" } }, { "pubchem_cid": 74787553, "pubchem_sid": [ "49684238", "135252141", "160681716", "162037389", "223366213", "254761315", "317223400", "318163824", "341173007", "375577124", "384571090", "384572809", "385645404", "386481759", "404770154", "440796027", "251916147", "251917495", "251971430", "319346450", "349990690", "355040169", "423212991", "163123673", "312654959", "319411487", "336349003", "342476450", "162224767", "428401475", "434315580", "438494097", "204360730", "223447400", "273912055", "319379127", "319382809", "319397924", "319407907", "319408885", "319413523", "319413976", "319416183", "319417053", "319423608", "319430515", "363741354", "364854391", "375581407", "375675360", "428558798" ], "inchikey": "BUROJSBIWGDYCN-OZFXDLPUSA-N", "generic_name": "ridaforolimus", "designated_date": "2005-08-12", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Merck Sharp & Dohme Corp.|Merck & Co., Inc. Subsidiary|P. Box 1000, UG-2C50|North Wales|Oregon|19454|United States", "orphan_designation": { "original_text": "Treatment of soft tissue sarcoma", "umls": "C1261473", "parsed_text": "Soft tissue sarcoma" } }, { "pubchem_cid": 71308311, "pubchem_sid": [ "49684238", "135252141", "160681716", "162037389", "223366213", "254761315", "317223400", "318163824", "341173007", "375577124", "384571090", "384572809", "385645404", "386481759", "404770154", "440796027", "251916147", "251917495", "251971430", "319346450", "349990690", "355040169", "423212991", "163123673", "312654959", "319411487", "336349003", "342476450", "162224767", "428401475", "434315580", "438494097", "204360730", "223447400", "273912055", "319379127", "319382809", "319397924", "319407907", "319408885", "319413523", "319413976", "319416183", "319417053", "319423608", "319430515", "363741354", "364854391", "375581407", "375675360", "428558798" ], "inchikey": "BUROJSBIWGDYCN-QVXCGEACSA-N", "generic_name": "ridaforolimus", "designated_date": "2005-08-12", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Merck Sharp & Dohme Corp.|Merck & Co., Inc. Subsidiary|P. Box 1000, UG-2C50|North Wales|Oregon|19454|United States", "orphan_designation": { "original_text": "Treatment of soft tissue sarcoma", "umls": "C1261473", "parsed_text": "Soft tissue sarcoma" } }, { "pubchem_cid": 71433797, "pubchem_sid": [ "49684238", "135252141", "160681716", "162037389", "223366213", "254761315", "317223400", "318163824", "341173007", "375577124", "384571090", "384572809", "385645404", "386481759", "404770154", "440796027", "251916147", "251917495", "251971430", "319346450", "349990690", "355040169", "423212991", "163123673", "312654959", "319411487", "336349003", "342476450", "162224767", "428401475", "434315580", "438494097", "204360730", "223447400", "273912055", "319379127", "319382809", "319397924", "319407907", "319408885", "319413523", "319413976", "319416183", "319417053", "319423608", "319430515", "363741354", "364854391", "375581407", "375675360", "428558798" ], "inchikey": "BUROJSBIWGDYCN-UHFFFAOYSA-N", "generic_name": "ridaforolimus", "designated_date": "2005-08-12", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Merck Sharp & Dohme Corp.|Merck & Co., Inc. Subsidiary|P. Box 1000, UG-2C50|North Wales|Oregon|19454|United States", "orphan_designation": { "original_text": "Treatment of soft tissue sarcoma", "umls": "C1261473", "parsed_text": "Soft tissue sarcoma" } }, { "pubchem_cid": 25199957, "pubchem_sid": [ "49684238", "135252141", "160681716", "162037389", "223366213", "254761315", "317223400", "318163824", "341173007", "375577124", "384571090", "384572809", "385645404", "386481759", "404770154", "440796027", "251916147", "251917495", "251971430", "319346450", "349990690", "355040169", "423212991", "163123673", "312654959", "319411487", "336349003", "342476450", "162224767", "428401475", "434315580", "438494097", "204360730", "223447400", "273912055", "319379127", "319382809", "319397924", "319407907", "319408885", "319413523", "319413976", "319416183", "319417053", "319423608", "319430515", "363741354", "364854391", "375581407", "375675360", "428558798" ], "inchikey": "BUROJSBIWGDYCN-QHPXJTPRSA-N", "generic_name": "ridaforolimus", "designated_date": "2005-08-12", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Merck Sharp & Dohme Corp.|Merck & Co., Inc. Subsidiary|P. Box 1000, UG-2C50|North Wales|Oregon|19454|United States", "orphan_designation": { "original_text": "Treatment of soft tissue sarcoma", "umls": "C1261473", "parsed_text": "Soft tissue sarcoma" } }, { "pubchem_cid": 11520894, "pubchem_sid": [ "49684238", "135252141", "160681716", "162037389", "223366213", "254761315", "317223400", "318163824", "341173007", "375577124", "384571090", "384572809", "385645404", "386481759", "404770154", "440796027", "251916147", "251917495", "251971430", "319346450", "349990690", "355040169", "423212991", "163123673", "312654959", "319411487", "336349003", "342476450", "162224767", "428401475", "434315580", "438494097", "204360730", "223447400", "273912055", "319379127", "319382809", "319397924", "319407907", "319408885", "319413523", "319413976", "319416183", "319417053", "319423608", "319430515", "363741354", "364854391", "375581407", "375675360", "428558798" ], "inchikey": "BUROJSBIWGDYCN-GAUTUEMISA-N", "generic_name": "ridaforolimus", "designated_date": "2005-08-12", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Merck Sharp & Dohme Corp.|Merck & Co., Inc. Subsidiary|P. Box 1000, UG-2C50|North Wales|Oregon|19454|United States", "orphan_designation": { "original_text": "Treatment of soft tissue sarcoma", "umls": "C1261473", "parsed_text": "Soft tissue sarcoma" } }, { "pubchem_cid": 66558664, "pubchem_sid": [ "178103590", "210274978", "210280616", "249820378", "252160839", "312387361", "312616229", "319407144", "341200180", "347829042", "355039502", "375578110", "378138548", "384569100", "385653587", "386490045", "387207321", "433835407", "434311605", "438490560", "440702114", "440809566", "332853780", "332853781" ], "inchikey": "NSQSAUGJQHDYNO-UHFFFAOYSA-N", "generic_name": "tazemetostat", "designated_date": "2017-06-15", "designation_status": "Designated/Approved", "approved_labeled_indication": "TAZVERIK is indicated for the treatment of adults and pediatric patients aged 16 years and older with metastatic or locally advanced epithelioid sarcoma not eligible for complete resection.", "marketing_approval_date": "2020-01-23", "exclusivity_end_date": "2027-01-23", "sponsor": "Epizyme, Inc.|400 Technology Square|4th Floor|Cambridge|Massachusetts|2139|United States", "orphan_designation": { "original_text": "Treatment of soft tissue sarcoma", "umls": "C1261473", "parsed_text": "Soft tissue sarcoma" } }, { "pubchem_sid": 387061104, "generic_name": "Atezolizumab", "designated_date": "2020-10-08", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genentech, Inc.|1 DNA Way|South San Francisco|California|94080-4990|United States", "orphan_designation": { "original_text": "treatment of soft tissue sarcoma", "umls": "C1261473", "parsed_text": "Soft tissue sarcoma" } }, { "pubchem_sid": 385327550, "generic_name": "Atezolizumab", "designated_date": "2020-10-08", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genentech, Inc.|1 DNA Way|South San Francisco|California|94080-4990|United States", "orphan_designation": { "original_text": "treatment of soft tissue sarcoma", "umls": "C1261473", "parsed_text": "Soft tissue sarcoma" } }, { "pubchem_sid": 384647093, "generic_name": "Atezolizumab", "designated_date": "2020-10-08", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genentech, Inc.|1 DNA Way|South San Francisco|California|94080-4990|United States", "orphan_designation": { "original_text": "treatment of soft tissue sarcoma", "umls": "C1261473", "parsed_text": "Soft tissue sarcoma" } }, { "pubchem_sid": 377483935, "generic_name": "Atezolizumab", "designated_date": "2020-10-08", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genentech, Inc.|1 DNA Way|South San Francisco|California|94080-4990|United States", "orphan_designation": { "original_text": "treatment of soft tissue sarcoma", "umls": "C1261473", "parsed_text": "Soft tissue sarcoma" } }, { "pubchem_sid": 354355059, "generic_name": "Atezolizumab", "designated_date": "2020-10-08", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genentech, Inc.|1 DNA Way|South San Francisco|California|94080-4990|United States", "orphan_designation": { "original_text": "treatment of soft tissue sarcoma", "umls": "C1261473", "parsed_text": "Soft tissue sarcoma" } }, { "pubchem_sid": 347911207, "generic_name": "Atezolizumab", "designated_date": "2020-10-08", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genentech, Inc.|1 DNA Way|South San Francisco|California|94080-4990|United States", "orphan_designation": { "original_text": "treatment of soft tissue sarcoma", "umls": "C1261473", "parsed_text": "Soft tissue sarcoma" } }, { "pubchem_sid": 318464470, "generic_name": "Atezolizumab", "designated_date": "2020-10-08", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genentech, Inc.|1 DNA Way|South San Francisco|California|94080-4990|United States", "orphan_designation": { "original_text": "treatment of soft tissue sarcoma", "umls": "C1261473", "parsed_text": "Soft tissue sarcoma" } }, { "pubchem_sid": 249565671, "generic_name": "Atezolizumab", "designated_date": "2020-10-08", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genentech, Inc.|1 DNA Way|South San Francisco|California|94080-4990|United States", "orphan_designation": { "original_text": "treatment of soft tissue sarcoma", "umls": "C1261473", "parsed_text": "Soft tissue sarcoma" } }, { "pubchem_sid": 354702230, "generic_name": "Fibromun", "designated_date": "2018-12-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Philogen S.p.A.|La Lizza 7|I-53100 Siena|Siena|Tuscany|Italy", "orphan_designation": { "original_text": "Treatment of Soft Tissue 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Box 2072.|Toowong Business Center|Australia", "orphan_designation": { "original_text": "Treatment of AIDS-related Kaposi's sarcoma.", "umls": "C0276535", "parsed_text": "AIDS-related Kaposi's sarcoma" } }, { "pubchem_cid": 36314, "pubchem_sid": [ "418145", "598380", "841111", "7980243", "12013494", "26683888", "26697100", "46506910", "48416372", "53787331", "56310806", "56310828", "56311576", "56311706", "56311708", "56311902", "56312138", "56312386", "56312626", "56312945", "56313331", "56313985", "56314179", "56314556", "56314557", "56463361", "57394139", "81044596", "93576959", "103916211", "124974065", "134998291", "135652721", "144205660", "162231831", "163614581", "163621148", "163686494", "165235324", "174528934", "176262473", "184812010", "201248881", "223376506", "223716407", "223743720", "241062053", "249839812", "250230395", "251914968", "252401436", "254760734", "255428601", "273570728", "274952737", "275872251", "310278465", "312228733", "313081798", "315673983", "316542516", "317227624", "318126557", "318161892", "319086976", "319450121", "319552671", "322069780", "329991932", "340126133", "340521018", "341137759", "341139702", "341834703", "342407450", "347759496", "348283806", "348978875", "354283815", "355039710", "355229248", "363666986", "363906093", "375577660", "375660040", "376054474", "381117554", "384255303", "385633321", "386623126", "387147318", "404630041", "406674026", "433986904", "434132041", "434315358", "438473069", "438493718", "438498235", "438625807", "440064692", "440771264", "440790582", "9598", "8150059", "74382495", "87558555", "92303414", "92710505", "126631813", "126658135", "144075668", "162170581", "196105609", "223396182", "241182126", "252215987", "268734407", "315445848", "318238302", "348273301", "354265544", "354742347", "363602488", "376227271", "384568039", "384571788", "386989192", "387147320", "85088433", "103911116", "126670773", "162092369", "184021953", "211535948", "223251764", "249971604", "312630528", "313077255", "319062999", "342586964", "363604598", "376154637", "382159990", "404755030", "433780076", "277299310", "340471410", "375784664", "10321464", "51030891", "85154876", "252451077", "319222110", "319422955", "341825594", "404760853", "855687", "26663012", "47193750", "49881022", "56422161", "56463253", "103913958", "221672922", "250134802", "250213893", "252302082", "252448596", "312616203", "312808208", "319379844", "319434061", "340550202", "377310845", "419551214", "441075217" ], "inchikey": "RCINICONZNJXQF-MZXODVADSA-N", "generic_name": "Paclitaxel", "trade_name": "Taxol", "designated_date": "1997-03-25", "designation_status": "Designated/Approved", "approved_labeled_indication": "For the second line treatment of AIDS-related Kaposi's sarcoma.", "marketing_approval_date": "1997-08-04", "exclusivity_end_date": "2004-08-04", "sponsor": "Bristol-Myers Squibb Pharmaceutical Research Institute|5 Research Parkway|P.O. Box 5100|Wallingford|Connecticut|6492|United States", "orphan_designation": { "original_text": "Treatment of AIDS-related Kaposi's sarcoma.", "umls": "C0276535", "parsed_text": "AIDS-related Kaposi's sarcoma" } }, { "pubchem_cid": 36314, "pubchem_sid": [ "418145", "598380", "841111", "7980243", "12013494", "26683888", "26697100", "46506910", "48416372", "53787331", "56310806", "56310828", "56311576", "56311706", "56311708", "56311902", "56312138", "56312386", "56312626", "56312945", "56313331", "56313985", "56314179", "56314556", "56314557", "56463361", "57394139", "81044596", "93576959", "103916211", "124974065", "134998291", "135652721", "144205660", "162231831", "163614581", "163621148", "163686494", "165235324", "174528934", "176262473", "184812010", "201248881", "223376506", "223716407", "223743720", "241062053", "249839812", "250230395", "251914968", "252401436", "254760734", "255428601", "273570728", "274952737", "275872251", "310278465", "312228733", "313081798", "315673983", "316542516", "317227624", "318126557", "318161892", "319086976", "319450121", "319552671", "322069780", "329991932", "340126133", "340521018", "341137759", "341139702", "341834703", "342407450", "347759496", "348283806", "348978875", "354283815", "355039710", "355229248", "363666986", "363906093", "375577660", "375660040", "376054474", "381117554", "384255303", "385633321", "386623126", "387147318", "404630041", "406674026", "433986904", "434132041", "434315358", "438473069", "438493718", "438498235", "438625807", "440064692", "440771264", "440790582", "9598", "8150059", "74382495", "87558555", "92303414", "92710505", "126631813", "126658135", "144075668", "162170581", "196105609", "223396182", "241182126", "252215987", "268734407", "315445848", "318238302", "348273301", "354265544", "354742347", "363602488", "376227271", "384568039", "384571788", "386989192", "387147320", "85088433", "103911116", "126670773", "162092369", "184021953", "211535948", "223251764", "249971604", "312630528", "313077255", "319062999", "342586964", "363604598", "376154637", "382159990", "404755030", "433780076", "277299310", "340471410", "375784664", "10321464", "51030891", "85154876", "252451077", "319222110", "319422955", "341825594", "404760853", "855687", "26663012", "47193750", "49881022", "56422161", "56463253", "103913958", "221672922", "250134802", "250213893", "252302082", "252448596", "312616203", "312808208", "319379844", "319434061", "340550202", "377310845", "419551214", "441075217" ], "inchikey": "RCINICONZNJXQF-MZXODVADSA-N", "generic_name": "Paclitaxel", "designated_date": "1997-04-15", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Baker Norton Pharmaceuticals, Inc.|4400 Biscayne Boulevard|Miami|Florida|33137|United States", "orphan_designation": { "original_text": "Treatment of AIDS-related Kaposi's sarcoma.", "umls": "C0276535", "parsed_text": "AIDS-related Kaposi's sarcoma" } }, { "pubchem_sid": 46506712, "generic_name": "Interferon alfa-2a (recombinant)", "trade_name": "Roferon-A", "designated_date": "1987-12-14", "designation_status": "Designated/Approved", "marketing_approval_date": "1988-11-21", "exclusivity_end_date": "1995-11-21", "sponsor": "Hoffmann-La Roche, Inc.|340 Kingsland Street|Nutley|New Jersey|7110|United States", "orphan_designation": { "original_text": "Treatment of AIDS related Kaposi's sarcoma.", "umls": "C0276535", "parsed_text": "AIDS Related Kaposi's Sarcoma" } } ] }, { "_id": "UMLS:C0036220", "fda_orphan_drug": [ { "pubchem_cid": 449171, "pubchem_sid": [ "11528315", "12014910", "24702085", "46507356", "53790163", "85240518", "103194296", "126645184", "127324022", "127324023", "127324024", "127324025", "127324026", "134985140", "135651418", "136342471", "160963868", "162172151", "310270343", "312217688", "313391998", "316538952", "318163351", "348275782", "349986946", "355141289", "376229699", "385784250", "386621124", "404615685", "434139548", "438446289", "440776372", "85165342", "349976458", "387153240" ], "inchikey": "SHGAZHPCJJPHSC-ZVCIMWCZSA-N", "generic_name": "alitretinoin", "designated_date": "2021-01-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Galephar Pharmaceutical Research, Inc.|Carr 925 Km 6.1 Bo. Junquito|HC-04 BOX 4540|Humacao|Puerto Rico|791|United States", "orphan_designation": { "original_text": "Treatment of systemic (non-cutaneous) kaposi sarcoma", "umls": "C0036220", "parsed_text": "Kaposi Sarcoma" } }, { "pubchem_cid": 134780, "pubchem_sid": [ "50086901", "53787052", "103221733", "135089995", "135727444", "136367549", "136946644", "152258286", "160647125", "162011791", "162012270", "162037668", "162189531", "163398415", "173568531", "175427148", "178103920", "186007045", "203856046", "223798539", "251971242", "252220187", "253660183", "254760265", "254804819", "272830543", "277293312", "310279125", "312245298", "312602521", "312616273", "316906215", "318163809", "318243312", "319155184", "319552023", "329861099", "332867260", "336909162", "340462707", "340563288", "341136372", "341137222", "341143396", "341846760", "347756802", "347912771", "348273307", "349984283", "350079484", "350083086", "354270896", "355040614", "363662638", "370477613", "374041922", "375351555", "375576804", "375583177", "375790536", "376033422", "376137493", "377019780", "377309970", "378132148", "381141282", "384439419", "384568836", "384572873", "385271827", "385639805", "386270690", "386634021", "387162144", "402287803", "402409199", "404633877", "405056931", "405072516", "419498890", "433841372", "434315461", "438475747", "438493917", "438645304", "438651074", "440800631", "440803403", "441030352", "441104011", "221672910", "318695084", "405544231", "439576423", "441071219" ], "inchikey": "UVSMNLNDYGZFPF-UHFFFAOYSA-N", "generic_name": "pomalidomide", "trade_name": "POMALYST", "designated_date": "2018-04-04", "designation_status": "Designated/Approved", "approved_labeled_indication": "POMALYST (pomalidomide) is indicated for the treatment of adult patients with AIDS-related Kaposi sarcoma (KS) after failure of highly active antiretroviral therapy (HAART); and for the treatment of Kaposi sarcoma (KS) in adult patients who are HIV-negative.", "marketing_approval_date": "2020-05-14", "exclusivity_end_date": "2027-05-14", "exclusivity_protected_indication": "Indicated for the treatment of adult patients with AIDS-related Kaposi sarcoma (KS) after failure of highly active antiretroviral therapy (HAART); and for the treatment of Kaposi sarcoma (KS) in adult patients who are HIV-negative.", "sponsor": "Celgene Corporation|86 Morris Avenue|Summit|New Jersey|7901|United States", "orphan_designation": { "original_text": "Treatment of Kaposi sarcoma", "umls": "C0036220", "parsed_text": "Kaposi Sarcoma" } }, { "pubchem_cid": 9872764, "pubchem_sid": [ "328083259", "348622828" ], "inchikey": "UEAYTGFTNACKMX-VMXHOPILSA-N", "generic_name": "H-Leu-Pro-Pro-Ser-Arg-OH", "designated_date": "2012-09-20", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Immuno Tech, Inc.|6329 256th Street, NE|Arlington|Washington|98223|United States", "orphan_designation": { "original_text": "Treatment of Kaposi sarcoma", "umls": "C0036220", "parsed_text": "Kaposi Sarcoma" } }, { "pubchem_cid": 5426, "pubchem_sid": [ "10112", "111716", "482479", "596356", "7980770", "8150004", "10321467", "10524356", "11532929", "12013404", "46505665", "48416605", "48422030", "48424173", "49854363", "50104515", "53790759", "56314575", "57288570", "57288853", "78114245", "85089839", "92711690", "103168313", "103913961", "103964042", "104170098", "126630614", "126657153", "126670559", "134971183", "135697990", "144075631", "144203826", "144210897", "160964376", "163379272", "163620804", "163686129", "170465323", "172080723", "174560204", "176262144", "178103899", "196110038", "223739128", "241181800", "250053105", "251891499", "251912024", "251915098", "252122161", "252220266", "252357399", "252402585", "268734961", "274086531", "277309577", "310280840", "312694838", "312811257", "313075769", "313082358", "315673225", "318162415", "318241055", "319063529", "319461271", "319553051", "340516009", "340520354", "340562278", "341139130", "341835493", "346433002", "347913547", "348273553", "348283638", "349083706", "349993647", "350095871", "354284063", "354326253", "363662541", "363894758", "363906098", "374016662", "374190637", "375139760", "375563344", "375577695", "376192192", "376226696", "377309486", "381369564", "382161608", "384567913", "384571599", "385632692", "386266069", "386481966", "387065063", "387065628", "387158826", "387158827", "406855624", "419533589", "434132024", "434315458", "437412648", "438472722", "438493919", "438502714", "440064735", "440790619", "78114247", "93165950", "164194053", "385849682", "386033616", "78114248", "434123881", "78114246", "221673146", "318695808", "438632216", "441071215" ], "inchikey": "UEJJHQNACJXSKW-UHFFFAOYSA-N", "generic_name": "Thalidomide", "designated_date": "1998-07-29", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Celgene Corporation|P.O. Box 4914|7 Powder Horn Srive|Warren|New Jersey|7059|United States", "orphan_designation": { "original_text": "Treatment of Kaposi's sarcoma.", "umls": "C0036220", "parsed_text": "Kaposi's Sarcoma" } } ] }, { "_id": "MONDO:0012883", "fda_orphan_drug": [ { "pubchem_cid": 449171, "pubchem_sid": [ "11528315", "12014910", "24702085", "46507356", "53790163", "85240518", "103194296", "126645184", "127324022", "127324023", "127324024", "127324025", "127324026", "134985140", "135651418", "136342471", "160963868", "162172151", "310270343", "312217688", "313391998", "316538952", "318163351", "348275782", "349986946", "355141289", "376229699", "385784250", "386621124", "404615685", "434139548", "438446289", "440776372", "85165342", "349976458", "387153240" ], "inchikey": "SHGAZHPCJJPHSC-ZVCIMWCZSA-N", "generic_name": "Alitretinoin", "designated_date": "1992-04-10", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Ligand Pharmaceuticals, Inc.|10275 Science Center Dr.|San Diego|California|92121|United States", "orphan_designation": { "original_text": "Treatment of acute promyelocytic leukemia", "umls": "C0023487", "parsed_text": "Acute Promyelocytic Leukemia" } }, { "pubchem_cid": 14888, "pubchem_sid": [ "623898", "10500631", "46506448", "50843743", "103770961", "162231862", "224813979", "310267035", "316398034", "318160262", "375088034", "853981", "49854936", "134981320", "349982070", "363896028", "363903405", "387145578", "434132521", "53786879", "56352915", "144075984", "163644323", "242575069", "268735205", "386043103", "10506726", "160964503", "348283749", "440791074", "14708981", "49742627", "348281378", "2501", "398774", "529128", "50843742", "376229493", "385641391", "386635662", "441066189" ], "inchikey": "QTLQKAJBUDWPIB-UHFFFAOYSA-N", "generic_name": "Arsenic trioxide", "trade_name": "Trisenox", "designated_date": "1998-03-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "In combination with tretinoin for treatment of adults with newly-diagnosed low-risk acute promyelocytic leukemia (APL) whose APL is characterized by the presence of the t(15;17) translocation or PML/RAR-alpha gene expression.", "marketing_approval_date": "2018-01-12", "exclusivity_end_date": "2025-01-12", "exclusivity_protected_indication": "In combination with tretinoin for treatment of adults with newly-diagnosed low-risk acute promyelocytic leukemia (APL) whose APL is characterized by the presence of the t(15;17) translocation or PML/RAR-alpha gene expression.", "sponsor": "Teva Branded Pharmaceutical Products R&D, Inc.|41 Moores Road|Frazer|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of acute promyelocytic leukemia.", "umls": "C0023487", "parsed_text": "Acute Promyelocytic Leukemia" } }, { "pubchem_cid": 14888, "pubchem_sid": [ "623898", "10500631", "46506448", "50843743", "103770961", "162231862", "224813979", "310267035", "316398034", "318160262", "375088034", "853981", "49854936", "134981320", "349982070", "363896028", "363903405", "387145578", "434132521", "53786879", "56352915", "144075984", "163644323", "242575069", "268735205", "386043103", "10506726", "160964503", "348283749", "440791074", "14708981", "49742627", "348281378", "2501", "398774", "529128", "50843742", "376229493", "385641391", "386635662", "441066189" ], "inchikey": "QTLQKAJBUDWPIB-UHFFFAOYSA-N", "generic_name": "Arsenic trioxide", "trade_name": "Trisenox", "designated_date": "1998-03-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "For induction of remission and consolidation in patients with acute promyelocytic leukemia (APL) who are refractory to, or have relapsed from, retinoid and anthracycline chemotherapy, and whose APL is characterized by the presence of the t(15;17) translocation of PML/RAR-alpha gene expression.", "marketing_approval_date": "2000-09-25", "exclusivity_end_date": "2007-09-25", "sponsor": "Teva Branded Pharmaceutical Products R&D, Inc.|41 Moores Road|Frazer|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of acute promyelocytic leukemia.", "umls": "C0023487", "parsed_text": "Acute Promyelocytic Leukemia" } }, { "pubchem_cid": 5258, "pubchem_sid": [ "12014956", "50064540", "53786771", "103701587", "134979866", "163835443", "252156710", "312694077", "318050537", "318152710", "319063421", "319554213", "332865618", "341136446", "341150739", "347913960", "349991161", "354703255", "355039317", "375080605", "377310203", "382157175", "385640956", "386635219", "387060549", "387165953", "404425828", "404616844", "433910286", "434132300", "434311858", "438490437", "440066200", "441005021", "163266957", "254760153", "318160067", "375685005", "383232179", "438482966", "322094464", "348833276", "144076078" ], "inchikey": "VPZRWNZGLKXFOE-UHFFFAOYSA-M", "generic_name": "Phenylbutyrate", "designated_date": "2000-01-19", "designation_status": 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"Aldesleukin", "trade_name": "Proleukin", "designated_date": "1996-09-10", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of adults with metastatic melanoma.", "marketing_approval_date": "1998-01-09", "exclusivity_end_date": "2005-01-09", "sponsor": "Chiron Corporation|4560 Horton Street|Emeryville|California|94608|United States", "orphan_designation": { "original_text": "Treatment of metastatic melanoma.", "umls": "C0278883", "parsed_text": "Metastatic melanoma" } }, { "pubchem_sid": 597554, "generic_name": "Aldesleukin", "trade_name": "Proleukin", "designated_date": "1996-09-10", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of adults with metastatic melanoma.", "marketing_approval_date": "1998-01-09", "exclusivity_end_date": "2005-01-09", "sponsor": "Chiron Corporation|4560 Horton Street|Emeryville|California|94608|United States", "orphan_designation": { "original_text": "Treatment of metastatic melanoma.", "umls": "C0278883", "parsed_text": "Metastatic melanoma" } }, { "pubchem_sid": 10105, "generic_name": "Aldesleukin", "trade_name": "Proleukin", "designated_date": "1996-09-10", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of adults with metastatic melanoma.", "marketing_approval_date": "1998-01-09", "exclusivity_end_date": "2005-01-09", "sponsor": "Chiron Corporation|4560 Horton Street|Emeryville|California|94608|United States", "orphan_designation": { "original_text": "Treatment of metastatic melanoma.", "umls": "C0278883", "parsed_text": "Metastatic melanoma" } }, { "pubchem_sid": 160693120, "generic_name": "Bacillus Calmette-Guerin vaccine", "designated_date": "2006-08-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "OncoVac Corporation|P. O. Box 970|Pacific Palisades|California|90272|United States", "orphan_designation": { "original_text": "Treatment of stage IIb through IV metastatic melanoma", "umls": "C0278883", "parsed_text": "Metastatic melanoma" } }, { "pubchem_sid": 349427652, "generic_name": "etaracizumab", "designated_date": "2004-04-14", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "MedImmune, LLC|One MedImmune Way|Gaithersburg|Maryland|20878|United States", "orphan_designation": { "original_text": "For the treatment of of stage IIb, stage IIc, stage III and stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 347911300, "generic_name": "etaracizumab", "designated_date": "2004-04-14", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "MedImmune, LLC|One MedImmune Way|Gaithersburg|Maryland|20878|United States", "orphan_designation": { "original_text": "For the treatment of of stage IIb, stage IIc, stage III and stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 178103209, "generic_name": "etaracizumab", "designated_date": "2004-04-14", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "MedImmune, LLC|One MedImmune Way|Gaithersburg|Maryland|20878|United States", "orphan_designation": { "original_text": "For the treatment of of stage IIb, stage IIc, stage III and stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 160666626, "generic_name": "etaracizumab", "designated_date": "2004-04-14", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "MedImmune, LLC|One MedImmune Way|Gaithersburg|Maryland|20878|United States", "orphan_designation": { "original_text": "For the treatment of of stage IIb, stage IIc, stage III and stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 135323725, "generic_name": "etaracizumab", "designated_date": "2004-04-14", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "MedImmune, LLC|One MedImmune Way|Gaithersburg|Maryland|20878|United States", "orphan_designation": { "original_text": "For the treatment of of stage IIb, stage IIc, stage III and stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 347911239, "generic_name": "tremelimumab", "designated_date": "2006-09-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "MedImmune|One Medimmune Way|Gaithersburg|Maryland|20878|United States", "orphan_designation": { "original_text": "Treatment of stage IIb to stage IV metastatic melanoma", "umls": "C0278883", "parsed_text": "Metastatic melanoma" } }, { "pubchem_sid": 252166672, "generic_name": "tremelimumab", "designated_date": "2006-09-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "MedImmune|One Medimmune Way|Gaithersburg|Maryland|20878|United States", "orphan_designation": { "original_text": "Treatment of stage IIb to stage IV metastatic melanoma", "umls": "C0278883", "parsed_text": "Metastatic melanoma" } }, { "pubchem_sid": 160698442, "generic_name": "tremelimumab", "designated_date": "2006-09-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "MedImmune|One Medimmune Way|Gaithersburg|Maryland|20878|United States", "orphan_designation": { "original_text": "Treatment of stage IIb to stage IV metastatic melanoma", "umls": "C0278883", "parsed_text": "Metastatic melanoma" } }, { "pubchem_sid": 135304002, "generic_name": "tremelimumab", "designated_date": "2006-09-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "MedImmune|One Medimmune Way|Gaithersburg|Maryland|20878|United States", "orphan_designation": { "original_text": "Treatment of stage IIb to stage IV metastatic melanoma", "umls": "C0278883", "parsed_text": "Metastatic melanoma" } }, { "pubchem_sid": 387061107, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of unresectable or metastatic melanoma", "marketing_approval_date": "2011-03-25", "exclusivity_end_date": "2018-03-25", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 385327547, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of unresectable or metastatic melanoma", "marketing_approval_date": "2011-03-25", "exclusivity_end_date": "2018-03-25", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 384647090, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of unresectable or metastatic melanoma", "marketing_approval_date": "2011-03-25", "exclusivity_end_date": "2018-03-25", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 354355037, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of unresectable or metastatic melanoma", "marketing_approval_date": "2011-03-25", "exclusivity_end_date": "2018-03-25", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 349072362, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of unresectable or metastatic melanoma", "marketing_approval_date": "2011-03-25", "exclusivity_end_date": "2018-03-25", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 347910341, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of unresectable or metastatic melanoma", "marketing_approval_date": "2011-03-25", "exclusivity_end_date": "2018-03-25", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 329976988, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of unresectable or metastatic melanoma", "marketing_approval_date": "2011-03-25", "exclusivity_end_date": "2018-03-25", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 178103470, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of unresectable or metastatic melanoma", "marketing_approval_date": "2011-03-25", "exclusivity_end_date": "2018-03-25", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 135287563, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of unresectable or metastatic melanoma", "marketing_approval_date": "2011-03-25", "exclusivity_end_date": "2018-03-25", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 131273201, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of unresectable or metastatic melanoma", "marketing_approval_date": "2011-03-25", "exclusivity_end_date": "2018-03-25", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 440813738, "generic_name": "talimogene laherparepvec", "trade_name": "IMLYGIC", "designated_date": "2011-03-14", "designation_status": "Designated/Approved", "approved_labeled_indication": "Indicated for the local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery", "marketing_approval_date": "2015-10-27", "exclusivity_end_date": "2022-10-27", "exclusivity_protected_indication": "Indicated for the local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery", "sponsor": "BioVex, Inc. (subsidiary of Amgen)|Amgen|Mail Stop 17-2-B|Thousand Oaks|California|91320|United States", "orphan_designation": { "original_text": "Treatment of stage IIb-stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 347911465, "generic_name": "talimogene laherparepvec", "trade_name": "IMLYGIC", "designated_date": "2011-03-14", "designation_status": "Designated/Approved", "approved_labeled_indication": "Indicated for the local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery", "marketing_approval_date": "2015-10-27", "exclusivity_end_date": "2022-10-27", "exclusivity_protected_indication": "Indicated for the local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery", "sponsor": "BioVex, Inc. (subsidiary of Amgen)|Amgen|Mail Stop 17-2-B|Thousand Oaks|California|91320|United States", "orphan_designation": { "original_text": "Treatment of stage IIb-stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 160698451, "generic_name": "talimogene laherparepvec", "trade_name": "IMLYGIC", "designated_date": "2011-03-14", "designation_status": "Designated/Approved", "approved_labeled_indication": "Indicated for the local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery", "marketing_approval_date": "2015-10-27", "exclusivity_end_date": "2022-10-27", "exclusivity_protected_indication": "Indicated for the local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery", "sponsor": "BioVex, Inc. (subsidiary of Amgen)|Amgen|Mail Stop 17-2-B|Thousand Oaks|California|91320|United States", "orphan_designation": { "original_text": "Treatment of stage IIb-stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 135343059, "generic_name": "talimogene laherparepvec", "trade_name": "IMLYGIC", "designated_date": "2011-03-14", "designation_status": "Designated/Approved", "approved_labeled_indication": "Indicated for the local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery", "marketing_approval_date": "2015-10-27", "exclusivity_end_date": "2022-10-27", "exclusivity_protected_indication": "Indicated for the local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery", "sponsor": "BioVex, Inc. (subsidiary of Amgen)|Amgen|Mail Stop 17-2-B|Thousand Oaks|California|91320|United States", "orphan_designation": { "original_text": "Treatment of stage IIb-stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 135287563, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "For the adjuvant treatment of patients with cutaneous melanoma with pathologic involvement of regional lymph nodes of more than 1 mm, who have undergone complete resection including total lymphadenectomy.", "marketing_approval_date": "2015-10-28", "exclusivity_end_date": "2022-10-28", "exclusivity_protected_indication": "For the adjuvant treatment of patients with cutaneous melanoma with pathologic involvement of regional lymph nodes of more than 1 mm, who have undergone complete resection including total lymphadenectomy.", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 131273201, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "For the adjuvant treatment of patients with cutaneous melanoma with pathologic involvement of regional lymph nodes of more than 1 mm, who have undergone complete resection including total lymphadenectomy.", "marketing_approval_date": "2015-10-28", "exclusivity_end_date": "2022-10-28", "exclusivity_protected_indication": "For the adjuvant treatment of patients with cutaneous melanoma with pathologic involvement of regional lymph nodes of more than 1 mm, who have undergone complete resection including total lymphadenectomy.", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 387061107, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "For the adjuvant treatment of patients with cutaneous melanoma with pathologic involvement of regional lymph nodes of more than 1 mm, who have undergone complete resection including total lymphadenectomy.", "marketing_approval_date": "2015-10-28", "exclusivity_end_date": "2022-10-28", "exclusivity_protected_indication": "For the adjuvant treatment of patients with cutaneous melanoma with pathologic involvement of regional lymph nodes of more than 1 mm, who have undergone complete resection including total lymphadenectomy.", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 385327547, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "For the adjuvant treatment of patients with cutaneous melanoma with pathologic involvement of regional lymph nodes of more than 1 mm, who have undergone complete resection including total lymphadenectomy.", "marketing_approval_date": "2015-10-28", "exclusivity_end_date": "2022-10-28", "exclusivity_protected_indication": "For the adjuvant treatment of patients with cutaneous melanoma with pathologic involvement of regional lymph nodes of more than 1 mm, who have undergone complete resection including total lymphadenectomy.", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 384647090, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "For the adjuvant treatment of patients with cutaneous melanoma with pathologic involvement of regional lymph nodes of more than 1 mm, who have undergone complete resection including total lymphadenectomy.", "marketing_approval_date": "2015-10-28", "exclusivity_end_date": "2022-10-28", "exclusivity_protected_indication": "For the adjuvant treatment of patients with cutaneous melanoma with pathologic involvement of regional lymph nodes of more than 1 mm, who have undergone complete resection including total lymphadenectomy.", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 354355037, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "For the adjuvant treatment of patients with cutaneous melanoma with pathologic involvement of regional lymph nodes of more than 1 mm, who have undergone complete resection including total lymphadenectomy.", "marketing_approval_date": "2015-10-28", "exclusivity_end_date": "2022-10-28", "exclusivity_protected_indication": "For the adjuvant treatment of patients with cutaneous melanoma with pathologic involvement of regional lymph nodes of more than 1 mm, who have undergone complete resection including total lymphadenectomy.", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 349072362, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "For the adjuvant treatment of patients with cutaneous melanoma with pathologic involvement of regional lymph nodes of more than 1 mm, who have undergone complete resection including total lymphadenectomy.", "marketing_approval_date": "2015-10-28", "exclusivity_end_date": "2022-10-28", "exclusivity_protected_indication": "For the adjuvant treatment of patients with cutaneous melanoma with pathologic involvement of regional lymph nodes of more than 1 mm, who have undergone complete resection including total lymphadenectomy.", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 347910341, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "For the adjuvant treatment of patients with cutaneous melanoma with pathologic involvement of regional lymph nodes of more than 1 mm, who have undergone complete resection including total lymphadenectomy.", "marketing_approval_date": "2015-10-28", "exclusivity_end_date": "2022-10-28", "exclusivity_protected_indication": "For the adjuvant treatment of patients with cutaneous melanoma with pathologic involvement of regional lymph nodes of more than 1 mm, who have undergone complete resection including total lymphadenectomy.", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 329976988, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "For the adjuvant treatment of patients with cutaneous melanoma with pathologic involvement of regional lymph nodes of more than 1 mm, who have undergone complete resection including total lymphadenectomy.", "marketing_approval_date": "2015-10-28", "exclusivity_end_date": "2022-10-28", "exclusivity_protected_indication": "For the adjuvant treatment of patients with cutaneous melanoma with pathologic involvement of regional lymph nodes of more than 1 mm, who have undergone complete resection including total lymphadenectomy.", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 178103470, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "For the adjuvant treatment of patients with cutaneous melanoma with pathologic involvement of regional lymph nodes of more than 1 mm, who have undergone complete resection including total lymphadenectomy.", "marketing_approval_date": "2015-10-28", "exclusivity_end_date": "2022-10-28", "exclusivity_protected_indication": "For the adjuvant treatment of patients with cutaneous melanoma with pathologic involvement of regional lymph nodes of more than 1 mm, who have undergone complete resection including total lymphadenectomy.", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 387061107, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "treatment of unresectable or metastatic melanoma in adults and pediatric patients (12 years and older)", "marketing_approval_date": "2017-07-21", "exclusivity_end_date": "2024-07-21", "exclusivity_protected_indication": "treatment of unresectable or metastatic melanoma in pediatric patients (12 years and older).", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 385327547, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "treatment of unresectable or metastatic melanoma in adults and pediatric patients (12 years and older)", "marketing_approval_date": "2017-07-21", "exclusivity_end_date": "2024-07-21", "exclusivity_protected_indication": "treatment of unresectable or metastatic melanoma in pediatric patients (12 years and older).", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 384647090, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "treatment of unresectable or metastatic melanoma in adults and pediatric patients (12 years and older)", "marketing_approval_date": "2017-07-21", "exclusivity_end_date": "2024-07-21", "exclusivity_protected_indication": "treatment of unresectable or metastatic melanoma in pediatric patients (12 years and older).", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 354355037, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "treatment of unresectable or metastatic melanoma in adults and pediatric patients (12 years and older)", "marketing_approval_date": "2017-07-21", "exclusivity_end_date": "2024-07-21", "exclusivity_protected_indication": "treatment of unresectable or metastatic melanoma in pediatric patients (12 years and older).", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 349072362, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "treatment of unresectable or metastatic melanoma in adults and pediatric patients (12 years and older)", "marketing_approval_date": "2017-07-21", "exclusivity_end_date": "2024-07-21", "exclusivity_protected_indication": "treatment of unresectable or metastatic melanoma in pediatric patients (12 years and older).", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 347910341, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "treatment of unresectable or metastatic melanoma in adults and pediatric patients (12 years and older)", "marketing_approval_date": "2017-07-21", "exclusivity_end_date": "2024-07-21", "exclusivity_protected_indication": "treatment of unresectable or metastatic melanoma in pediatric patients (12 years and older).", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 329976988, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "treatment of unresectable or metastatic melanoma in adults and pediatric patients (12 years and older)", "marketing_approval_date": "2017-07-21", "exclusivity_end_date": "2024-07-21", "exclusivity_protected_indication": "treatment of unresectable or metastatic melanoma in pediatric patients (12 years and older).", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 178103470, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "treatment of unresectable or metastatic melanoma in adults and pediatric patients (12 years and older)", "marketing_approval_date": "2017-07-21", "exclusivity_end_date": "2024-07-21", "exclusivity_protected_indication": "treatment of unresectable or metastatic melanoma in pediatric patients (12 years and older).", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 135287563, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "treatment of unresectable or metastatic melanoma in adults and pediatric patients (12 years and older)", "marketing_approval_date": "2017-07-21", "exclusivity_end_date": "2024-07-21", "exclusivity_protected_indication": "treatment of unresectable or metastatic melanoma in pediatric patients (12 years and older).", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 131273201, "generic_name": "ipilimumab", "trade_name": "Yervoy", "designated_date": "2004-06-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "treatment of unresectable or metastatic melanoma in adults and pediatric patients (12 years and older)", "marketing_approval_date": "2017-07-21", "exclusivity_end_date": "2024-07-21", "exclusivity_protected_indication": "treatment of unresectable or metastatic melanoma in pediatric patients (12 years and older).", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of high risk Stage II, Stage III, and Stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } }, { "pubchem_sid": 381128027, "generic_name": "Coxsackievirus A21", "designated_date": "2005-12-15", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Viralytics Limited|P. O. Box 1045|Unit 8/33 Ryde Road,|Pymble,|NSW|Australia", "orphan_designation": { "original_text": "Treatment of stage II (T4), stage III, and stage IV melanoma", "umls": "C0278883", "parsed_text": "stage iv melanoma" } } ] }, { "_id": "UMLS:C3463824", "fda_orphan_drug": [ { "pubchem_cid": 2141, "pubchem_sid": [ "147094", "22425559", "50009355", "53788323", "103267861", "134995666", "160964477", "250133497", "318160183", "319061493", "348283725", "349980321", "12013090", "79732291", "318039390" ], "inchikey": "JKOQGQFVAUAYPM-UHFFFAOYSA-N", "generic_name": "Amifostine", "designated_date": "1999-10-04", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Clinigen, Inc.|790 Township Line Road|Suite 120|Yardley|Pennsylvania|19067|United States", "orphan_designation": { "original_text": "Treatment of myelodysplastic syndromes.", "umls": "C3463824", "parsed_text": "Myelodysplastic Syndromes" } }, { "pubchem_cid": 123805, "pubchem_sid": [ 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"designated_date": "1999-03-08", "designation_status": "Designated/Approved", "approved_labeled_indication": "for treatment of patients with myelodysplastic syndromes (MDS) including previously treated and untreated, de novo and secondary MDS of all French-American-British subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, and chronic myelomonocytic leukemia) and intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System groups", "marketing_approval_date": "2006-05-02", "exclusivity_end_date": "2013-05-02", "sponsor": "Otsuka Pharmaceutical Development|and Commercialization, Inc.|508 Carnegie Center Drive|Princeton|New Jersey|8540|United States", "orphan_designation": { "original_text": "Treatment of myelodysplastic syndromes.", "umls": "C3463824", "parsed_text": "Myelodysplastic Syndromes" } }, { "pubchem_cid": 71657455, "pubchem_sid": [ 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Pharm.D.|University Of Illinois At Chicago College of Pharm|833 South Wood Street (m/c886) Rm. 176|Chicago|Illinois|60612|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex.", "umls": "C0026914", "parsed_text": "Mycobacterium avium Complex" } }, { "pubchem_cid": 54368, "pubchem_sid": [ "463557", "50035076", "50663483", "135012947", "160693381", "319098383", "385762588", "386599263", "596153" ], "inchikey": "IOEMETRLOWNXGW-UHFFFAOYSA-N", "generic_name": "Piritrexim isethionate", "designated_date": "1988-06-13", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Burroughs Wellcome Company|3030 Cornwallis Road|P.O. Box 12700|Research Triangle Park|North Carolina|27709|United States", "orphan_designation": { "original_text": "Treatment of infections caused by Pneumocystis carinii, Toxoplasma gondii, and Mycobacterium avium-intracellulare.", "umls": [ "C0026914", "C0040557", "C3714514" ], "parsed_text": [ "Mycobacterium avium-intracellulare", "Toxoplasma gondii", "Infections" ] } }, { "pubchem_cid": 137313637, "pubchem_sid": [ "9444", "596006", "7890228", "7980515", "12012943", "17388744", "26697101", "46506468", "48425598", "50044776", "56323065", "57288827", "103914671", "126592920", "126628917", "135011172", "144204563", "144207956", "173905832", "223937742", "241131852", "250133567", "252220205", "252356474", "254761728", "310279728", "312228274", "315446114", "318154425", "318162198", "318166547", "341141592", "341835220", "347713867", "347759842", "385642307", "386478581", "387060858", "419536186", "438475565", "53787673", "75049157", "92717216", "103535182", "124766242", "126684731", "172080843", "251916303", "251917650", "275071157", "313082072", "315677114", "319311857", "319461145", "349979339", "381340159", "381358021", "382164364", "384255218", "384995394", "385453584", "440790604", "152258736", "160647581", "162170553", "164233446", "312695542", "319552594", "347914222", "377309763", "434305011", "438488046", "162224750", "348283269", "354265537", "375677640", "26719829", "49681785", "75049153", "75049154", "163137856", "172080344", "313074772", "347235005", "383548025", "387071533", "387071536", "387071537", "405500216", "440046872", "104170218", "185990407", "336348462", "346441003", "355050038", "377624929", "381183566", "384568084", "384571840", "46386607", "56463170", "75049155", "75049156", "117480934", "312650100", "318040690", "319433074", "342565542", "349275687", "354283700", "363671366", "375201744", "375562339", "375573268", "384585868", "434122534", "441079331" ], "inchikey": "ZWBTYMGEBZUQTK-SHULBWHJSA-N", "generic_name": "Rifabutin", "trade_name": "Mycobutin", "designated_date": "1989-12-18", "designation_status": "Designated/Approved", "approved_labeled_indication": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infections.", "marketing_approval_date": "1992-12-23", "exclusivity_end_date": "1999-12-23", "sponsor": "Adria Laboratories, Inc.|P.O. Box 16529|Columbus|Ohio|43216|United States", "orphan_designation": { "original_text": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infection.", "umls": [ "C0019693", "C0026914" ], "parsed_text": [ "HIV infection", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 137295261, "pubchem_sid": [ "9444", "596006", "7890228", "7980515", "12012943", "17388744", "26697101", "46506468", "48425598", "50044776", "56323065", "57288827", "103914671", "126592920", "126628917", "135011172", "144204563", "144207956", "173905832", "223937742", "241131852", "250133567", "252220205", "252356474", "254761728", "310279728", "312228274", "315446114", "318154425", "318162198", "318166547", "341141592", "341835220", "347713867", "347759842", "385642307", "386478581", "387060858", "419536186", "438475565", "53787673", "75049157", "92717216", "103535182", "124766242", "126684731", "172080843", "251916303", "251917650", "275071157", "313082072", "315677114", "319311857", "319461145", "349979339", "381340159", "381358021", "382164364", "384255218", "384995394", "385453584", "440790604", "152258736", "160647581", "162170553", "164233446", "312695542", "319552594", "347914222", "377309763", "434305011", "438488046", "162224750", "348283269", "354265537", "375677640", "26719829", "49681785", "75049153", "75049154", "163137856", "172080344", "313074772", "347235005", "383548025", "387071533", "387071536", "387071537", "405500216", "440046872", "104170218", "185990407", "336348462", "346441003", "355050038", "377624929", "381183566", "384568084", "384571840", "46386607", "56463170", "75049155", "75049156", "117480934", "312650100", "318040690", "319433074", "342565542", "349275687", "354283700", "363671366", "375201744", "375562339", "375573268", "384585868", "434122534", "441079331" ], "inchikey": "ZWBTYMGEBZUQTK-HBZSWILLSA-N", "generic_name": "Rifabutin", "trade_name": "Mycobutin", "designated_date": "1989-12-18", "designation_status": "Designated/Approved", "approved_labeled_indication": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infections.", "marketing_approval_date": "1992-12-23", "exclusivity_end_date": "1999-12-23", "sponsor": "Adria Laboratories, Inc.|P.O. Box 16529|Columbus|Ohio|43216|United States", "orphan_designation": { "original_text": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infection.", "umls": [ "C0019693", "C0026914" ], "parsed_text": [ "HIV infection", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135925740, "pubchem_sid": [ "9444", "596006", "7890228", "7980515", "12012943", "17388744", "26697101", "46506468", "48425598", "50044776", "56323065", "57288827", "103914671", "126592920", "126628917", "135011172", "144204563", "144207956", "173905832", "223937742", "241131852", "250133567", "252220205", "252356474", "254761728", "310279728", "312228274", "315446114", "318154425", "318162198", "318166547", "341141592", "341835220", "347713867", "347759842", "385642307", "386478581", "387060858", "419536186", "438475565", "53787673", "75049157", "92717216", "103535182", "124766242", "126684731", "172080843", "251916303", "251917650", "275071157", "313082072", "315677114", "319311857", "319461145", "349979339", "381340159", "381358021", "382164364", "384255218", "384995394", "385453584", "440790604", "152258736", "160647581", "162170553", "164233446", "312695542", "319552594", "347914222", "377309763", "434305011", "438488046", "162224750", "348283269", "354265537", "375677640", "26719829", "49681785", "75049153", "75049154", "163137856", "172080344", "313074772", "347235005", "383548025", "387071533", "387071536", "387071537", "405500216", "440046872", "104170218", "185990407", "336348462", "346441003", "355050038", "377624929", "381183566", "384568084", "384571840", "46386607", "56463170", "75049155", "75049156", "117480934", "312650100", "318040690", "319433074", "342565542", "349275687", "354283700", "363671366", "375201744", "375562339", "375573268", "384585868", "434122534", "441079331" ], "inchikey": "ZWBTYMGEBZUQTK-DNEPJJHDSA-N", "generic_name": "Rifabutin", "trade_name": "Mycobutin", "designated_date": "1989-12-18", "designation_status": "Designated/Approved", "approved_labeled_indication": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infections.", "marketing_approval_date": "1992-12-23", "exclusivity_end_date": "1999-12-23", "sponsor": "Adria Laboratories, Inc.|P.O. Box 16529|Columbus|Ohio|43216|United States", "orphan_designation": { "original_text": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infection.", "umls": [ "C0019693", "C0026914" ], "parsed_text": [ "HIV infection", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 136257950, "pubchem_sid": [ "9444", "596006", "7890228", "7980515", "12012943", "17388744", "26697101", "46506468", "48425598", "50044776", "56323065", "57288827", "103914671", "126592920", "126628917", "135011172", "144204563", "144207956", "173905832", "223937742", "241131852", "250133567", "252220205", "252356474", "254761728", "310279728", "312228274", "315446114", "318154425", "318162198", "318166547", "341141592", "341835220", "347713867", "347759842", "385642307", "386478581", "387060858", "419536186", "438475565", "53787673", "75049157", "92717216", "103535182", "124766242", "126684731", "172080843", "251916303", "251917650", "275071157", "313082072", "315677114", "319311857", "319461145", "349979339", "381340159", "381358021", "382164364", "384255218", "384995394", "385453584", "440790604", "152258736", "160647581", "162170553", "164233446", "312695542", "319552594", "347914222", "377309763", "434305011", "438488046", "162224750", "348283269", "354265537", "375677640", "26719829", "49681785", "75049153", "75049154", "163137856", "172080344", "313074772", "347235005", "383548025", "387071533", "387071536", "387071537", "405500216", "440046872", "104170218", "185990407", "336348462", "346441003", "355050038", "377624929", "381183566", "384568084", "384571840", "46386607", "56463170", "75049155", "75049156", "117480934", "312650100", "318040690", "319433074", "342565542", "349275687", "354283700", "363671366", "375201744", "375562339", "375573268", "384585868", "434122534", "441079331" ], "inchikey": "ZWBTYMGEBZUQTK-OZKAIBRQSA-N", "generic_name": "Rifabutin", "trade_name": "Mycobutin", "designated_date": "1989-12-18", "designation_status": "Designated/Approved", "approved_labeled_indication": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infections.", "marketing_approval_date": "1992-12-23", "exclusivity_end_date": "1999-12-23", "sponsor": "Adria Laboratories, Inc.|P.O. Box 16529|Columbus|Ohio|43216|United States", "orphan_designation": { "original_text": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infection.", "umls": [ "C0019693", "C0026914" ], "parsed_text": [ "HIV infection", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 136053407, "pubchem_sid": [ "9444", "596006", "7890228", "7980515", "12012943", "17388744", "26697101", "46506468", "48425598", "50044776", "56323065", "57288827", "103914671", "126592920", "126628917", "135011172", "144204563", "144207956", "173905832", "223937742", "241131852", "250133567", "252220205", "252356474", "254761728", "310279728", "312228274", "315446114", "318154425", "318162198", "318166547", "341141592", "341835220", "347713867", "347759842", "385642307", "386478581", "387060858", "419536186", "438475565", "53787673", "75049157", "92717216", "103535182", "124766242", "126684731", "172080843", "251916303", "251917650", "275071157", "313082072", "315677114", "319311857", "319461145", "349979339", "381340159", "381358021", "382164364", "384255218", "384995394", "385453584", "440790604", "152258736", "160647581", "162170553", "164233446", "312695542", "319552594", "347914222", "377309763", "434305011", "438488046", "162224750", "348283269", "354265537", "375677640", "26719829", "49681785", "75049153", "75049154", "163137856", "172080344", "313074772", "347235005", "383548025", "387071533", "387071536", "387071537", "405500216", "440046872", "104170218", "185990407", "336348462", "346441003", "355050038", "377624929", "381183566", "384568084", "384571840", "46386607", "56463170", "75049155", "75049156", "117480934", "312650100", "318040690", "319433074", "342565542", "349275687", "354283700", "363671366", "375201744", "375562339", "375573268", "384585868", "434122534", "441079331" ], "inchikey": "ZWBTYMGEBZUQTK-WOYMMMRKSA-N", "generic_name": "Rifabutin", "trade_name": "Mycobutin", "designated_date": "1989-12-18", "designation_status": "Designated/Approved", "approved_labeled_indication": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infections.", "marketing_approval_date": "1992-12-23", "exclusivity_end_date": "1999-12-23", "sponsor": "Adria Laboratories, Inc.|P.O. Box 16529|Columbus|Ohio|43216|United States", "orphan_designation": { "original_text": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infection.", "umls": [ "C0019693", "C0026914" ], "parsed_text": [ "HIV infection", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135458645, "pubchem_sid": [ "9444", "596006", "7890228", "7980515", "12012943", "17388744", "26697101", "46506468", "48425598", "50044776", "56323065", "57288827", "103914671", "126592920", "126628917", "135011172", "144204563", "144207956", "173905832", "223937742", "241131852", "250133567", "252220205", "252356474", "254761728", "310279728", "312228274", "315446114", "318154425", "318162198", "318166547", "341141592", "341835220", "347713867", "347759842", "385642307", "386478581", "387060858", "419536186", "438475565", "53787673", "75049157", "92717216", "103535182", "124766242", "126684731", "172080843", "251916303", "251917650", "275071157", "313082072", "315677114", "319311857", "319461145", "349979339", "381340159", "381358021", "382164364", "384255218", "384995394", "385453584", "440790604", "152258736", "160647581", "162170553", "164233446", "312695542", "319552594", "347914222", "377309763", "434305011", "438488046", "162224750", "348283269", "354265537", "375677640", "26719829", "49681785", "75049153", "75049154", "163137856", "172080344", "313074772", "347235005", "383548025", "387071533", "387071536", "387071537", "405500216", "440046872", "104170218", "185990407", "336348462", "346441003", "355050038", "377624929", "381183566", "384568084", "384571840", "46386607", "56463170", "75049155", "75049156", "117480934", "312650100", "318040690", "319433074", "342565542", "349275687", "354283700", "363671366", "375201744", "375562339", "375573268", "384585868", "434122534", "441079331" ], "inchikey": "ZWBTYMGEBZUQTK-OZFKZPMASA-N", "generic_name": "Rifabutin", "trade_name": "Mycobutin", "designated_date": "1989-12-18", "designation_status": "Designated/Approved", "approved_labeled_indication": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infections.", "marketing_approval_date": "1992-12-23", "exclusivity_end_date": "1999-12-23", "sponsor": "Adria Laboratories, Inc.|P.O. Box 16529|Columbus|Ohio|43216|United States", "orphan_designation": { "original_text": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infection.", "umls": [ "C0019693", "C0026914" ], "parsed_text": [ "HIV infection", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 136117120, "pubchem_sid": [ "9444", "596006", "7890228", "7980515", "12012943", "17388744", "26697101", "46506468", "48425598", "50044776", "56323065", "57288827", "103914671", "126592920", "126628917", "135011172", "144204563", "144207956", "173905832", "223937742", "241131852", "250133567", "252220205", "252356474", "254761728", "310279728", "312228274", "315446114", "318154425", "318162198", "318166547", "341141592", "341835220", "347713867", "347759842", "385642307", "386478581", "387060858", "419536186", "438475565", "53787673", "75049157", "92717216", "103535182", "124766242", "126684731", "172080843", "251916303", "251917650", "275071157", "313082072", "315677114", "319311857", "319461145", "349979339", "381340159", "381358021", "382164364", "384255218", "384995394", "385453584", "440790604", "152258736", "160647581", "162170553", "164233446", "312695542", "319552594", "347914222", "377309763", "434305011", "438488046", "162224750", "348283269", "354265537", "375677640", "26719829", "49681785", "75049153", "75049154", "163137856", "172080344", "313074772", "347235005", "383548025", "387071533", "387071536", "387071537", "405500216", "440046872", "104170218", "185990407", "336348462", "346441003", "355050038", "377624929", "381183566", "384568084", "384571840", "46386607", "56463170", "75049155", "75049156", "117480934", "312650100", "318040690", "319433074", "342565542", "349275687", "354283700", "363671366", "375201744", "375562339", "375573268", "384585868", "434122534", "441079331" ], "inchikey": "ZWBTYMGEBZUQTK-AMWSOCTHSA-N", "generic_name": "Rifabutin", "trade_name": "Mycobutin", "designated_date": "1989-12-18", "designation_status": "Designated/Approved", "approved_labeled_indication": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infections.", "marketing_approval_date": "1992-12-23", "exclusivity_end_date": "1999-12-23", "sponsor": "Adria Laboratories, Inc.|P.O. Box 16529|Columbus|Ohio|43216|United States", "orphan_designation": { "original_text": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infection.", "umls": [ "C0019693", "C0026914" ], "parsed_text": [ "HIV infection", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135950975, "pubchem_sid": [ "9444", "596006", "7890228", "7980515", "12012943", "17388744", "26697101", "46506468", "48425598", "50044776", "56323065", "57288827", "103914671", "126592920", "126628917", "135011172", "144204563", "144207956", "173905832", "223937742", "241131852", "250133567", "252220205", "252356474", "254761728", "310279728", "312228274", "315446114", "318154425", "318162198", "318166547", "341141592", "341835220", "347713867", "347759842", "385642307", "386478581", "387060858", "419536186", "438475565", "53787673", "75049157", "92717216", "103535182", "124766242", "126684731", "172080843", "251916303", "251917650", "275071157", "313082072", "315677114", "319311857", "319461145", "349979339", "381340159", "381358021", "382164364", "384255218", "384995394", "385453584", "440790604", "152258736", "160647581", "162170553", "164233446", "312695542", "319552594", "347914222", "377309763", "434305011", "438488046", "162224750", "348283269", "354265537", "375677640", "26719829", "49681785", "75049153", "75049154", "163137856", "172080344", "313074772", "347235005", "383548025", "387071533", "387071536", "387071537", "405500216", "440046872", "104170218", "185990407", "336348462", "346441003", "355050038", "377624929", "381183566", "384568084", "384571840", "46386607", "56463170", "75049155", "75049156", "117480934", "312650100", "318040690", "319433074", "342565542", "349275687", "354283700", "363671366", "375201744", "375562339", "375573268", "384585868", "434122534", "441079331" ], "inchikey": "ZWBTYMGEBZUQTK-ASBZJICESA-N", "generic_name": "Rifabutin", "trade_name": "Mycobutin", "designated_date": "1989-12-18", "designation_status": "Designated/Approved", "approved_labeled_indication": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infections.", "marketing_approval_date": "1992-12-23", "exclusivity_end_date": "1999-12-23", "sponsor": "Adria Laboratories, Inc.|P.O. 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"designated_date": "1989-12-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Pfizer Inc.|235 East 42nd Street|New York|New York|10017|United States", "orphan_designation": { "original_text": "Treatment of disseminated Mycobacterium avium complex disease.", "umls": "C0026914", "parsed_text": "Mycobacterium avium Complex" } }, { "pubchem_cid": 136117120, "pubchem_sid": [ "9444", "596006", "7890228", "7980515", "12012943", "17388744", "26697101", "46506468", "48425598", "50044776", "56323065", "57288827", "103914671", "126592920", "126628917", "135011172", "144204563", "144207956", "173905832", "223937742", "241131852", "250133567", "252220205", "252356474", "254761728", "310279728", "312228274", "315446114", "318154425", "318162198", "318166547", "341141592", "341835220", "347713867", "347759842", "385642307", "386478581", "387060858", "419536186", "438475565", "53787673", "75049157", "92717216", "103535182", "124766242", 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"generic_name": "Rifabutin", "designated_date": "1989-12-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Pfizer Inc.|235 East 42nd Street|New York|New York|10017|United States", "orphan_designation": { "original_text": "Treatment of disseminated Mycobacterium avium complex disease.", "umls": "C0026914", "parsed_text": "Mycobacterium avium Complex" } }, { "pubchem_cid": 135398743, "pubchem_sid": [ "9444", "596006", "7890228", "7980515", "12012943", "17388744", "26697101", "46506468", "48425598", "50044776", "56323065", "57288827", "103914671", "126592920", "126628917", "135011172", "144204563", "144207956", "173905832", "223937742", "241131852", "250133567", "252220205", "252356474", "254761728", "310279728", "312228274", "315446114", "318154425", "318162198", "318166547", "341141592", "341835220", "347713867", "347759842", "385642307", "386478581", "387060858", "419536186", "438475565", "53787673", "75049157", 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"ZWBTYMGEBZUQTK-PVLSIAFMSA-N", "generic_name": "Rifabutin", "designated_date": "1989-12-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Pfizer Inc.|235 East 42nd Street|New York|New York|10017|United States", "orphan_designation": { "original_text": "Treatment of disseminated Mycobacterium avium complex disease.", "umls": "C0026914", "parsed_text": "Mycobacterium avium Complex" } }, { "pubchem_cid": 138404395, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", 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"WDZCUPBHRAEYDL-YUUOHQKVSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 137235501, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-RDKOBYJASA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 137091916, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-LBJWSYCFSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 137030254, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-USLNORDZSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135887820, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-QFWJEWEWSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 136122663, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-FHDUCCPFSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135932821, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-METIDKAPSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135925739, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-PWXWUWFUSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135749824, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-HJTBMZGUSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 136246610, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-BGZYVKEHSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135968625, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-LYDPARFQSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 136238508, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-NAFQJNKLSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 136073066, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-QEDJKGLXSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135659016, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-PRHSTWAZSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135403821, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-GZAUEHORSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 138404395, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-YUUOHQKVSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 137235501, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-RDKOBYJASA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 137091916, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-LBJWSYCFSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 137030254, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-USLNORDZSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135887820, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-QFWJEWEWSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 136122663, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-FHDUCCPFSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135932821, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-METIDKAPSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135925739, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-PWXWUWFUSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135749824, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-HJTBMZGUSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 136246610, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-BGZYVKEHSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135968625, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-LYDPARFQSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 136238508, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-NAFQJNKLSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 136073066, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-QEDJKGLXSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135659016, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-PRHSTWAZSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135403821, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-GZAUEHORSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } } ] }, { "_id": "MONDO:0005477", "fda_orphan_drug": [ { "pubchem_cid": 2157, "pubchem_sid": [ "9041", "650805", "4266412", "7978689", "8145887", "46507387", "46519007", "48415552", "49988107", "53789126", "57288745", "78623712", "93166583", "93167164", "103188875", "126691820", "127307239", "127307240", "127307241", "134981709", "135649937", "144203634", "160964452", "162174499", "170464920", "210275062", "210280700", "249950506", "251898013", "252390172", "254769795", "255441459", "274348942", "310266762", "312228645", "313075700", "315444013", "315673281", "318160203", "319061506", "319552841", "341140082", "341843560", "347814505", "348283703", "348401484", "348874032", "349989353", "355039470", "363593818", "363907306", "375092751", "375666873", "380998483", "381986765", "385783989", "386213771", "386620849", "387065046", "387107021", "404616648", "404754547", "405467723", "419533141", "433778692", "434140932", "438448453", "438610834", "440681798", "440790259", "85083745", "103755983", "348397099", "78623713", "163132631", "441073597" ], "inchikey": "IYIKLHRQXLHMJQ-UHFFFAOYSA-N", "generic_name": "Amiodarone", "designated_date": "1993-08-17", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Academic Pharmaceuticals, Inc.|21 N. Skokie Valley Highway|Suite G3|Lake Bluff|Illinois|60044|United States", "orphan_designation": { "original_text": "Treatment of incessant ventricular tachycardia.", "umls": "C0042514", "parsed_text": "Ventricular tachycardia" } }, { "pubchem_cid": 441325, "pubchem_sid": [ "134992824", "162175495", "187072739", "273013788", "312694173", "318153718", "318241386", "319222139", "347913467", "348877603", "350096048", "363662737", "375577789", "377309543", "382157941", "440062146", "49988108", "126665895", "160964452", "318160203" ], "inchikey": "ITPDYQOUSLNIHG-UHFFFAOYSA-N", "generic_name": "Amiodarone HCl", "trade_name": "Cordarone", "designated_date": "1994-03-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "For initiation of treatment and prophylaxis of frequently recurring ventricular fibrillation and hemodynamically unstable ventricular tachycardia in patients refractory to other therapy.", "marketing_approval_date": "1995-08-03", "exclusivity_end_date": "2002-08-03", "sponsor": "Wyeth-Ayerst Laboratories|P.O. Box 8299|Philadelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "For the acute treatment and prophylaxis of life-threatening ventricular tachycardia or ventricular fibrillation.", "umls": [ "C0042510", "C0042514" ], "parsed_text": [ "Ventricular Fibrillation", "Ventricular tachycardia" ] } } ] }, { "_id": "MONDO:0000190", "fda_orphan_drug": [ { "pubchem_cid": 441325, "pubchem_sid": [ "134992824", "162175495", "187072739", "273013788", "312694173", "318153718", "318241386", "319222139", "347913467", "348877603", "350096048", "363662737", "375577789", "377309543", "382157941", "440062146", "49988108", "126665895", "160964452", "318160203" ], "inchikey": "ITPDYQOUSLNIHG-UHFFFAOYSA-N", "generic_name": "Amiodarone HCl", "trade_name": "Cordarone", "designated_date": "1994-03-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "For initiation of treatment and prophylaxis of frequently recurring ventricular fibrillation and hemodynamically unstable ventricular tachycardia in patients refractory to other therapy.", "marketing_approval_date": "1995-08-03", "exclusivity_end_date": "2002-08-03", "sponsor": "Wyeth-Ayerst Laboratories|P.O. Box 8299|Philadelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "For the acute treatment and prophylaxis of life-threatening ventricular tachycardia or ventricular fibrillation.", "umls": [ "C0042510", "C0042514" ], "parsed_text": [ "Ventricular Fibrillation", "Ventricular tachycardia" ] } }, { "pubchem_cid": 16048622, "pubchem_sid": [ "160709295", "340516596", "347260842", "381413145", "385780109", "386616805", "404616596", "405438808", "439985476", "794603", "49974301", "74707395", "134222405", "134974541", "160963565", "318160365", "319061058", "319066630", "341311437", "348282925", "387181616", "406774", "319373564", "434143273", "438449547", "24434806" ], "inchikey": "YTIJUXVIZLYQTB-UHFFFAOYSA-N", "generic_name": "Bethanidine sulfate", "designated_date": "1989-11-24", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Medco Research, Inc.|8455 Beverly Blvd.|Suite 308|Los Angeles|California|90048|United States", "orphan_designation": { "original_text": "Prevention of recurrence of primary ventricular fibrillation.", "umls": "C0042510", "parsed_text": "Ventricular Fibrillation" } }, { "pubchem_cid": 23615599, "pubchem_sid": [ "160709295", "340516596", "347260842", "381413145", "385780109", "386616805", "404616596", "405438808", "439985476", "794603", "49974301", "74707395", "134222405", "134974541", "160963565", "318160365", "319061058", "319066630", "341311437", "348282925", "387181616", "406774", "319373564", "434143273", "438449547", "24434806" ], "inchikey": "WDLCALUKNKNIDF-UHFFFAOYSA-N", "generic_name": "Bethanidine sulfate", "designated_date": "1989-11-24", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Medco Research, Inc.|8455 Beverly Blvd.|Suite 308|Los Angeles|California|90048|United States", "orphan_designation": { "original_text": "Prevention of recurrence of primary 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Arques Avenue|Sunnyvale|California|94085|United States", "orphan_designation": { "original_text": "Treatment of follicular lymphoma", "umls": "C0024301", "parsed_text": "follicular lymphoma" } }, { "pubchem_cid": 135565596, "pubchem_sid": [ "152258695", "160647529", "163643093", "223366204", "242613212", "252155314", "255343066", "315706346", "317223391", "318698080", "319381987", "319436538", "319468181", "329878768", "336425826", "340575831", "341189834", "346405739", "347828721", "354279017", "355040286", "375980344", "375989707", "383521005", "384568782", "384572793", "385645351", "386481706", "387141778", "387256501", "401963061", "405131996", "440799029", "440804529", "440810951", "335987030", "375758803" ], "inchikey": "MWYDSXOGIBMAET-UHFFFAOYSA-N", "generic_name": "copanlisib", "trade_name": "ALIQOPA", "designated_date": "2015-02-05", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of adult patients with relapsed follicular lymphoma who have received at least two prior systemic therapies", "marketing_approval_date": "2017-09-14", "exclusivity_end_date": "2024-09-14", "exclusivity_protected_indication": "Treatment of adult patients with relapsed follicular lymphoma who have received at least two prior systemic therapies", "sponsor": "Bayer US LLC|100 Bayer Boulevard|P. O. Box 915|Hanover|New Jersey|7981|United States", "orphan_designation": { "original_text": "Treatment of follicular lymphoma", "umls": "C0024301", "parsed_text": "follicular lymphoma" } }, { "pubchem_cid": 50905713, "pubchem_sid": [ "152258752", "160647597", "164117634", "174510187", "223366126", "255344084", "277425227", "312616237", "312811442", "315713272", "319553498", "336302237", "341482333", "346410390", "347713936", "347828278", "355040244", "374387250", "375076051", "375577364", "375590831", "375675492", "384569009", "385650693", "386487143", "439576129", "440062354", "176251577", "405442061", "439988728" ], "inchikey": "SJVQHLPISAIATJ-ZDUSSCGKSA-N", "generic_name": "duvelisib", "trade_name": "COPIKTRA", "designated_date": "2013-08-01", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of adult patients with relapsed or refractory follicular lymphoma after at least two prior therapies", "marketing_approval_date": "2018-09-24", 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indicated for: the treatment of adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) whose tumors are positive for an EZH2 mutation as detected by an FDA-approved test and who have received at least 2 prior systemic therapies; and the treatment of adult patients with R/R FL who have no satisfactory alternative treatment options.", "marketing_approval_date": "2020-06-18", "sponsor": "Epizyme, Inc.|400 Technology Square|4th Floor|Cambridge|Massachusetts|2139|United States", "orphan_designation": { "original_text": "Treatment of follicular lymphoma", "umls": "C0024301", "parsed_text": "follicular lymphoma" } }, { "pubchem_cid": 86677874, "pubchem_sid": [ "348351247", "355193653", "375588996", "375589278", "376241122", "381120174", "381974236", "404613106", "404719128", "404772313", "405043662", "406847986", "434136627", "438443733", "381118808", "382146170" ], "inchikey": "ZQPDJCIXJHUERQ-QWRGUYRKSA-N", "generic_name": "Parsaclisib", "designated_date": "2019-09-25", 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"original_text": "1. Treatment of graft versus host disease in patients receiving bone marrow transplantation 2. 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Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Prevention of Graft versus Host Disease", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 386474197, "generic_name": "abatacept", "designated_date": "2017-12-26", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Co.|P. O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Prevention of Graft versus Host Disease", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 386467149, "generic_name": "abatacept", "designated_date": "2017-12-26", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Co.|P. O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Prevention of Graft versus Host Disease", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 178103473, "generic_name": "abatacept", "designated_date": "2017-12-26", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Co.|P. O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Prevention of Graft versus Host Disease", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 135355129, "generic_name": "abatacept", "designated_date": "2017-12-26", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Co.|P. O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Prevention of Graft versus Host Disease", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 103771521, "generic_name": "abatacept", "designated_date": "2017-12-26", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Co.|P. O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Prevention of Graft versus Host Disease", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 50070209, "generic_name": "abatacept", "designated_date": "2017-12-26", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Co.|P. O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Prevention of Graft versus Host Disease", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 46509198, "generic_name": "abatacept", "designated_date": "2017-12-26", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Co.|P. O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Prevention of Graft versus Host Disease", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 404719581, "generic_name": "alpha-1 proteinase inhibitor (human)", "designated_date": "2017-07-31", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Kamada, Ltd.|2 Holtzman Street|Rehovot|Israel", "orphan_designation": { "original_text": "Prevention of graft versus host disease", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 46506364, "generic_name": "alpha-1 proteinase inhibitor (human)", "designated_date": "2017-07-31", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Kamada, Ltd.|2 Holtzman Street|Rehovot|Israel", "orphan_designation": { "original_text": "Prevention of graft versus host disease", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 404719581, "generic_name": "alpha-1 proteinase inhibitor (human)", "designated_date": "2014-10-23", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Kamada Ltd.|2 Holtsman Street|Rehovot|Center District|Israel", "orphan_designation": { "original_text": "Treatment of graft versus host disease", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 46506364, "generic_name": "alpha-1 proteinase inhibitor (human)", "designated_date": "2014-10-23", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Kamada Ltd.|2 Holtsman Street|Rehovot|Center District|Israel", "orphan_designation": { "original_text": "Treatment of graft versus host disease", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 46506920, "generic_name": "anti-T-lymphocyte immune globulin, rabbit", "designated_date": "2010-03-26", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Neovii Biotech NA, Inc.|6 Liberty Square|PMB #295|Boston|Massachusetts|2109|United States", "orphan_designation": { "original_text": "Prevention of graft versus host disease (GVHD)", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 387060190, "generic_name": "Vedolizumab", "designated_date": "2017-03-21", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Millennium Pharmaceuticals, Inc.|40 Landsdowne Street|Cambridge|Massachusetts|2139|United States", "orphan_designation": { "original_text": "Treatment of graft versus host disease", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 385327554, "generic_name": "Vedolizumab", "designated_date": "2017-03-21", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Millennium Pharmaceuticals, Inc.|40 Landsdowne Street|Cambridge|Massachusetts|2139|United States", "orphan_designation": { "original_text": "Treatment of graft versus host disease", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 384647097, "generic_name": "Vedolizumab", "designated_date": "2017-03-21", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Millennium Pharmaceuticals, Inc.|40 Landsdowne Street|Cambridge|Massachusetts|2139|United States", "orphan_designation": { "original_text": "Treatment of graft versus host disease", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 354355051, "generic_name": "Vedolizumab", "designated_date": "2017-03-21", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Millennium Pharmaceuticals, Inc.|40 Landsdowne Street|Cambridge|Massachusetts|2139|United States", "orphan_designation": { "original_text": "Treatment of graft versus host disease", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 347910392, "generic_name": "Vedolizumab", "designated_date": "2017-03-21", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Millennium Pharmaceuticals, Inc.|40 Landsdowne Street|Cambridge|Massachusetts|2139|United States", "orphan_designation": { "original_text": "Treatment of graft versus host disease", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 178104009, "generic_name": "Vedolizumab", "designated_date": "2017-03-21", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Millennium Pharmaceuticals, Inc.|40 Landsdowne Street|Cambridge|Massachusetts|2139|United States", "orphan_designation": { "original_text": "Treatment of graft versus host disease", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 160666632, "generic_name": "Vedolizumab", "designated_date": "2017-03-21", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Millennium Pharmaceuticals, Inc.|40 Landsdowne Street|Cambridge|Massachusetts|2139|United States", "orphan_designation": { "original_text": "Treatment of graft versus host disease", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 135277804, "generic_name": "Vedolizumab", "designated_date": "2017-03-21", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Millennium Pharmaceuticals, Inc.|40 Landsdowne Street|Cambridge|Massachusetts|2139|United States", "orphan_designation": { "original_text": "Treatment of graft versus host disease", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 319259212, "generic_name": "talactoferrin alfa", "designated_date": "2003-08-20", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Agennix, Inc.|8 Greenway Plaza|Suite 910|Houston|Texas|77046|United States", "orphan_designation": { "original_text": "For the prevention of graft-versus-host disease", "umls": "C0018133", "parsed_text": "Graft-Versus-Host Disease" } }, { "pubchem_sid": 160676985, "generic_name": "talactoferrin alfa", "designated_date": "2003-08-20", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Agennix, Inc.|8 Greenway Plaza|Suite 910|Houston|Texas|77046|United States", "orphan_designation": { "original_text": "For the prevention of graft-versus-host disease", "umls": "C0018133", "parsed_text": "Graft-Versus-Host Disease" } }, { "pubchem_sid": 135286186, "generic_name": "talactoferrin alfa", "designated_date": "2003-08-20", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Agennix, Inc.|8 Greenway Plaza|Suite 910|Houston|Texas|77046|United States", "orphan_designation": { "original_text": "For the prevention of graft-versus-host disease", "umls": "C0018133", "parsed_text": "Graft-Versus-Host Disease" } }, { "pubchem_sid": 319259212, "generic_name": "talactoferrin alfa", "designated_date": "2003-08-20", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Agennix, Inc.|8 Greenway Plaza|Suite 910|Houston|Texas|77046|United States", "orphan_designation": { "original_text": "For the treatment of graft versus host disease", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 160676985, "generic_name": "talactoferrin alfa", "designated_date": "2003-08-20", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Agennix, Inc.|8 Greenway Plaza|Suite 910|Houston|Texas|77046|United States", "orphan_designation": { "original_text": "For the treatment of graft versus host disease", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 135286186, "generic_name": "talactoferrin alfa", "designated_date": "2003-08-20", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Agennix, Inc.|8 Greenway Plaza|Suite 910|Houston|Texas|77046|United States", "orphan_designation": { "original_text": "For the treatment of graft versus host disease", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 387060190, "generic_name": "vedolizumab", "designated_date": "2016-08-04", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Millennium Pharmaceuticals, Inc.|40 Landsdowne Street|Cambridge|Massachusetts|2139|United States", "orphan_designation": { "original_text": "Prevention of graft versus host disease.", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 385327554, "generic_name": "vedolizumab", "designated_date": "2016-08-04", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Millennium Pharmaceuticals, Inc.|40 Landsdowne Street|Cambridge|Massachusetts|2139|United States", "orphan_designation": { "original_text": "Prevention of graft versus host disease.", "umls": "C0018133", "parsed_text": "GRAFT VERSUS HOST DISEASE" } }, { "pubchem_sid": 384647097, "generic_name": "vedolizumab", "designated_date": "2016-08-04", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Millennium Pharmaceuticals, Inc.|40 Landsdowne Street|Cambridge|Massachusetts|2139|United States", "orphan_designation": { "original_text": 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York|10038|United States", "orphan_designation": { "original_text": "Treatment of AIDS-myelopathy.", "umls": [ "C0001175", "C0005956" ], "parsed_text": [ "AIDS", "myelopathy" ] } }, { "pubchem_cid": 23700083, "pubchem_sid": [ "103770833", "135017377", "135061797", "162183088", "184812943", "187071904", "223401724", "224357613", "252218510", "252350137", "254763278", "313081044", "316966941", "318168127", "319378167", "319378168", "346376750", "347759206", "355170075", "367383333", "375201988", "375632318", "376048984", "381415666", "384442059", "385727939", "386564558", "402287724", "402288192", "405425607", "434252468", "438456825", "439649607", "441026441", "49974979", "76491727", "92729715", "160964340", "318161200", "349979290", "126622253", "196106202", "316899206", "341215984", "355043084", "319312216", "348539004", "375574556", "404639316", "533593", "50028177", "126621270", "126648001", "126669933", "316899497", "319137263", "375296981", "433862530", "441076721" ], "inchikey": 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Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 137235501, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-RDKOBYJASA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 137091916, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-LBJWSYCFSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 137030254, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-USLNORDZSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135887820, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-QFWJEWEWSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 136122663, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-FHDUCCPFSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135932821, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-METIDKAPSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135925739, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-PWXWUWFUSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135749824, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-HJTBMZGUSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 136246610, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-BGZYVKEHSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135968625, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-LYDPARFQSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 136238508, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-NAFQJNKLSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 136073066, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-QEDJKGLXSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135659016, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-PRHSTWAZSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135403821, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-GZAUEHORSA-N", "generic_name": "Rifapentine", "designated_date": "1996-03-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel, Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Prophylactic treatment of Mycobacterium avium complex in patients with AIDS and a CD4+ count less than or equal to 75/mm3.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 76961227, "pubchem_sid": [ "611216", "12012645", "49974828", "77105216", "103504484", "103977023", "126591589", "126688706", "223442568", "241088157", "252559424", "254760962", "318041087", "318162965", "319298260", "347829107", "355181616", "404615922", "406167160", "406168308", "77105217", "249905171", "385639439", "386633650", "426078398", "434315414", "438493775", "77105218", "349067367", "375233855", "405486588", "440033246", "134973137", "384445465", "387126679", "543857", "549846", "77105219", "77105220", "77105221", "77105222", "127320128", "127320129", "127320130", "127320131", "127320132", "127320133", "127320134", "127320135", "127320136", "127320137", "127320138", "438637565", "439575205", "441071646" ], "inchikey": "MXWHMTNPTTVWDM-VEPASKNESA-N", "generic_name": "Mitoguazone", "designated_date": "1994-03-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "ILEX Oncology, Inc.|4545 Horizon Hill Blvd.|San Antonio|Texas|78229|United States", "orphan_designation": { "original_text": "Treatment of diffuse non-Hodgkin's lymphoma, including AIDS-related diffuse non-Hodgkin's lymphoma.", "umls": [ "C0001175", "C0348394" ], "parsed_text": [ "AIDS", "Diffuse non-Hodgkin's lymphoma" ] } }, { "pubchem_cid": 9991, "pubchem_sid": [ "611216", "12012645", "49974828", "77105216", "103504484", "103977023", "126591589", "126688706", "223442568", "241088157", "252559424", "254760962", "318041087", "318162965", "319298260", "347829107", "355181616", "404615922", "406167160", "406168308", "77105217", "249905171", "385639439", "386633650", "426078398", "434315414", "438493775", "77105218", "349067367", "375233855", "405486588", "440033246", "134973137", "384445465", "387126679", "543857", "549846", "77105219", "77105220", "77105221", "77105222", "127320128", "127320129", "127320130", "127320131", "127320132", "127320133", "127320134", "127320135", "127320136", "127320137", "127320138", "438637565", "439575205", "441071646" ], "inchikey": "MXWHMTNPTTVWDM-UHFFFAOYSA-N", "generic_name": "Mitoguazone", "designated_date": "1994-03-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "ILEX Oncology, Inc.|4545 Horizon Hill Blvd.|San Antonio|Texas|78229|United States", "orphan_designation": { "original_text": "Treatment of diffuse non-Hodgkin's lymphoma, including AIDS-related diffuse non-Hodgkin's lymphoma.", "umls": [ "C0001175", "C0348394" ], "parsed_text": [ "AIDS", "Diffuse non-Hodgkin's lymphoma" ] } }, { "pubchem_cid": 5363702, "pubchem_sid": [ "611216", "12012645", "49974828", "77105216", "103504484", "103977023", "126591589", "126688706", "223442568", "241088157", "252559424", "254760962", "318041087", "318162965", "319298260", "347829107", "355181616", "404615922", "406167160", "406168308", "77105217", "249905171", "385639439", "386633650", "426078398", "434315414", "438493775", "77105218", "349067367", "375233855", "405486588", "440033246", "134973137", "384445465", "387126679", "543857", "549846", "77105219", "77105220", "77105221", "77105222", "127320128", "127320129", "127320130", "127320131", "127320132", "127320133", "127320134", "127320135", "127320136", "127320137", "127320138", "438637565", "439575205", "441071646" ], "inchikey": "MXWHMTNPTTVWDM-XVVXZDBVSA-N", "generic_name": "Mitoguazone", "designated_date": "1994-03-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "ILEX Oncology, Inc.|4545 Horizon Hill Blvd.|San Antonio|Texas|78229|United States", "orphan_designation": { "original_text": "Treatment of diffuse non-Hodgkin's lymphoma, including AIDS-related diffuse non-Hodgkin's lymphoma.", "umls": [ "C0001175", "C0348394" ], "parsed_text": [ "AIDS", "Diffuse non-Hodgkin's lymphoma" ] } }, { "pubchem_cid": 5351154, "pubchem_sid": [ "611216", "12012645", "49974828", "77105216", "103504484", "103977023", "126591589", "126688706", "223442568", "241088157", "252559424", "254760962", "318041087", "318162965", "319298260", "347829107", "355181616", "404615922", "406167160", "406168308", "77105217", "249905171", "385639439", "386633650", "426078398", "434315414", "438493775", "77105218", "349067367", "375233855", "405486588", "440033246", "134973137", "384445465", "387126679", "543857", "549846", "77105219", "77105220", "77105221", "77105222", "127320128", "127320129", "127320130", "127320131", "127320132", "127320133", "127320134", "127320135", "127320136", "127320137", "127320138", "438637565", "439575205", "441071646" ], "inchikey": "MXWHMTNPTTVWDM-NXOFHUPFSA-N", "generic_name": "Mitoguazone", "designated_date": "1994-03-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "ILEX Oncology, Inc.|4545 Horizon Hill Blvd.|San Antonio|Texas|78229|United States", "orphan_designation": { "original_text": "Treatment of diffuse non-Hodgkin's lymphoma, including AIDS-related diffuse non-Hodgkin's lymphoma.", "umls": [ "C0001175", "C0348394" ], "parsed_text": [ "AIDS", "Diffuse non-Hodgkin's lymphoma" ] } }, { "pubchem_cid": 16132412, "pubchem_sid": [ "50070151", "126682713", "135236477", "277306965", "319360640", "340470689", "341312536", "341835253", "375640125", "385109733", "404617765", "434311125", "438489692", "439498162", "441060056", "277324764", "318162248", "319360641", "341105142", "349377778", "103771338", "178103577", "252218909", "319422362" ], "inchikey": "BVLCEKWPOSAKSZ-YQMCHIOTSA-N", "generic_name": "Sermorelin acetate", "designated_date": "1991-12-05", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "EMD Serono, Inc.|One Technology Place|Rockland|Massachusetts|2370|United States", "orphan_designation": { "original_text": "Treatment of AIDS-associated catabolism/weight loss.", "umls": [ "C0001175", "C4084774" ], "parsed_text": [ "AIDS", "Weight loss" ] } }, { "pubchem_cid": 24066, "pubchem_sid": [ "9416", "595891", "7980904", "11528334", "46386814", "46507879", "50613509", "53788261", "87322635", "87559804", "92711292", "103223943", "103914689", "104034365", "126631015", "126657446", "134987108", "144076133", "160964282", "163621070", "163686393", "178101530", "196110180", "252220297", "252451126", "254761771", "255370809", "273937038", "310281807", "312228515", "312343912", "312695583", "315446592", "316434515", "316968015", "318154519", "318162635", "318241159", "319079282", "319553721", "347912866", "348283552", "349291419", "349996132", 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"46508240", "48416710", "49718184", "49855311", "53787019", "56423125", "56463503", "57288875", "85148362", "87322636", "87559740", "103030916", "103188975", "103916892", "104170164", "134999198", "144075478", "160963841", "162103188", "162176907", "172080789", "178101527", "185969569", "211534963", "222449012", "223379717", "223769174", "242083114", "250133540", "252391243", "274875682", "277300196", "277309310", "310267193", "312228182", "312694464", "313082602", "316968019", "318162640", "319086468", "341140700", "346437783", "347757906", "348283164", "348961647", "349976250", "354267198", "354283911", "363593943", "374123362", "375202950", "375577874", "377309834", "382159716", "384255295", "384571765", "385644539", "386480866", "387060714", "387097737", "404758045", "409028757", "434314764", "438472563", "438494697", "438519130", "440790285", "441005551", "483676", "10320668", "126665393", "162030008", "250022689", "313075726", "370625866", "375586036", "386219458", "387097736", "126657343", "163621089", "163686412", "196110438", "255370813", "318154517", "319553962", "381831960", "404760991", "126624075", "170505133", "316898641", "375637562", "405478658", "440025317", "50071339", "50071528", "50071529", "74396021", "74396022", "74396023", "187072268", "252216641", "348268095", "438634465", "441076596" ], "inchikey": "HBOMLICNUCNMMY-XLPZGREQSA-N", "generic_name": "zidovudine", "trade_name": "Retrovir", "designated_date": "1985-07-17", "designation_status": "Designated/Approved", "approved_labeled_indication": "Management of certain adult patients with symptomatic HIV infection (AIDS and advanced ARC) who have a history of cytologically confirmed Pneumocytis carinii pneumonia (PCP) or an absolute CD4 (T4 helper/induccer) lymphocyte count of less than 200/mm in the peripheral blood before therapy is begun", "marketing_approval_date": "1987-03-19", "exclusivity_end_date": "1994-03-19", "sponsor": "Glaxo Wellcome Inc.|5 Moore Drive|Research Triangle Park|North Carolina|27709|United States", "orphan_designation": { "original_text": "Treatment of AIDS", "umls": "C0001175", "parsed_text": "AIDS" } }, { "pubchem_cid": 11683, "pubchem_sid": [ "10351", "114395", "856018", "24702330", "26719675", "46505827", "50019472", "50829485", "53789650", "56313653", "56394838", "56422102", "103771122", "103914544", "126624829", "134978661", "144089067", "144204020", "144212677", "160810968", "162177769", "162878405", "170465406", "172080272", "204369755", "210274924", "210280560", "251887750", "252218593", "252359523", "252401507", "254761372", "255370325", "273005686", "310276094", "312223991", "312695190", "313077356", "315445487", "315678281", "316898427", "318153706", "318161537", "319069892", "319447941", "322070048", "329907000", "336851391", "340509717", "340515716", "340519458", "341145455", "341834001", "347236982", "347807155", "347912602", "348397327", "349147978", "354267485", "355049886", "363898322", "374383785", "375577709", "381364505", "382162908", "384568021", "384571766", "385638982", "386633178", "387150131", "404615703", "404756889", "433699127", "433780503", "434131268", "434315839", "438469844", "438473357", "438644794", "438654496", "440063882", "441005220", "10528842", "50829484", "85087478", "126675730", "162092124", "176262261", "250110724", "375417560", "387208255", "438493466", "50829492", "144075109", "268734605", "376226555", "160963698", "316967204", "316967205", "375635533", "127316661", "127316662", "127316663", "127316664", "127316665", "127316666", "127316667", "127316668", "127316669", "127316670", "127316671", "127316672", "127316673", "127316674", "127316675", "127316676", "363662582", "377311007", "419581170", "46386702", "50829486", "50829487", "50829488", "50829489", "50829490", "50829491", "87560201", "136948188", "348283041", "441072669" ], "inchikey": "RQZAXGRLVPAYTJ-GQFGMJRRSA-N", "generic_name": "Megestrol acetate", "trade_name": "Megace", "designated_date": "1988-04-13", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of anorexia, cachexia, or an unexplained significant weight loss in patients with a diagnosis of acquired immune deficiency syndrome.", "marketing_approval_date": "1993-09-10", "exclusivity_end_date": "2000-09-10", "sponsor": "Bristol-Myers Squibb Pharmaceutical Research Institute|2400 West Lloyd Expressway|Evansville|Indiana|47721|United States", "orphan_designation": { "original_text": "Treatment of patients with anorexia, cachexia, or significant weight loss (=/>10% of baseline body weight) and confirmed diagnosis of AIDS.", "umls": [ "C0001175", "C0006625", "C4084774" ], "parsed_text": [ "AIDS", "Cachexia", "Weight loss" ] } }, { "pubchem_cid": 138404395, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-YUUOHQKVSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 137235501, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-RDKOBYJASA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 137091916, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-LBJWSYCFSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 137030254, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-USLNORDZSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135887820, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-QFWJEWEWSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 136122663, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-FHDUCCPFSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135932821, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-METIDKAPSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135925739, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-PWXWUWFUSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135749824, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-HJTBMZGUSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 136246610, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-BGZYVKEHSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135968625, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-LYDPARFQSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 136238508, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-NAFQJNKLSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 136073066, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-QEDJKGLXSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135659016, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-PRHSTWAZSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135403821, "pubchem_sid": [ "10259", "602920", "7890313", "12012718", "26697128", "46507322", "53787525", "56314339", "103630897", "126592919", "135005384", "144205988", "162175338", "187072496", "251912502", "251915482", "277309570", "312236611", "313082076", "315678520", "318154426", "340125456", "341142741", "341835224", "376051431", "381340405", "381363490", "384242519", "384570848", "384572141", "387060979", "387168649", "401961214", "440791049", "74986831", "124766243", "126670697", "312695261", "319311859", "319407172", "349987166", "355256559", "382163158", "384428729", "385782045", "386618781", "419493146", "434305194", "438488054", "126630082", "126656635", "196111631", "210279412", "210281735", "316898295", "319391863", "319554943", "335959485", "347914224", "355038791", "355042691", "162224751", "252216198", "273581120", "319408883", "319422442", "319426442", "322093686", "348283780", "375682388", "433909881", "164233457", "172080823", "315446115", "318162200", "319331747", "341136405", "387168650", "313098706", "405494553", "440041211", "26719839", "46386637", "49681773", "74986828", "93166827", "104170198", "136948047", "162248925", "223434755", "312650092", "318043068", "319332179", "319390862", "319391828", "319409096", "346418687", "363671367", "387148519", "50044762", "74986829", "74986830", "172080413", "241111074", "319431110", "329823987", "342577085", "349167303", "364195977", "375573280", "375991172", "377309791", "384586076", "441075669" ], "inchikey": "WDZCUPBHRAEYDL-GZAUEHORSA-N", "generic_name": "Rifapentine", "designated_date": "1995-06-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoechst Marion Roussel , Inc.|P.O. Box 9627|Mail Station: H3-M2516|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Mycobacterium avium complex in patients with AIDS.", "umls": [ "C0001175", "C0026914" ], "parsed_text": [ "AIDS", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 5215, "pubchem_sid": [ "9860", "92611", "602777", "855872", "7980699", "8149533", "10321441", "10532663", "11533903", "46506164", "50048769", "53789615", "57288844", "74579279", "85089635", "87560164", "103165644", "103913935", "104052024", "117528268", "124766246", "126631011", "126677933", "134971211", "144075225", "144203911", "160963706", "162177254", "174548883", "175442771", "187072281", "210279416", "210281739", "223704353", "223792891", "250064291", "252220242", "252356874", "252402264", "254761637", "255370654", "268734708", "275385710", "310280373", "312228084", "312695440", "313075600", "313082259", "313102087", "315342170", "315446269", "315680164", "316397509", "316898052", "318153261", "318162308", "319063382", "319450995", "319554947", "336300404", "340510654", "340515804", "341140441", "341832364", "347912887", "348283049", "348397534", "349245925", "349979672", "355038788", "363592680", "374032365", "374222846", "375197843", "375442010", "375575468", "375686019", "376074118", "376145490", "376226180", "377309478", "382163906", "385156116", "385650533", "386209693", "386486982", "387021962", "387152656", "387195866", "404617362", "404755137", "405068249", "405112811", "405498069", "406545685", "419580873", "433780652", "433781181", "433788291", "433788292", "434131794", "434310908", "438469956", "438472774", "438489433", "438644935", "440044725", "440064337", "440770280", "440790614", "441121337", "204359678", "342509453", "441070902" ], "inchikey": "SEEPANYCNGTZFQ-UHFFFAOYSA-N", "generic_name": "Sulfadiazine", "designated_date": "1994-03-14", "designation_status": "Designated/Approved", "approved_labeled_indication": "Toxoplasmosis, as adjunctive with pyrimethamine", "marketing_approval_date": "1994-07-29", "exclusivity_end_date": "2001-07-29", "sponsor": "Eon Labs Manufacturing, Inc.|227-15 North Conduit Avenue|Laurelton|New York|11413|United States", "orphan_designation": { "original_text": "For use in combination with pyrimethamine for the treatment of Toxoplasma gondii encephalitis in patients with and without AIDS.", "umls": [ "C0001175", "C0085315" ], "parsed_text": [ "AIDS", "toxoplasma gondii encephalitis" ] } }, { "pubchem_cid": 28343, "pubchem_sid": [ "57393539", "85343987", "127338651", "127338652", "224254170", "274608179", "438794267", "46504708", "49899241", "53788606", "103250414", "164157349" ], "inchikey": "LMBWSYZSUOEYSN-UHFFFAOYSA-M", "generic_name": "Diethyldithiocarbamate", "designated_date": "1986-04-03", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Connaught Laboratories|Route 611, P.O. Box 187|Swiftwater|Pennsylvania|18370|United States", "orphan_designation": { "original_text": "Treatment of AIDS.", "umls": "C0001175", "parsed_text": "AIDS" } }, { "pubchem_cid": 16078, "pubchem_sid": [ "9186", "841255", "12013925", "46508472", "49855137", "53790103", "57389325", "78512325", "93166585", "103167825", "103920666", "134982169", "135651250", "160644679", "160963816", "175444080", "312228161", "318163419", "340514813", "340514814", "340514815", "349991117", "387021470", "387021471", "387082508", "434250264", "438454315", "10512406", "78512324", "250115591", "78512323", "78512326", "78512327", "78512328", "78512329", "78512330" ], "inchikey": "CYQFCXCEBYINGO-IAGOWNOFSA-N", "generic_name": "Dronabinol", "trade_name": "Marinol", "designated_date": "1991-01-15", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of anorexia associated with weight loss in patients with AIDS.", "marketing_approval_date": "1992-12-22", "exclusivity_end_date": "1999-12-22", "sponsor": "Unimed Pharmaceuticals, Inc.|901 Sawyer Rd.|Marietta|Georgia|30062|United States", "orphan_designation": { "original_text": "For the stimulation of appetite and prevention of weight loss in patients with a confirmed diagnosis of AIDS.", "umls": [ "C0001175", "C4084774" ], "parsed_text": [ "AIDS", "Weight loss" ] } }, { "pubchem_cid": 24066, "pubchem_sid": [ "9416", "595891", "7980904", "11528334", "46386814", "46507879", "50613509", "53788261", "87322635", "87559804", "92711292", "103223943", "103914689", "104034365", "126631015", "126657446", "134987108", "144076133", "160964282", "163621070", "163686393", "178101530", "196110180", "252220297", "252451126", "254761771", "255370809", "273937038", "310281807", "312228515", "312343912", "312695583", "315446592", "316434515", "316968015", "318154519", "318162635", "318241159", "319079282", "319553721", "347912866", "348283552", "349291419", "349996132", "354284139", "355039772", "363603141", "373439604", 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"orphan_designation": { "original_text": "Treatment of AIDS.", "umls": "C0001175", "parsed_text": "AIDS" } }, { "pubchem_sid": 348287571, "generic_name": "Poloxamer 331", "designated_date": "1991-03-21", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "CytRx Corporation|154 Technology Parkway|Norcross|Georgia|30092|United States", "orphan_designation": { "original_text": "Initial therapy of toxoplasmosis in patients with AIDS.", "umls": [ "C0001175", "C0040558" ], "parsed_text": [ "AIDS", "Toxoplasmosis" ] } }, { "pubchem_sid": 336269933, "generic_name": "Poloxamer 331", "designated_date": "1991-03-21", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "CytRx Corporation|154 Technology Parkway|Norcross|Georgia|30092|United States", "orphan_designation": { "original_text": "Initial therapy of toxoplasmosis in patients with AIDS.", "umls": [ "C0001175", "C0040558" ], 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Box 9627|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Pneumocystis carinii pneumonia in AIDS patients.", "umls": "C1535939", "parsed_text": "Pneumocystis carinii Pneumonia" } }, { "pubchem_cid": 8813, "pubchem_sid": [ "8149601", "10321798", "17388789", "48423906", "48425509", "53787921", "103914283", "126669892", "134974925", "135668169", "136379450", "143491827", "144204293", "144208920", "144210632", "152236064", "160687798", "170464786", "223392205", "249827981", "252157151", "252402526", "312693848", "313081839", "315674290", "318047717", "318154989", "319067190", "335984969", "340510183", "348397430", "348746280", "354283867", "355037933", "375384449", "375659686", "375760842", "376067435", "376220139", "376227098", "382155582", "384430728", "385647940", "386484346", "387178309", "387208835", "433779598", "434132548", "434303078", "438473035", "438485389", "440066257", "441028512", "49877520", "160964081", "252626039", 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"designated_date": "1984-02-28", "designation_status": "Designated/Approved", "marketing_approval_date": "1984-10-16", "exclusivity_end_date": "1991-10-16", "sponsor": "Fujisawa USA, Inc.|3 Parkway North Center|Deerfield|Illinois|60015|United States", "orphan_designation": { "original_text": "Treatment of Pneumocystis carinii pneumonia.", "umls": "C1535939", "parsed_text": "Pneumocystis carinii Pneumonia" } }, { "pubchem_cid": 6135, "pubchem_sid": [ "50042261", "103217286", "134971807", "252359401", "312695316", "313077478", "319064151", "322070063", "341142553", "341835082", "355038269", "374016076", "375134235", "385755091", "386591750", "404617438", "434131759", "434304436", "438472941", "438486966", "440066247", "441049121", "50042258", "53790596", "251884683", "310279223", "318162092", "319063172", "431058", "8137165", "254752626", "319219810", "354283930", "196105780", "255370546", "319406568", "441071451" ], "inchikey": "GJOHLWZHWQUKAU-UHFFFAOYSA-N", "generic_name": "Primaquine 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"parsed_text": [ "HIV", "toxoplasma gondii encephalitis" ] } }, { "pubchem_cid": 5215, "pubchem_sid": [ "9860", "92611", "602777", "855872", "7980699", "8149533", "10321441", "10532663", "11533903", "46506164", "50048769", "53789615", "57288844", "74579279", "85089635", "87560164", "103165644", "103913935", "104052024", "117528268", "124766246", "126631011", "126677933", "134971211", "144075225", "144203911", "160963706", "162177254", "174548883", "175442771", "187072281", "210279416", "210281739", "223704353", "223792891", "250064291", "252220242", "252356874", "252402264", "254761637", "255370654", "268734708", "275385710", "310280373", "312228084", "312695440", "313075600", "313082259", "313102087", "315342170", "315446269", "315680164", "316397509", "316898052", "318153261", "318162308", "319063382", "319450995", "319554947", "336300404", "340510654", "340515804", "341140441", "341832364", "347912887", "348283049", "348397534", "349245925", "349979672", "355038788", "363592680", 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"orphan_designation": { "original_text": "For use in combination with pyrimethamine for the treatment of Toxoplasma gondii encephalitis in patients with and without AIDS.", "umls": [ "C0001175", "C0085315" ], "parsed_text": [ "AIDS", "toxoplasma gondii encephalitis" ] } } ] }, { "_id": "MONDO:0005377", "fda_orphan_drug": [ { "pubchem_cid": 159594, "pubchem_sid": [ "12014999", "50068170", "50253083", "53788137", "53789849", "85335558", "103168476", "135115498", "178100477", "185974011", "224005477", "254760165", "310267108", "318031342", "318152141", "319174780", "319553421", "341150962", "341855294", "348836861", "349995542", "355039931", "375675581", "376229596", "378132298", "381389983", "382157264", "384569071", "385641612", "386477873", "404770146", "406809971", "419500662", "433860438", "441007350", "50253081", "434142196", "438449124", "439431179", "50253085", "405462802", "440009464", "162803143", "438580809", "50253080", "50253082", "50253084", "163620866", "163686195", 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329976960, "generic_name": "rituximab", "trade_name": "Rituxan", "designated_date": "2006-02-14", "designation_status": "Designated/Approved", "approved_labeled_indication": "For the use of Rituxan (rituximab) in combination with glucocorticoids for the treatment of patients with Wegener's Granulomatosis (WG) and Microscopic Polyangiitis (MPA). )", "marketing_approval_date": "2011-04-19", "exclusivity_end_date": "2018-04-19", "sponsor": "Genentech, Inc.|1 DNA Way|South San Francisco|California|94080|United States", "orphan_designation": { "original_text": "Treatment of patients with anti-neutrophil cytoplasmic antibody-associated vasculitis (Wegener's Granulomatosis, Microscopic Polyangiitis, and Churg-Strauss Syndrome)", "umls": [ "C0008728", "C2347126", "C2717865", "C3495801" ], "parsed_text": [ "Churg-Strauss Syndrome", "Microscopic Polyangiitis", "Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis", "Wegener's Granulomatosis" ] } }, { "pubchem_sid": 319478935, "generic_name": "rituximab", "trade_name": "Rituxan", "designated_date": "2006-02-14", "designation_status": "Designated/Approved", "approved_labeled_indication": "For the use of Rituxan (rituximab) in combination with glucocorticoids for the treatment of patients with Wegener's Granulomatosis (WG) and Microscopic Polyangiitis (MPA). )", "marketing_approval_date": "2011-04-19", "exclusivity_end_date": "2018-04-19", "sponsor": "Genentech, Inc.|1 DNA Way|South San Francisco|California|94080|United States", "orphan_designation": { "original_text": "Treatment of patients with anti-neutrophil cytoplasmic antibody-associated vasculitis (Wegener's Granulomatosis, Microscopic Polyangiitis, and Churg-Strauss Syndrome)", "umls": [ "C0008728", "C2347126", "C2717865", "C3495801" ], "parsed_text": [ "Churg-Strauss Syndrome", "Microscopic Polyangiitis", "Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis", "Wegener's Granulomatosis" ] } }, { "pubchem_sid": 319435463, "generic_name": 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"generic_name": "rituximab", "trade_name": "Rituxan", "designated_date": "2006-02-14", "designation_status": "Designated/Approved", "approved_labeled_indication": "RITUXAN, in combination with glucocorticoids, is indicated for the treatment of adult and pediatric patients 2 years of age and older with Granulomatosis with Polyangiitis (GPA) (Wegeners Granulomatosis) and Microscopic Polyangiitis (MPA)", "marketing_approval_date": "2019-09-27", "exclusivity_end_date": "2026-09-27", "sponsor": "Genentech, Inc.|1 DNA Way|South San Francisco|California|94080|United States", "orphan_designation": { "original_text": "Treatment of patients with anti-neutrophil cytoplasmic antibody-associated vasculitis (Wegener's Granulomatosis, Microscopic Polyangiitis, and Churg-Strauss Syndrome)", "umls": [ "C0008728", "C2347126", "C2717865", "C3495801" ], "parsed_text": [ "Churg-Strauss Syndrome", "Microscopic Polyangiitis", "Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis", "Wegener's 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178103553, "generic_name": "romiplostim", "trade_name": "Nplate", "designated_date": "2003-03-27", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenic purpura (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy", "marketing_approval_date": "2008-08-22", "exclusivity_end_date": "2015-08-22", "sponsor": "Amgen, Inc.|One Amgen Center Drive|Mail Stop: 27-2-D|Thousand Oaks|California|91320|United States", "orphan_designation": { "original_text": "Treatment of immune thrombocytopenic purpura", "umls": "C0398650", "parsed_text": "Immune thrombocytopenic purpura" } }, { "pubchem_sid": 135347108, "generic_name": "romiplostim", "trade_name": "Nplate", "designated_date": "2003-03-27", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of thrombocytopenia in patients with chronic immune (idiopathic) 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"Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Biogen IDEC Inc.|3030 Callan Road|San Diego|California|92121|United States", "orphan_designation": { "original_text": "Treatment of immune thrombocytopenic purpura", "umls": "C0398650", "parsed_text": "Immune thrombocytopenic purpura" } }, { "pubchem_sid": 135287061, "generic_name": "toralizumab", "designated_date": "2002-03-14", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Biogen IDEC Inc.|3030 Callan Road|San Diego|California|92121|United States", "orphan_designation": { "original_text": "Treatment of immune thrombocytopenic purpura", "umls": "C0398650", "parsed_text": "Immune thrombocytopenic purpura" } }, { "pubchem_sid": 404720054, "generic_name": "efgartigimod", "designated_date": "2019-02-01", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "argenx BVBA|7 Industriepark-Zwijnaarde|Gent|Vlaanderen|Belgium", "orphan_designation": { "original_text": "Treatment of Primary Immune Thrombocytopenia (ITP)", "umls": [ "C0242584", "C0398650" ], "parsed_text": [ "Immune thrombocytopenia", "Immune Thrombocytopenia" ] } }, { "pubchem_sid": 440962803, "generic_name": "romiplostim", "trade_name": "Nplate", "designated_date": "2003-03-27", "designation_status": "Designated/Approved", "approved_labeled_indication": "Nplate is indicated for the treatment of thrombocytopenia in adult patients with immune thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy", "marketing_approval_date": "2019-10-17", "exclusivity_end_date": "2026-10-17", "exclusivity_protected_indication": "For the treatment of thrombocytopenia in adult patients with non-chronic immune thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.", "sponsor": "Amgen, 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850 Ridgeview Drive|Horsham|Pennsylvania|19044|United States", "orphan_designation": { "original_text": "Treatment of pediatric systemic lupus erythematosus", "umls": "C0024141", "parsed_text": "SYSTEMIC LUPUS ERYTHEMATOSUS" } }, { "pubchem_sid": 354355053, "generic_name": "ustekinumab", "designated_date": "2017-07-18", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Janssen Biotech, Inc.|800 & 850 Ridgeview Drive|Horsham|Pennsylvania|19044|United States", "orphan_designation": { "original_text": "Treatment of pediatric systemic lupus erythematosus", "umls": "C0024141", "parsed_text": "SYSTEMIC LUPUS ERYTHEMATOSUS" } }, { "pubchem_sid": 349905851, "generic_name": "ustekinumab", "designated_date": "2017-07-18", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Janssen Biotech, Inc.|800 & 850 Ridgeview Drive|Horsham|Pennsylvania|19044|United States", "orphan_designation": { "original_text": "Treatment of pediatric systemic lupus erythematosus", "umls": "C0024141", "parsed_text": "SYSTEMIC LUPUS ERYTHEMATOSUS" } }, { "pubchem_sid": 347910190, "generic_name": "ustekinumab", "designated_date": "2017-07-18", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Janssen Biotech, Inc.|800 & 850 Ridgeview Drive|Horsham|Pennsylvania|19044|United States", "orphan_designation": { "original_text": "Treatment of pediatric systemic lupus erythematosus", "umls": "C0024141", "parsed_text": "SYSTEMIC LUPUS ERYTHEMATOSUS" } }, { "pubchem_sid": 178103467, "generic_name": "ustekinumab", "designated_date": "2017-07-18", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Janssen Biotech, Inc.|800 & 850 Ridgeview Drive|Horsham|Pennsylvania|19044|United States", "orphan_designation": { "original_text": "Treatment of pediatric systemic lupus erythematosus", "umls": "C0024141", "parsed_text": "SYSTEMIC LUPUS ERYTHEMATOSUS" } }, { "pubchem_sid": 135302166, "generic_name": "ustekinumab", "designated_date": "2017-07-18", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Janssen Biotech, Inc.|800 & 850 Ridgeview Drive|Horsham|Pennsylvania|19044|United States", "orphan_designation": { "original_text": "Treatment of pediatric systemic lupus erythematosus", "umls": "C0024141", "parsed_text": "SYSTEMIC LUPUS ERYTHEMATOSUS" } }, { "pubchem_sid": 103771533, "generic_name": "ustekinumab", "designated_date": "2017-07-18", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Janssen Biotech, Inc.|800 & 850 Ridgeview Drive|Horsham|Pennsylvania|19044|United States", "orphan_designation": { "original_text": "Treatment of pediatric systemic lupus erythematosus", "umls": "C0024141", "parsed_text": "SYSTEMIC LUPUS ERYTHEMATOSUS" } }, { "pubchem_sid": 387124020, "generic_name": "ustekinumab", "designated_date": "2017-07-18", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Janssen Biotech, Inc.|800 & 850 Ridgeview Drive|Horsham|Pennsylvania|19044|United States", "orphan_designation": { "original_text": "Treatment of pediatric systemic lupus erythematosus", "umls": "C0024141", "parsed_text": "SYSTEMIC LUPUS ERYTHEMATOSUS" } }, { "pubchem_sid": 377017835, "generic_name": "ustekinumab", "designated_date": "2017-07-18", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Janssen Biotech, Inc.|800 & 850 Ridgeview Drive|Horsham|Pennsylvania|19044|United States", "orphan_designation": { "original_text": "Treatment of pediatric systemic lupus erythematosus", "umls": "C0024141", "parsed_text": "SYSTEMIC LUPUS ERYTHEMATOSUS" } }, { "pubchem_sid": 355433663, "generic_name": "ustekinumab", "designated_date": "2017-07-18", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Janssen Biotech, Inc.|800 & 850 Ridgeview Drive|Horsham|Pennsylvania|19044|United States", "orphan_designation": { "original_text": "Treatment of pediatric systemic lupus erythematosus", "umls": "C0024141", "parsed_text": "SYSTEMIC LUPUS ERYTHEMATOSUS" } }, { "pubchem_sid": 349984255, "generic_name": "ustekinumab", "designated_date": "2017-07-18", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Janssen Biotech, Inc.|800 & 850 Ridgeview Drive|Horsham|Pennsylvania|19044|United States", "orphan_designation": { "original_text": "Treatment of pediatric systemic lupus erythematosus", "umls": "C0024141", "parsed_text": "SYSTEMIC LUPUS ERYTHEMATOSUS" } } ] }, { "_id": "UMLS:C0009450", "fda_orphan_drug": [ { 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States", "orphan_designation": { "original_text": "Treatment of human immunodeficiency virus type 1 (HIV-1) infection in pediatric patients", "umls": [ "C0009450", "C0019704" ], "parsed_text": [ "Infection", "Human immunodeficiency virus type 1" ] } }, { "pubchem_cid": 148195, "pubchem_sid": [ "12015147", "46514799", "50067857", "53730874", "53790412", "103241637", "126671481", "127339627", "127339628", "127339629", "135105633", "135707678", "136895225", "152258621", "160647456", "162205081", "162222422", "164194111", "170558848", "186007024", "249565704", "252215710", "254760273", "313063239", "315733338", "317223374", "318036053", "319166073", "319432848", "319553157", "340503162", "341137411", "341164067", "346615757", "348273634", "348870946", "349994481", "350081075", "355040341", "367157087", "375577903", "375590818", "375690478", "376026885", "377311050", "378132290", "381971989", "382157821", "384416331", "384568925", "384573017", "385781960", "386618694", "387022706", "404770253", "406528206", "406803198", "419530284", "433805307", "436380257", "438476528", "440703208", "46514773", "53730876", "242060144", "363739616", "365266864", "405467290", "434316057", "438493685", "440013952", "53730872", "53730873", "53730875", "53730877", "53730878", "348267901" ], "inchikey": "DHMTURDWPRKSOA-RUZDIDTESA-N", "generic_name": "lonafarnib", "designated_date": "2013-11-19", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Eiger Biopharmaceuticals, Inc.|PO Box 430|San Carlos|California|94070|United States", "orphan_designation": { "original_text": "Treatment of Hepatitis Delta Virus (HDV)infection", "umls": [ "C0009450", "C0011220" ], "parsed_text": [ "Infection", "Hepatitis Delta Virus" ] } }, { "pubchem_cid": 73303, "pubchem_sid": [ "78931074", "85335672", "103596434", "126665586", "135030316", "144206061", "170465085", "174527594", "175437740", "210279313", "210281636", "241037251", "251915669", "251917019", "251963975", "251970995", "252215304", "252348782", "254759935", "310264862", "310272409", "312234357", "313076713", "313080773", "313083753", "315444826", "315682141", "316966708", "318153492", "318163449", "319112878", "319446371", "319554808", "322093455", "329873002", "336350734", "341141896", "341837732", "348029781", "348296757", "348781757", "349977997", "355038874", "376229601", "377018299", "381375682", "381939462", "382156201", "385157813", "385270194", "385782278", "386619023", "387071905", "404639718", "406860487", "419501778", "438792494", "439432570", "440791111", "78931071", "162183561", "163121746", "204375223", "211535313", "354268943", "126624291", "126655541", "196110410", "312624545", "316898583", "355047659", "387071904", "405456419", "440003081", "375636439", "384570850", "438600749", "12014776", "336267758", "78931072", "78931073", "123088800", "251895481", "342545488", "368494561", "375587311", "441073273" ], "inchikey": "AVAACINZEOAHHE-VFZPANTDSA-N", "generic_name": "Doripenem", "designated_date": "2004-07-16", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Shionogi, Inc.|300 Campus Drive|Suite 300|Florham Park|New Jersey|7932|United States", "orphan_designation": { "original_text": "Treatment of bronchopulmonary infection in patients with cystic fibrosis who are colonized with Pseudomonas aeruginosa or Burkholderia cepacia.", "umls": [ "C0009450", "C0010674" ], "parsed_text": [ "Infection", "Cystic Fibrosis" ] } }, { "pubchem_cid": 25151504, "pubchem_sid": [ "135021019", "152258234", "160647070", "160697744", "160962910", "164045125", "187051836", "194945367", "252215178", "252451833", "255343979", "273990879", "310264999", "312245190", "315703898", "317223667", "318163547", "319382728", "319481599", "319552755", "329874033", "340588701", "341141419", "347737422", "348225368", "348274036", "349977168", "354270321", "354326186", "374074849", "374386884", "375577912", "375587783", "375640269", "378132283", "381419325", "383416043", "385632635", "386623783", "387183307", "419906595", "433834581", "434316080", "438493706", "441048599", "254759265", "255495996", "342411126", "384275900", "386194460", "387183308", "319433780" ], "inchikey": "ZCIGNRJZKPOIKD-CQXVEOKZSA-N", "generic_name": "cobicistat", "trade_name": "Tybost", "designated_date": "2017-06-15", "designation_status": "Designated/Approved", "approved_labeled_indication": "TYBOST is a CYP3A inhibitor indicated to increase systemic exposure of atazanavir in combination with other antiretroviral agents in the treatment of HIV-1 infection in pediatric patients weighing at least 35 kg", "marketing_approval_date": "2019-08-22", "exclusivity_end_date": "2026-08-22", "exclusivity_protected_indication": "Indicated to increase systemic exposure of atazanavir in combination with other antiretroviral agents in the treatment of HIV-1 infection in pediatric patients weighing at least 35 kg.", "sponsor": "Gilead Sciences, Inc.|333 Lakeside Drive|Foster City|California|94404|United States", "orphan_designation": { "original_text": "Treatment of human immunodeficiency virus type 1 (HIV-1) infection in pediatric patients", "umls": [ "C0009450", "C0019704" ], "parsed_text": [ "Infection", "Human immunodeficiency virus type 1" ] } }, { "pubchem_cid": 57004, "pubchem_sid": [ "103675549", "162177030", "251915896", "251917244", "374035884", "384231567", "385109725", "126645773", "135015827", "319222156", "196106720", "318160939", "318239183", "349995060", "349258398" ], "inchikey": "VKDGNNYJFSHYKD-UHFFFAOYSA-N", "generic_name": "Eflornithine HCl", "trade_name": "Ornidyl", "designated_date": "1986-04-23", "designation_status": "Designated/Approved", "marketing_approval_date": "1990-11-28", "exclusivity_end_date": "1997-11-28", "sponsor": "Hoechst Marion Roussel|P.O. Box 9627|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Trypanosoma brucei gambiense infection (sleeping sickness).", "umls": [ "C0009450", "C0041218", "C0041228" ], "parsed_text": [ "Infection", "Trypanosoma brucei gambiense", "Sleeping sickness" ] } }, { "pubchem_sid": 441072525, "generic_name": "adalimumab", "trade_name": "Humira", "designated_date": "2014-05-13", "designation_status": "Designated/Approved", "approved_labeled_indication": "Indicated for the treatment of non-infectious intermediate, posterior and panuveitis in adult patients", "marketing_approval_date": "2016-06-30", "exclusivity_end_date": "2023-06-30", "exclusivity_protected_indication": "Indicated for the treatment of non-infectious intermediate, posterior and panuveitis in adult patients", "sponsor": "AbbVie, Inc.|1 North Waukegan Road|North Chicago|Illinois|60064|United States", "orphan_designation": { "original_text": "Treatment of non-infectious intermediate, posterior, or panuveitis, or chronic non-infectious anterior uveitis", "umls": [ "C0009450", "C0030343", "C0339317", "C0751438" ], "parsed_text": [ "Infectious", "Panuveitis", "Non-infectious anterior uveitis", "Posterior" ] } }, { "pubchem_sid": 440790468, "generic_name": "adalimumab", "trade_name": "Humira", "designated_date": "2014-05-13", "designation_status": "Designated/Approved", "approved_labeled_indication": "Indicated for the treatment of non-infectious intermediate, posterior and panuveitis in adult patients", "marketing_approval_date": "2016-06-30", "exclusivity_end_date": "2023-06-30", "exclusivity_protected_indication": "Indicated for the treatment of non-infectious intermediate, posterior and panuveitis in adult patients", "sponsor": "AbbVie, Inc.|1 North Waukegan Road|North Chicago|Illinois|60064|United States", "orphan_designation": { "original_text": "Treatment of non-infectious intermediate, posterior, or panuveitis, or chronic non-infectious anterior uveitis", "umls": [ "C0009450", "C0030343", "C0339317", "C0751438" ], "parsed_text": [ "Infectious", "Panuveitis", "Non-infectious anterior uveitis", "Posterior" ] } }, { "pubchem_sid": 387061214, "generic_name": "adalimumab", "trade_name": "Humira", "designated_date": "2014-05-13", "designation_status": "Designated/Approved", "approved_labeled_indication": "Indicated for the treatment of non-infectious intermediate, posterior and panuveitis in adult patients", "marketing_approval_date": "2016-06-30", "exclusivity_end_date": "2023-06-30", "exclusivity_protected_indication": "Indicated for the treatment of non-infectious intermediate, posterior and panuveitis in adult patients", "sponsor": "AbbVie, Inc.|1 North Waukegan Road|North Chicago|Illinois|60064|United States", "orphan_designation": { "original_text": "Treatment of non-infectious intermediate, posterior, or panuveitis, or chronic non-infectious anterior uveitis", "umls": [ "C0009450", "C0030343", "C0339317", "C0751438" ], "parsed_text": [ 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Arques Avenue|Sunnyvale|California|94085|United States", "orphan_designation": { "original_text": "Treatment of nodal marginal zone lymphoma", "umls": "C0242647", "parsed_text": "Nodal marginal zone lymphoma" } }, { "pubchem_cid": 72950888, "pubchem_sid": [ "310264697", "312321733", "312322062", "336270090", "355037279", "374322969", "375564119", "375580906", "382371124", "384569377", "404772434", "434315067", "438493878", "440702694" ], "inchikey": "IUVCFHHAEHNCFT-INIZCTEOSA-N", "generic_name": "umbralisib", "trade_name": "Ukoniq", "designated_date": "2019-04-11", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of adult patients with relapsed or refractory marginal zone lymphoma (MZL) who have received at least one prior anti-CD20-based regimen", "marketing_approval_date": "2021-02-05", "sponsor": "TG Therapeutics, Inc.|2 Gansevoort Street|9th Floor|New York|New York|10014|United States", "orphan_designation": { "original_text": "Treatment of nodal marginal zone lymphoma", "umls": "C0242647", "parsed_text": "Nodal marginal zone lymphoma" } }, { "pubchem_cid": 86677874, "pubchem_sid": [ "348351247", "355193653", "375588996", "375589278", "376241122", "381120174", "381974236", "404613106", "404719128", "404772313", "405043662", "406847986", "434136627", "438443733", "381118808", "382146170" ], "inchikey": "ZQPDJCIXJHUERQ-QWRGUYRKSA-N", "generic_name": "parsaclisib", "designated_date": "2019-10-07", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Incyte Corporation|1801 Augustine Cut-Off|Wilmington|Delaware|19803|United States", "orphan_designation": { "original_text": "Treatment of nodal marginal zone lymphoma", "umls": "C0242647", "parsed_text": "Nodal marginal zone lymphoma" } }, { "pubchem_cid": 135565596, "pubchem_sid": [ "152258695", "160647529", "163643093", "223366204", "242613212", "252155314", "255343066", "315706346", "317223391", "318698080", "319381987", "319436538", "319468181", "329878768", "336425826", "340575831", "341189834", "346405739", "347828721", "354279017", "355040286", "375980344", "375989707", "383521005", "384568782", "384572793", "385645351", "386481706", "387141778", "387256501", "401963061", "405131996", "440799029", "440804529", "440810951", "335987030", "375758803" ], "inchikey": "MWYDSXOGIBMAET-UHFFFAOYSA-N", "generic_name": "copanlisib", "designated_date": "2017-02-07", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bayer HealthCare Pharmaceuticals, Inc.|100 Bayer Boulevard|P. O. Box 915|Hanover|New Jersey|7981|United States", "orphan_designation": { "original_text": "Treatment of nodal marginal zone lymphoma", "umls": "C0242647", "parsed_text": "Nodal marginal zone lymphoma" } }, { "pubchem_sid": 381129647, "generic_name": "axicabtagene ciloleucel", "designated_date": "2020-03-24", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Kite Pharma, Inc.|2400 Broadway|Santa Monica|California|90404|United States", "orphan_designation": { "original_text": "Treatment of nodal marginal zone lymphoma", "umls": "C0242647", "parsed_text": "Nodal marginal zone lymphoma" } }, { "pubchem_sid": 374364294, "generic_name": "axicabtagene ciloleucel", "designated_date": "2020-03-24", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Kite Pharma, Inc.|2400 Broadway|Santa Monica|California|90404|United States", "orphan_designation": { "original_text": "Treatment of 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"67088", "11528256", "85165104", "103176121", "134972781", "318692182", "348275425", "386264197", "143627254", "143631876", "241035464", "241055249", "241084981", "252237271", "348276321", "348276836", "348282721", "348282722" ], "inchikey": "WPYMKLBDIGXBTP-UHFFFAOYSA-M", "generic_name": "benzoate", "designated_date": "2012-07-06", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "SyneuRx International (Taiwan) Corp.|1380 S. Marengo Ave|Pasadena|California|91106|United States", "orphan_designation": { "original_text": "Treatment of pediatric schizophrenia", "umls": "C0036341", "parsed_text": "Schizophrenia" } }, { "pubchem_cid": 644019, "pubchem_sid": [ "9781", "12012585", "57309735", "57390495", "80649036", "103460177", "104014242", "104152845", "113635231", "134990571", "135697550", "160711726", "162224688", "178100971", "224092533", "242059005", "250138861", "252159978", "274950707", "275388672", "319243269", "340472111", "341824828", "347759096", "347827820", "348741391", "349990645", "368250367", "381028723", "381118550", "384446666", "384571298", "385785099", "386187095", "386187097", "386622023", "387143314", "404647975", "419505161", "434144021", "438450697", "438790203", "439641511", "50044344", "74382081", "80649034", "312216657", "341142865", "387143313", "80649035", "250092611", "382155556", "387143312", "8150126", "80649037", "250137329", "366203546", "375590317", "385858634", "386187096", "387155362", "441079652" ], "inchikey": "QHMBSVQNZZTUGM-ZWKOTPCHSA-N", "generic_name": "cannabidiol", "designated_date": "2014-11-17", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Benuvia Therapeutics Inc.|444 South Ellis Street|Chandler|Alaska|85224|United States", "orphan_designation": { "original_text": "Treatment of pediatric schizophrenia (pediatrics is defined as 0 through 16 years of age)", "umls": "C0036341", "parsed_text": "Schizophrenia" } } ] }, { "_id": "UMLS:C1568868", "fda_orphan_drug": [ { "pubchem_cid": 65464, "pubchem_sid": [ "10321428", "49665028", "49982454", "50086597", "50995000", "56320699", "56322635", "85273707", "92721963", "103913922", "126627469", "135022789", "144075986", "144203971", "160677222", "160809947", "160871060", "163088302", "170465576", "187072125", "225087149", "251901033", "312693734", "313077035", "315681132", "316538308", "318034467", "319106091", "332861090", "332875487", "340515338", "341149518", "346419063", "347914006", "348273944", "348698123", "355037984", "355148466", "374180712", "375105336", "375651014", "384417171", "385756753", "386593413", "387021847", "387100034", "405416526", "406548682", "433779742", "434132229", "434143576", "438449432", "439442462", "440064268", "441005735", "76715658", "268735211", "318163355", "93618276", "441072189" ], "inchikey": "HNNIWKQLJSNAEQ-UHFFFAOYSA-N", "generic_name": "Benzydamine hydrochloride", "designated_date": "1998-05-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Angelini Pharmaceuticals, Inc.|70 Grand Avenue|River Edge|New Jersey|7661|United States", "orphan_designation": { "original_text": "Prophylactic treatment of oral mucositis resulting from radiation therapy for head and neck cancer.", "umls": [ "C1568868", "C3887461" ], "parsed_text": [ "Oral Mucositis", "Head and Neck Cancer" ] } }, { "pubchem_cid": 121494085, "pubchem_sid": [ "160963711", "178103634", "348283053", "385786988", "386624038", "419590850", "46508862", "53790009", "135002494", "387105325", "440790948", "254761221", "310280346", "347713307", "375666600", "8139948", "52479101", "126657350", "196108173", "376050030", "434132519", "9522", "597363", "7980693", "49866446", "118043405", "126670547", "162214213", "164233862", "174520585", "223441266", "313075819", "313189028", "319479695", "341138406", "342578654", "349112212", "349981023", "354702332", "354702424", "364196654", "374263525", "377351549", "377495226", "381367286", "405490598", "434313113", "438489400", "438596632", "440037256", "441072311" ], "inchikey": "YZPQNHUVIVIUCT-JTJNLBSYSA-F", "generic_name": "Sucralfate", "designated_date": "1993-07-15", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Fuisz Technologies, Ltd.|14555 Avion at Lakeside|Chantilly|Virginia|20151|United States", "orphan_designation": { "original_text": "Treatment of oral mucositis and stomatitis following radiation therapy for head and neck cancer.", "umls": [ "C0038362", "C1568868", "C1578499", "C3887461" ], "parsed_text": [ "Oral mucositis", "Oral Mucositis", "Radiation Therapy", "Head and Neck Cancer" ] } } ] }, { "_id": "UMLS:C3887461", "fda_orphan_drug": [ { "pubchem_cid": 65464, "pubchem_sid": [ "10321428", "49665028", "49982454", "50086597", "50995000", "56320699", "56322635", "85273707", "92721963", "103913922", "126627469", "135022789", "144075986", "144203971", "160677222", "160809947", "160871060", "163088302", "170465576", "187072125", "225087149", "251901033", "312693734", "313077035", "315681132", "316538308", "318034467", "319106091", "332861090", "332875487", "340515338", "341149518", "346419063", "347914006", "348273944", "348698123", "355037984", "355148466", "374180712", "375105336", "375651014", "384417171", "385756753", "386593413", "387021847", "387100034", "405416526", "406548682", "433779742", "434132229", "434143576", "438449432", "439442462", "440064268", "441005735", "76715658", "268735211", "318163355", "93618276", "441072189" ], "inchikey": "HNNIWKQLJSNAEQ-UHFFFAOYSA-N", "generic_name": "Benzydamine hydrochloride", "designated_date": "1998-05-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Angelini Pharmaceuticals, Inc.|70 Grand Avenue|River Edge|New Jersey|7661|United States", "orphan_designation": { "original_text": "Prophylactic treatment of oral mucositis resulting from radiation therapy for head and neck cancer.", "umls": [ "C1568868", "C3887461" ], "parsed_text": [ "Oral Mucositis", "Head and Neck Cancer" ] } }, { "pubchem_cid": 13116, "pubchem_sid": [ "604699", "49873338", "50283989", "81065634", "103589989", "134976633", "164232747", "275390243", "319071304", "341108340", "341161768", "347736225", "349902762", "349991457", "355196175", "363895900", "381354963", "385755440", "386215818", "386592099", "386990718", "404615718", "404770274", "434304116", "438486815", "439574279", "105809", "8138301", "26732566", "50283988", "250108436", "384568512", "409199650", "438584269", "405503622", "440050278", "50283990", "342563259", "441078520" ], "inchikey": "HRHKSTOGXBBQCB-VFWICMBZSA-N", "generic_name": "Porfiromycin", "designated_date": "1995-09-19", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Boehringer Ingelheim Pharmaceuticals, Inc.|900 Ridgebury Road|P. O. Box 368|Ridgefield|Connecticut|6877|United States", "orphan_designation": { "original_text": "Treatment of head and neck cancer.", "umls": "C3887461", "parsed_text": "Head and Neck Cancer" } }, { "pubchem_cid": 121494085, "pubchem_sid": [ "160963711", "178103634", "348283053", "385786988", "386624038", "419590850", "46508862", "53790009", "135002494", "387105325", "440790948", "254761221", "310280346", "347713307", "375666600", "8139948", "52479101", "126657350", "196108173", "376050030", "434132519", "9522", "597363", "7980693", "49866446", "118043405", "126670547", "162214213", "164233862", "174520585", "223441266", "313075819", "313189028", "319479695", "341138406", "342578654", "349112212", "349981023", "354702332", "354702424", "364196654", "374263525", "377351549", "377495226", "381367286", "405490598", "434313113", "438489400", "438596632", "440037256", "441072311" ], "inchikey": "YZPQNHUVIVIUCT-JTJNLBSYSA-F", "generic_name": "Sucralfate", "designated_date": "1993-07-15", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Fuisz Technologies, Ltd.|14555 Avion at Lakeside|Chantilly|Virginia|20151|United States", "orphan_designation": { "original_text": "Treatment of oral mucositis and stomatitis following radiation therapy for head and neck cancer.", "umls": [ "C0038362", "C1568868", "C1578499", "C3887461" ], "parsed_text": [ "Oral mucositis", "Oral Mucositis", "Radiation Therapy", "Head and Neck Cancer" ] } }, { "pubchem_cid": 5910, "pubchem_sid": [ "9677", "605273", "7980323", "26697419", "29204486", "46507475", "48414341", "48416432", "49857637", "53787438", "77139700", "93166375", "103177090", "103921296", "126687572", "134972029", "135650824", "144204438", "160964419", "170464845", "224815847", "241164666", "250133633", "275133813", "312228621", "315672080", "316427341", "318162003", "319063900", "329978364", "341138846", "341842141", "348283676", "349980533", "355195419", "363604817", "381326916", "381988038", "385782264", "386304092", "386619004", "387142344", "404755280", "434123590", "434302985", "438472991", "438486520", "439452255", "440791197", "440818069", "440818370", "441028403", "77139699", "249962147", "363663432", "375561727", "387142342", "387209602", "404755068", "405511172", "440057828", "77139701", "164127567", "77139698", "77139702", "77139703", "85088782", "85088783", "342555163", "438521979", "441076441" ], "inchikey": "QCHFTSOMWOSFHM-WPRPVWTQSA-N", "generic_name": "Pilocarpine", "trade_name": "Salagen", "designated_date": "1990-09-24", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of symptoms of xerostomia from salivary gland hypofunction caused by radiotherapy for cancer of the head and neck.", "marketing_approval_date": "1994-03-22", "exclusivity_end_date": "2001-03-22", "sponsor": "MGI Pharma, Inc.|5775 West Old Shakoppe Rd.|Suite 100|Bloomington|Minnesota|55437|United States", "orphan_designation": { "original_text": "Treatment of xerostomia induced by radiation therapy for head and neck cancer.", "umls": [ "C0043352", "C3887461" ], "parsed_text": [ "Xerostomia", "Head and Neck Cancer" ] } }, { "pubchem_cid": 135413511, "pubchem_sid": [ "12013911", "49891449", "50834069", "53790580", "103235497", "126650417", "134997716", "136946548", "162188998", "162226463", "175426868", "196111634", "210274967", "210280605", "223395390", "223444134", "223929392", "312811311", "318154674", "319085643", "319447966", "319554062", "340502957", "341137129", "341155597", "349984054", "355039510", "375109326", "375586068", "375628604", "376229674", "381380949", "382159337", "382370894", "383542700", "384569214", "384642808", "385783938", "386620795", "387061357", "387135362", "404607945", "405476922", "419503056", "419712679", "434313634", "436380064", "438475121", "438491720", "440023581", "440702646", "441125187", "341838379", "406179311", "275847239", "348942138" ], "inchikey": "ORYDPOVDJJZGHQ-UHFFFAOYSA-N", "generic_name": "tirapazamine", "designated_date": "2002-10-23", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Sanofi-Aventis US, Inc.|11 Great Valley Parkway|P. O. Box 3026|Malvern|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of head and neck cancer", "umls": "C3887461", "parsed_text": "Head and Neck Cancer" } } ] }, { "_id": "UMLS:C0002994", "fda_orphan_drug": [ { "pubchem_cid": 137528262, "pubchem_sid": [ "381608984", "434143224", "438449498", "439592773", "439595373", "440816716", "440201071" ], "inchikey": "UXNXMBYCBRBRFD-MUUNZHRXSA-N", "generic_name": "berotralstat", "trade_name": "ORLADEYO", "designated_date": "2017-11-01", "designation_status": "Designated/Approved", "approved_labeled_indication": "prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years of age and older", "marketing_approval_date": "2020-12-03", "exclusivity_end_date": "2027-12-03", "exclusivity_protected_indication": "prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years of age and older", "sponsor": "BioCryst Pharmaceuticals, 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Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of malignant pleural effusion.", "umls": "C0080032", "parsed_text": "malignant pleural effusion" } }, { "pubchem_cid": 21864427, "pubchem_sid": [ "49894847", "124766402", "135029663", "252215010", "255397894", "275889646", "313080277", "315444224", "316966360", "316966361", "319112310", "363896673", "363900420", "368501101", "376031241", "381349035", "382166173", "383414154", "385269974", "439350520", "439641884", "440062210", "162184456", "211534695", "249827195", "254762079", "310267964", "316542220", "317223545", "340551618", "341137156", "438474283", "136376425", "143498942", "152237903", "174476568", "313076965", "319369694", "319392673", "347912513", "348273431", "375636845", "376191892", "377311335", "406667522", "53788293", "160963638", "348282990", "349981033", "335962824", "375782348", "381938530", "406515932", "434144249", "438450055", "301184", "8137133", "136340222", "144205103", "144207148", "194186158", "250203576", "253659941", "318153077", "319304243", "319406449", "341146935", "342556257", "347248748", "349446807", "384568806", "384572827", "386207149", "441073716" ], "inchikey": "WUIABRMSWOKTOF-UHFFFAOYSA-N", "generic_name": "Bleomycin sulfate", "trade_name": "Blenoxane", "designated_date": "1993-09-17", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of malignant pleural effusion.", "marketing_approval_date": "1996-02-20", "exclusivity_end_date": "2003-02-20", "sponsor": "Bristol-Myers Squibb Pharmaceutical Research Institute|P.O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of malignant pleural effusion.", "umls": "C0080032", "parsed_text": "malignant pleural effusion" } }, { "pubchem_cid": 72466, "pubchem_sid": [ "49894847", "124766402", "135029663", "252215010", "255397894", "275889646", "313080277", "315444224", "316966360", "316966361", "319112310", "363896673", "363900420", "368501101", "376031241", "381349035", "382166173", "383414154", "385269974", "439350520", "439641884", "440062210", "162184456", "211534695", "249827195", "254762079", "310267964", "316542220", "317223545", "340551618", "341137156", "438474283", "136376425", "143498942", "152237903", "174476568", "313076965", "319369694", "319392673", "347912513", "348273431", "375636845", "376191892", "377311335", "406667522", "53788293", "160963638", "348282990", "349981033", "335962824", "375782348", "381938530", "406515932", "434144249", "438450055", "301184", "8137133", "136340222", "144205103", "144207148", "194186158", "250203576", "253659941", "318153077", "319304243", "319406449", "341146935", "342556257", "347248748", "349446807", "384568806", "384572827", "386207149", "441073716" ], "inchikey": "WUIABRMSWOKTOF-OCBSMOPSSA-N", "generic_name": "Bleomycin sulfate", "trade_name": "Blenoxane", "designated_date": "1993-09-17", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of malignant pleural effusion.", "marketing_approval_date": "1996-02-20", "exclusivity_end_date": "2003-02-20", "sponsor": "Bristol-Myers Squibb Pharmaceutical Research Institute|P.O. Box 4000|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of malignant pleural effusion.", "umls": "C0080032", "parsed_text": "malignant pleural effusion" } }, { "pubchem_cid": 54685925, "pubchem_sid": [ "8149431", "10321592", "49992529", "49994862", "87550941", "103770864", "103914082", "124766350", "126624756", "126656016", "135017464", "144075132", "144089161", "144204802", "144206734", "144211907", "160871228", "162228497", "170465049", "172080583", "196106010", "224452896", "251895970", "251912490", "251915470", "252157145", "252314562", "252358693", "252402363", "254759785", "255462050", "275943392", "310277252", "312693779", "312809931", "315680490", "316898446", "318238613", "319399905", "319399906", "341837959", "346441725", "348721762", "354290342", "354326266", "375587759", "375683057", "376220016", "376226386", "377351143", "377397680", "383435674", "384428627", "385660976", "385793129", "386497481", "387060401", "387094709", "433779763", "434132115", "434301358", "438463003", "438473239", "438503704", "438658623", "440063988", "440078634", "46500360", "56463321", "335986964", "341148431", "375758869", "855650", "53788191", "56352994", "92304293", "163564756", "252218679", "268734627", "312344927", "315445573", "316429110", "318161673", "322073931", "126622254", "252391171", "313076870", "316899207", "341137897", "375082507", "50106632", "50111155", "85273720", "104169958", "176251429", "252618505", "252633123", "268738346", "441071143" ], "inchikey": "WTJXVDPDEQKTCV-VQAITOIOSA-N", "generic_name": "Minocycline HCl", "designated_date": "1992-06-19", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Lederle Laboratories Division|Division of American Cyanamid Company|401 N. 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Box 786099|San Antonio|Texas|78278|United States", "orphan_designation": { "original_text": "Prevention of cystine nephrolithiasis in patients with homozygous cystinuria.", "umls": [ "C0010691", "C0022650" ], "parsed_text": [ "Cystinuria", "Nephrolithiasis" ] } }, { "pubchem_cid": 2724354, "pubchem_sid": [ "9800", "46504680", "50048359", "76750784", "81065526", "103771056", "134973780", "135610136", "162221387", "241059553", "255370644", "312236789", "312694458", "315674141", "318162295", "319246991", "341143136", "349975619", "355178528", "355202866", "355203258", "387068439", "402382156", "404613890", "404639074", "434313106", "438489392", "438791036", "440790456", "598336", "48416560", "76750782", "103849901", "117392416", "126670172", "135056651", "160963911", "249958968", "319067694", "348283226", "387068438", "405478573", "407344867", "440025232", "76750783", "204359704", "441073219" ], "inchikey": "ACTRVOBWPAIOHC-XIXRPRMCSA-N", "generic_name": "Succimer", "designated_date": "1990-11-05", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Sanofi Winthrop, Inc.|90 Park Avenue|New York|New York|10016|United States", "orphan_designation": { "original_text": "Prevention of cystine kidney stone formation in patients with homozygous cystinuria who are prone to stone development.", "umls": [ "C0010691", "C0022650" ], "parsed_text": [ "Cystinuria", "KIDNEY STONE" ] } }, { "pubchem_cid": 5483, "pubchem_sid": [ "583265", "610366", "48416637", "49973761", "75941066", "87560971", "92098527", "92714304", "103409496", "103931530", "118043432", "126624247", "126669587", "134981860", "144076409", "144205207", "162175489", "162228427", "170465217", "210279517", "210281840", "223441841", "252357562", "252453765", "254760279", "275844728", "312246002", "312598460", "312995310", "313082398", "315521003", "315674207", "316474599", "318162469", "319555055", "322075098", "332867519", "340510861", "341141763", "341835562", "346431324", "347827801", "348397573", "348481911", "349984426", "355038704", "363896125", "363899885", "374057531", "375082674", "375570227", "375639043", "377310210", "384417046", "384568176", "384571965", "385785006", "386621925", "387181596", "387181597", "404614296", "405467780", "414858523", "434132751", "434313609", "438491650", "438613555", "440014441", "440791057", "441049103", "75941068", "252088950", "381342835", "75941067", "406819142", "342552643", "441079279" ], "inchikey": "YTGJWQPHMWSCST-UHFFFAOYSA-N", "generic_name": "Tiopronin", "trade_name": "Thiola", "designated_date": "1986-01-17", "designation_status": "Designated/Approved", "approved_labeled_indication": "Indicated for the prevention of cystine (kidney) stone formation in patients with severe homozygous cystinuria with urinary cystine greater than 500 mg/day, who are resistant to treatment with conservative measures of high fluid intake, alkali and diet modification or who have adverse reactions to d-penicillamine.", "marketing_approval_date": "1988-08-11", "exclusivity_end_date": "1995-08-11", "sponsor": "Mission Pharmacal Company|P.O. Box 786099|San Antonio|Texas|78278|United States", "orphan_designation": { "original_text": "Prevention of cystine nephrolithiasis in patients with homozygous cystinuria.", "umls": [ "C0010691", "C0022650" ], "parsed_text": [ "Cystinuria", "Nephrolithiasis" ] } }, { "pubchem_cid": 5483, "pubchem_sid": [ "583265", "610366", "48416637", "49973761", "75941066", "87560971", "92098527", "92714304", "103409496", "103931530", "118043432", "126624247", "126669587", "134981860", "144076409", "144205207", "162175489", "162228427", "170465217", "210279517", "210281840", "223441841", "252357562", "252453765", "254760279", "275844728", "312246002", "312598460", "312995310", "313082398", "315521003", "315674207", "316474599", "318162469", "319555055", "322075098", "332867519", "340510861", "341141763", "341835562", "346431324", "347827801", "348397573", "348481911", "349984426", "355038704", "363896125", "363899885", "374057531", "375082674", "375570227", "375639043", "377310210", "384417046", "384568176", "384571965", "385785006", "386621925", "387181596", "387181597", "404614296", "405467780", "414858523", "434132751", "434313609", "438491650", "438613555", "440014441", "440791057", "441049103", "75941068", "252088950", "381342835", "75941067", "406819142", "342552643", "441079279" ], "inchikey": "YTGJWQPHMWSCST-UHFFFAOYSA-N", "generic_name": "Tiopronin", "trade_name": "Thiola", "designated_date": "1986-01-17", "designation_status": "Designated/Approved", "approved_labeled_indication": "THIOLA EC is indicated, in combination with high fluid intake, alkali, and diet modification, for the prevention of cystine stone formation in adults and pediatric patients 20 kg and greater with severe homozygous cystinuria, who are not responsive to these measures alone.", "marketing_approval_date": "2019-06-28", "exclusivity_end_date": "2026-06-28", "sponsor": "Mission Pharmacal Company|P.O. Box 786099|San Antonio|Texas|78278|United States", "orphan_designation": { "original_text": "Prevention of cystine nephrolithiasis in patients with homozygous cystinuria.", "umls": [ "C0010691", "C0022650" ], "parsed_text": [ "Cystinuria", "Nephrolithiasis" ] } } ] }, { "_id": "UMLS:C0241910", "fda_orphan_drug": [ { "pubchem_cid": 5281004, "pubchem_sid": [ "855541", "7978823", "10321783", "11528595", "12013315", "46504869", "53789711", "56394980", "56422168", "92303636", "92721807", "93576669", "103455384", "103914268", "126627527", "126651544", "127278596", "127278597", "127278598", "127278599", "127278600", "127278601", "127278602", "127278603", "127278604", "127278605", "127278606", "127278607", "127278608", "127278609", "127278610", "127278611", "127278612", "127278613", "127278614", "135019928", "136361536", "160871371", "162176915", "162227030", "163564128", "165235840", "170505998", "172080749", "175443769", "178104006", "187072297", "210274891", "210280526", "223827776", 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Inc.|25801 Industrial Boulevard|Suite B|Hayward|California|94545|United States", "orphan_designation": { "original_text": "Treatment of patients with immunoglobulin A nephropathy", "umls": "C0017661", "parsed_text": "Immunoglobulin A Nephropathy" } }, { "pubchem_sid": 160698348, "generic_name": "blisibimod", "designated_date": "2017-08-07", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Anthera Pharmaceuticals, Inc.|25801 Industrial Boulevard|Suite B|Hayward|California|94545|United States", "orphan_designation": { "original_text": "Treatment of patients with immunoglobulin A nephropathy", "umls": "C0017661", "parsed_text": "Immunoglobulin A Nephropathy" } } ] }, { "_id": "UMLS:C0035078", "fda_orphan_drug": [ { "pubchem_cid": 5281004, "pubchem_sid": [ "855541", "7978823", "10321783", "11528595", "12013315", "46504869", "53789711", "56394980", "56422168", "92303636", "92721807", "93576669", "103455384", "103914268", 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Box 300|Raritan|New Jersey|8869|United States", "orphan_designation": { "original_text": "Treatment of anemia associated with end stage renal disease.", "umls": [ "C0002871", "C0022661" ], "parsed_text": [ "Anemia", "End stage renal disease" ] } }, { "pubchem_sid": 49965745, "generic_name": "Epoetin alpha", "designated_date": "1987-08-27", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "R. W. Johnson Pharmaceutical Research Institute|Route 202 South|P.O. Box 300|Raritan|New Jersey|8869|United States", "orphan_designation": { "original_text": "Treatment of anemia associated with end stage renal disease.", "umls": [ "C0002871", "C0022661" ], "parsed_text": [ "Anemia", "End stage renal disease" ] } }, { "pubchem_sid": 46508122, "generic_name": "Erythropoietin (human, recombinant)", "designated_date": "1987-11-19", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Organon Teknika Corporation", "orphan_designation": { "original_text": "Treatment of anemia associated with end stage renal disease.", "umls": [ "C0002871", "C0022661" ], "parsed_text": [ "Anemia", "End stage renal disease" ] } }, { "pubchem_sid": 46508122, "generic_name": "Erythropoietin (recombinant human)", "designated_date": "1987-08-19", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "McDonnell Douglas Corp|P.O. 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"C0751674", "C1832033", "C2247022" ], "parsed_text": [ "Tuberous Sclerosis Complex", "Subependymal Giant Cell Astrocytoma", "Angiomyolipoma", "Lymphangioleiomyomatosis", "Subependymal Giant Cell Astrocytoma", "tuberous sclerosis complex" ] } }, { "pubchem_cid": 5284616, "pubchem_sid": [ "636884", "8145920", "11528741", "12014635", "46505675", "48416541", "56310631", "56310880", "56311583", "56311585", "56311714", "56311827", "56311949", "56311983", "56312137", "56312183", "56312184", "56312185", "56312186", "56312645", "56312887", "56313330", "56313354", "56313375", "56313498", "56313803", "56313974", "56314022", "56314023", "56314562", "57288669", "93167183", "103164548", "103959411", "118043176", "135022432", "144204421", "144206303", "162037398", "178102653", "223860387", "310279616", "312234699", "315674125", "318162259", "354702922", "375577133", "385640004", "386634234", "419506776", "433780360", "53787256", "80132194", "251912147", "251915220", "319297079", "349977797", 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"orphan_designation": { "original_text": "Treatment of tuberous sclerosis complex-related facial angiofibromas", "umls": [ "C0041341", "C0265319" ], "parsed_text": [ "Tuberous Sclerosis Complex", "Facial angiofibromas" ] } }, { "pubchem_cid": 6442177, "pubchem_sid": [ "12014881", "46505248", "50044230", "56311446", "56312241", "103987301", "104178855", "135156085", "136929859", "143493384", "144206063", "152236848", "152258132", "160645716", "160646971", "162189189", "174527790", "178102512", "203355779", "224627271", "254760057", "255345989", "312244639", "312616346", "315678225", "318161060", "319552082", "336851372", "341141968", "341852299", "342465019", "354270236", "374015776", "384431653", "385787399", "386624465", "405068250", "419533960", "433982778", "438477198", "439656880", "53788326", "126644878", "126662810", "251915630", "251916980", "251971409", "272722577", "283537548", "313053725", "313080931", "316538449", "319321081", "319434589", "319446374", "349988787", 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"UMLS:C3898147", "fda_orphan_drug": [ { "pubchem_cid": 644019, "pubchem_sid": [ "9781", "12012585", "57309735", "57390495", "80649036", "103460177", "104014242", "104152845", "113635231", "134990571", "135697550", "160711726", "162224688", "178100971", "224092533", "242059005", "250138861", "252159978", "274950707", "275388672", "319243269", "340472111", "341824828", "347759096", "347827820", "348741391", "349990645", "368250367", "381028723", "381118550", "384446666", "384571298", "385785099", "386187095", "386187097", "386622023", "387143314", "404647975", "419505161", "434144021", "438450697", "438790203", "439641511", "50044344", "74382081", "80649034", "312216657", "341142865", "387143313", "80649035", "250092611", "382155556", "387143312", "8150126", "80649037", "250137329", "366203546", "375590317", "385858634", "386187096", "387155362", "441079652" ], "inchikey": "QHMBSVQNZZTUGM-ZWKOTPCHSA-N", "generic_name": "cannabidiol", "designated_date": "2015-04-22", "designation_status": 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O. Box 9001|Shirley|New York|11967|United States", "orphan_designation": { "original_text": "Treatment of trigeminal neuralgia", "umls": "C0040997", "parsed_text": "Trigeminal Neuralgia" } }, { "pubchem_cid": 44602, "pubchem_sid": [ "135107526", "162255235", "319092574", "347828402", "123055284", "162255234", "348398423" ], "inchikey": "KPYSYYIEGFHWSV-QMMMGPOBSA-N", "generic_name": "L-baclofen", "designated_date": "1998-01-06", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Osmotica Pharmaceutical Corp.|1205 Culbreth Dr.|Suite 200|Wilmington|North Carolina|28405|United States", "orphan_designation": { "original_text": "Treatment of trigeminal neuralgia", "umls": "C0040997", "parsed_text": "Trigeminal Neuralgia" } }, { "pubchem_cid": 44600, "pubchem_sid": [ "135107526", "162255235", "319092574", "347828402", "123055284", "162255234", "348398423" ], "inchikey": "KPYSYYIEGFHWSV-MRVPVSSYSA-N", "generic_name": "L-baclofen", 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Street|Boston|Massachusetts|2118|United States", "orphan_designation": { "original_text": "Prevention of Primary Graft Dysfunction in transplant recipients receiving a lung or heart+ lung transplant.", "umls": "C0948031", "parsed_text": "Primary Graft Dysfunction" } }, { "pubchem_cid": 91864495, "pubchem_sid": [ "252300049", "319422654", "406588874", "434312185", "438490518", "347911287", "404720290" ], "inchikey": "LKVFMOMQYXIFRK-KSVAIKAXSA-N", "generic_name": "solnatide", "designated_date": "2016-02-04", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Apeptico Forschung und Entwicklung GmbH|c/o mingo bueros|Vienna|Austria", "orphan_designation": { "original_text": "Treatment of primary graft dysfunction following lung transplantation", "umls": "C0948031", "parsed_text": "Primary Graft Dysfunction" } } ] }, { "_id": "MONDO:0005109", "fda_orphan_drug": [ { "pubchem_cid": 135538685, "pubchem_sid": [ "487164", "596004", "50070430", "85612722", "103463438", "126671531", "135030850", "162257817", "162309227", "162778968", "312245708", "316966438", "319105742", "319112104", "336349842", "355053181", "252552948", "310269337", "319224352", "341110792", "348603136", "363900594", "76966740", "76966741", "76966742", "341171124", "438566558" ], "inchikey": "XSSYCIGJYCVRRK-NKWVEPMBSA-N", "generic_name": "Carbovir", "designated_date": "1989-12-13", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Glaxo Wellcome Inc.|5 Moore Drive|Research Triangle Park|North Carolina|27709|United States", "orphan_designation": { "original_text": "Treatment of persons with AIDS and in patients with symptomatic HIV infection and a CD4 count less than 200/mm3.", "umls": [ "C0001175", "C0019693" ], "parsed_text": [ "AIDS", "HIV infection" ] } }, { "pubchem_cid": 135433604, "pubchem_sid": [ "487164", "596004", "50070430", "85612722", "103463438", "126671531", 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Box 16529|Columbus|Ohio|43216|United States", "orphan_designation": { "original_text": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infection.", "umls": [ "C0019693", "C0026914" ], "parsed_text": [ "HIV infection", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 137295261, "pubchem_sid": [ "9444", "596006", "7890228", "7980515", "12012943", "17388744", "26697101", "46506468", "48425598", "50044776", "56323065", "57288827", "103914671", "126592920", "126628917", "135011172", "144204563", "144207956", "173905832", "223937742", "241131852", "250133567", "252220205", "252356474", "254761728", "310279728", "312228274", "315446114", "318154425", "318162198", "318166547", "341141592", "341835220", "347713867", "347759842", "385642307", "386478581", "387060858", "419536186", "438475565", "53787673", "75049157", "92717216", "103535182", "124766242", "126684731", "172080843", "251916303", "251917650", "275071157", "313082072", "315677114", "319311857", "319461145", "349979339", "381340159", "381358021", "382164364", "384255218", "384995394", "385453584", "440790604", "152258736", "160647581", "162170553", "164233446", "312695542", "319552594", "347914222", "377309763", "434305011", "438488046", "162224750", "348283269", "354265537", "375677640", "26719829", "49681785", "75049153", "75049154", "163137856", "172080344", "313074772", "347235005", "383548025", "387071533", "387071536", "387071537", "405500216", "440046872", "104170218", "185990407", "336348462", "346441003", "355050038", "377624929", "381183566", "384568084", "384571840", "46386607", "56463170", "75049155", "75049156", "117480934", "312650100", "318040690", "319433074", "342565542", "349275687", "354283700", "363671366", "375201744", "375562339", "375573268", "384585868", "434122534", "441079331" ], "inchikey": "ZWBTYMGEBZUQTK-HBZSWILLSA-N", "generic_name": "Rifabutin", "trade_name": "Mycobutin", "designated_date": "1989-12-18", "designation_status": "Designated/Approved", "approved_labeled_indication": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infections.", "marketing_approval_date": "1992-12-23", "exclusivity_end_date": "1999-12-23", "sponsor": "Adria Laboratories, Inc.|P.O. Box 16529|Columbus|Ohio|43216|United States", "orphan_designation": { "original_text": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infection.", "umls": [ "C0019693", "C0026914" ], "parsed_text": [ "HIV infection", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135925740, "pubchem_sid": [ "9444", "596006", "7890228", "7980515", "12012943", "17388744", "26697101", "46506468", "48425598", "50044776", "56323065", "57288827", "103914671", "126592920", "126628917", "135011172", "144204563", "144207956", "173905832", "223937742", "241131852", "250133567", "252220205", "252356474", "254761728", "310279728", "312228274", "315446114", "318154425", "318162198", "318166547", "341141592", "341835220", "347713867", "347759842", "385642307", "386478581", "387060858", "419536186", "438475565", "53787673", "75049157", "92717216", "103535182", "124766242", "126684731", "172080843", "251916303", "251917650", "275071157", "313082072", "315677114", "319311857", "319461145", "349979339", "381340159", "381358021", "382164364", "384255218", "384995394", "385453584", "440790604", "152258736", "160647581", "162170553", "164233446", "312695542", "319552594", "347914222", "377309763", "434305011", "438488046", "162224750", "348283269", "354265537", "375677640", "26719829", "49681785", "75049153", "75049154", "163137856", "172080344", "313074772", "347235005", "383548025", "387071533", "387071536", "387071537", "405500216", "440046872", "104170218", "185990407", "336348462", "346441003", "355050038", "377624929", "381183566", "384568084", "384571840", "46386607", "56463170", "75049155", "75049156", "117480934", "312650100", "318040690", "319433074", "342565542", "349275687", "354283700", "363671366", "375201744", "375562339", "375573268", "384585868", "434122534", "441079331" ], "inchikey": "ZWBTYMGEBZUQTK-DNEPJJHDSA-N", "generic_name": "Rifabutin", "trade_name": "Mycobutin", "designated_date": "1989-12-18", "designation_status": "Designated/Approved", "approved_labeled_indication": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infections.", "marketing_approval_date": "1992-12-23", "exclusivity_end_date": "1999-12-23", "sponsor": "Adria Laboratories, Inc.|P.O. Box 16529|Columbus|Ohio|43216|United States", "orphan_designation": { "original_text": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infection.", "umls": [ "C0019693", "C0026914" ], "parsed_text": [ "HIV infection", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 136257950, "pubchem_sid": [ "9444", "596006", "7890228", "7980515", "12012943", "17388744", "26697101", "46506468", "48425598", "50044776", "56323065", "57288827", "103914671", "126592920", "126628917", "135011172", "144204563", "144207956", "173905832", "223937742", "241131852", "250133567", "252220205", "252356474", "254761728", "310279728", "312228274", "315446114", "318154425", "318162198", "318166547", "341141592", "341835220", "347713867", "347759842", "385642307", "386478581", "387060858", "419536186", "438475565", "53787673", "75049157", "92717216", "103535182", "124766242", "126684731", "172080843", "251916303", "251917650", "275071157", "313082072", "315677114", "319311857", "319461145", "349979339", "381340159", "381358021", "382164364", "384255218", "384995394", "385453584", "440790604", "152258736", "160647581", "162170553", "164233446", "312695542", "319552594", "347914222", "377309763", "434305011", "438488046", "162224750", "348283269", "354265537", "375677640", "26719829", "49681785", "75049153", "75049154", "163137856", "172080344", "313074772", "347235005", "383548025", "387071533", "387071536", "387071537", "405500216", "440046872", "104170218", "185990407", "336348462", "346441003", "355050038", "377624929", "381183566", "384568084", "384571840", "46386607", "56463170", "75049155", "75049156", "117480934", "312650100", "318040690", "319433074", "342565542", "349275687", "354283700", "363671366", "375201744", "375562339", "375573268", "384585868", "434122534", "441079331" ], "inchikey": "ZWBTYMGEBZUQTK-OZKAIBRQSA-N", "generic_name": "Rifabutin", "trade_name": "Mycobutin", "designated_date": "1989-12-18", "designation_status": "Designated/Approved", "approved_labeled_indication": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infections.", "marketing_approval_date": "1992-12-23", "exclusivity_end_date": "1999-12-23", "sponsor": "Adria Laboratories, Inc.|P.O. Box 16529|Columbus|Ohio|43216|United States", "orphan_designation": { "original_text": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infection.", "umls": [ "C0019693", "C0026914" ], "parsed_text": [ "HIV infection", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 136053407, "pubchem_sid": [ "9444", "596006", "7890228", "7980515", "12012943", "17388744", "26697101", "46506468", "48425598", "50044776", "56323065", "57288827", "103914671", "126592920", "126628917", "135011172", "144204563", "144207956", "173905832", "223937742", "241131852", "250133567", "252220205", "252356474", "254761728", "310279728", "312228274", "315446114", "318154425", "318162198", "318166547", "341141592", "341835220", "347713867", "347759842", "385642307", "386478581", "387060858", "419536186", "438475565", "53787673", "75049157", "92717216", "103535182", "124766242", "126684731", "172080843", "251916303", "251917650", "275071157", "313082072", "315677114", "319311857", "319461145", "349979339", "381340159", "381358021", "382164364", "384255218", "384995394", "385453584", "440790604", "152258736", "160647581", "162170553", "164233446", "312695542", "319552594", "347914222", "377309763", "434305011", "438488046", "162224750", "348283269", "354265537", "375677640", "26719829", "49681785", "75049153", "75049154", "163137856", "172080344", "313074772", "347235005", "383548025", "387071533", "387071536", "387071537", "405500216", "440046872", "104170218", "185990407", "336348462", "346441003", "355050038", "377624929", "381183566", "384568084", "384571840", "46386607", "56463170", "75049155", "75049156", "117480934", "312650100", "318040690", "319433074", "342565542", "349275687", "354283700", "363671366", "375201744", "375562339", "375573268", "384585868", "434122534", "441079331" ], "inchikey": "ZWBTYMGEBZUQTK-WOYMMMRKSA-N", "generic_name": "Rifabutin", "trade_name": "Mycobutin", "designated_date": "1989-12-18", "designation_status": "Designated/Approved", "approved_labeled_indication": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infections.", "marketing_approval_date": "1992-12-23", "exclusivity_end_date": "1999-12-23", "sponsor": "Adria Laboratories, Inc.|P.O. Box 16529|Columbus|Ohio|43216|United States", "orphan_designation": { "original_text": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infection.", "umls": [ "C0019693", "C0026914" ], "parsed_text": [ "HIV infection", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135458645, "pubchem_sid": [ "9444", "596006", "7890228", "7980515", "12012943", "17388744", "26697101", "46506468", "48425598", "50044776", "56323065", "57288827", "103914671", "126592920", "126628917", "135011172", "144204563", "144207956", "173905832", "223937742", "241131852", "250133567", "252220205", "252356474", "254761728", "310279728", "312228274", "315446114", "318154425", "318162198", "318166547", "341141592", "341835220", "347713867", "347759842", "385642307", "386478581", "387060858", "419536186", "438475565", "53787673", "75049157", "92717216", "103535182", "124766242", "126684731", "172080843", "251916303", "251917650", "275071157", "313082072", "315677114", "319311857", "319461145", "349979339", "381340159", "381358021", "382164364", "384255218", "384995394", "385453584", "440790604", "152258736", "160647581", "162170553", "164233446", "312695542", "319552594", "347914222", "377309763", "434305011", "438488046", "162224750", "348283269", "354265537", "375677640", "26719829", "49681785", "75049153", "75049154", "163137856", "172080344", "313074772", "347235005", "383548025", "387071533", "387071536", "387071537", "405500216", "440046872", "104170218", "185990407", "336348462", "346441003", "355050038", "377624929", "381183566", "384568084", "384571840", "46386607", "56463170", "75049155", "75049156", "117480934", "312650100", "318040690", "319433074", "342565542", "349275687", "354283700", "363671366", "375201744", "375562339", "375573268", "384585868", "434122534", "441079331" ], "inchikey": "ZWBTYMGEBZUQTK-OZFKZPMASA-N", "generic_name": "Rifabutin", "trade_name": "Mycobutin", "designated_date": "1989-12-18", "designation_status": "Designated/Approved", "approved_labeled_indication": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infections.", "marketing_approval_date": "1992-12-23", "exclusivity_end_date": "1999-12-23", "sponsor": "Adria Laboratories, Inc.|P.O. Box 16529|Columbus|Ohio|43216|United States", "orphan_designation": { "original_text": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infection.", "umls": [ "C0019693", "C0026914" ], "parsed_text": [ "HIV infection", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 136117120, "pubchem_sid": [ "9444", "596006", "7890228", "7980515", "12012943", "17388744", "26697101", "46506468", "48425598", "50044776", "56323065", "57288827", "103914671", "126592920", "126628917", "135011172", "144204563", "144207956", "173905832", "223937742", "241131852", "250133567", "252220205", "252356474", "254761728", "310279728", "312228274", "315446114", "318154425", "318162198", "318166547", "341141592", "341835220", "347713867", "347759842", "385642307", "386478581", "387060858", "419536186", "438475565", "53787673", "75049157", "92717216", "103535182", "124766242", "126684731", "172080843", "251916303", "251917650", "275071157", "313082072", "315677114", "319311857", "319461145", "349979339", "381340159", "381358021", "382164364", "384255218", "384995394", "385453584", "440790604", "152258736", "160647581", "162170553", "164233446", "312695542", "319552594", "347914222", "377309763", "434305011", "438488046", "162224750", "348283269", "354265537", "375677640", "26719829", "49681785", "75049153", "75049154", "163137856", "172080344", "313074772", "347235005", "383548025", "387071533", "387071536", "387071537", "405500216", "440046872", "104170218", "185990407", "336348462", "346441003", "355050038", "377624929", "381183566", "384568084", "384571840", "46386607", "56463170", "75049155", "75049156", "117480934", "312650100", "318040690", "319433074", "342565542", "349275687", "354283700", "363671366", "375201744", "375562339", "375573268", "384585868", "434122534", "441079331" ], "inchikey": "ZWBTYMGEBZUQTK-AMWSOCTHSA-N", "generic_name": "Rifabutin", "trade_name": "Mycobutin", "designated_date": "1989-12-18", "designation_status": "Designated/Approved", "approved_labeled_indication": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infections.", "marketing_approval_date": "1992-12-23", "exclusivity_end_date": "1999-12-23", "sponsor": "Adria Laboratories, Inc.|P.O. Box 16529|Columbus|Ohio|43216|United States", "orphan_designation": { "original_text": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infection.", "umls": [ "C0019693", "C0026914" ], "parsed_text": [ "HIV infection", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135950975, "pubchem_sid": [ "9444", "596006", "7890228", "7980515", "12012943", "17388744", "26697101", "46506468", "48425598", "50044776", "56323065", "57288827", "103914671", "126592920", "126628917", "135011172", "144204563", "144207956", "173905832", "223937742", "241131852", "250133567", "252220205", "252356474", "254761728", "310279728", "312228274", "315446114", "318154425", "318162198", "318166547", "341141592", "341835220", "347713867", "347759842", "385642307", "386478581", "387060858", "419536186", "438475565", "53787673", "75049157", "92717216", "103535182", "124766242", "126684731", "172080843", "251916303", "251917650", "275071157", "313082072", "315677114", "319311857", "319461145", "349979339", "381340159", "381358021", "382164364", "384255218", "384995394", "385453584", "440790604", "152258736", "160647581", "162170553", "164233446", "312695542", "319552594", "347914222", "377309763", "434305011", "438488046", "162224750", "348283269", "354265537", "375677640", "26719829", "49681785", "75049153", "75049154", "163137856", "172080344", "313074772", "347235005", "383548025", "387071533", "387071536", "387071537", "405500216", "440046872", "104170218", "185990407", "336348462", "346441003", "355050038", "377624929", "381183566", "384568084", "384571840", "46386607", "56463170", "75049155", "75049156", "117480934", "312650100", "318040690", "319433074", "342565542", "349275687", "354283700", "363671366", "375201744", "375562339", "375573268", "384585868", "434122534", "441079331" ], "inchikey": "ZWBTYMGEBZUQTK-ASBZJICESA-N", "generic_name": "Rifabutin", "trade_name": "Mycobutin", "designated_date": "1989-12-18", "designation_status": "Designated/Approved", "approved_labeled_indication": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infections.", "marketing_approval_date": "1992-12-23", "exclusivity_end_date": "1999-12-23", "sponsor": "Adria Laboratories, Inc.|P.O. Box 16529|Columbus|Ohio|43216|United States", "orphan_designation": { "original_text": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infection.", "umls": [ "C0019693", "C0026914" ], "parsed_text": [ "HIV infection", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 135398743, "pubchem_sid": [ "9444", "596006", "7890228", "7980515", "12012943", "17388744", "26697101", "46506468", "48425598", "50044776", "56323065", "57288827", "103914671", "126592920", "126628917", "135011172", "144204563", "144207956", "173905832", "223937742", "241131852", "250133567", "252220205", "252356474", "254761728", "310279728", "312228274", "315446114", "318154425", "318162198", "318166547", "341141592", "341835220", "347713867", "347759842", "385642307", "386478581", "387060858", "419536186", "438475565", "53787673", "75049157", "92717216", "103535182", "124766242", "126684731", "172080843", "251916303", "251917650", "275071157", "313082072", "315677114", "319311857", "319461145", "349979339", "381340159", "381358021", "382164364", "384255218", "384995394", "385453584", "440790604", "152258736", "160647581", "162170553", "164233446", "312695542", "319552594", "347914222", "377309763", "434305011", "438488046", "162224750", "348283269", "354265537", "375677640", "26719829", "49681785", "75049153", "75049154", "163137856", "172080344", "313074772", "347235005", "383548025", "387071533", "387071536", "387071537", "405500216", "440046872", "104170218", "185990407", "336348462", "346441003", "355050038", "377624929", "381183566", "384568084", "384571840", "46386607", "56463170", "75049155", "75049156", "117480934", "312650100", "318040690", "319433074", "342565542", "349275687", "354283700", "363671366", "375201744", "375562339", "375573268", "384585868", "434122534", "441079331" ], "inchikey": "ZWBTYMGEBZUQTK-PVLSIAFMSA-N", "generic_name": "Rifabutin", "trade_name": "Mycobutin", "designated_date": "1989-12-18", "designation_status": "Designated/Approved", "approved_labeled_indication": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infections.", "marketing_approval_date": "1992-12-23", "exclusivity_end_date": "1999-12-23", "sponsor": "Adria Laboratories, Inc.|P.O. Box 16529|Columbus|Ohio|43216|United States", "orphan_designation": { "original_text": "Prevention of disseminated Mycobacterium avium complex disease in patients with advanced HIV infection.", "umls": [ "C0019693", "C0026914" ], "parsed_text": [ "HIV infection", "Mycobacterium avium Complex" ] } }, { "pubchem_cid": 464205, "pubchem_sid": [ "610434", "7980754", "12014981", "48424314", "92719253", "103169192", "126628295", "126655803", "127339001", "127339002", "135078595", "135606020", "135611180", "135682533", "135698226", "160963648", "162793588", "163813066", "187072518", "196111452", "210275003", "210280641", "223729777", "223775493", "249853011", "251889257", "251916125", "251917473", "252157439", "252216393", "252315350", "252358860", "253660231", "253667655", "255370678", "255392225", "274233527", "277309547", "312228042", "312616366", "313082310", "315446322", "315678010", "318152919", "318162392", "318242180", "319226732", "319554363", "329858884", "329979164", "336302080", 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"Epogen", "designated_date": "1991-07-01", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of AZT-induced anemia in HIV infected patients.", "marketing_approval_date": "1990-12-31", "exclusivity_end_date": "1997-12-31", "sponsor": "Amgen, Inc.|1840 Dehavilland Drive|Thousand Oaks|California|91320|United States", "orphan_designation": { "original_text": "Treatment of anemia associated with HIV infection or HIV treatment.", "umls": [ "C0002871", "C0019693" ], "parsed_text": [ "Anemia", "HIV infection" ] } }, { "pubchem_sid": 319423538, "generic_name": "Epoetin alpha", "designated_date": "1989-03-07", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "R. W. Johnson Pharmaceutical Research Institute|Route 202, P.O. Box 300|Raritan|New Jersey|8869|United States", "orphan_designation": { "original_text": "Treatment of HIV associated anemia related to HIV infection or HIV treatment.", "umls": [ "C0002871", "C0019693" ], "parsed_text": [ "Anemia", "HIV infection" ] } }, { "pubchem_sid": 135310353, "generic_name": "Epoetin alpha", "designated_date": "1989-03-07", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "R. W. Johnson Pharmaceutical Research Institute|Route 202, P.O. Box 300|Raritan|New Jersey|8869|United States", "orphan_designation": { "original_text": "Treatment of HIV associated anemia related to HIV infection or HIV treatment.", "umls": [ "C0002871", "C0019693" ], "parsed_text": [ "Anemia", "HIV infection" ] } }, { "pubchem_sid": 49965745, "generic_name": "Epoetin alpha", "designated_date": "1989-03-07", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "R. W. Johnson Pharmaceutical Research Institute|Route 202, P.O. 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"approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "ArTara Therapeutics|1 Little West 12th Street|Suite 254|New York|New York|10014|United States", "orphan_designation": { "original_text": "Treatment of choline deficiency, specifically the choline deficiency, hepatic steatosis, and cholestasis, associated with long-term parenteral nutrition.", "umls": [ "C0008370", "C0008412", "C2711227" ], "parsed_text": [ "Cholestasis", "Choline Deficiency", "Hepatic steatosis" ] } } ] }, { "_id": "UMLS:C0278791", "fda_orphan_drug": [ { "pubchem_cid": 30751, "pubchem_sid": [ "12012906", "48416019", "50027359", "103749729", "135058141", "135610126", "144075932", "160964407", "162178899", "174530365", "211535474", "223377444", "224054755", "249494129", "249866367", "251915628", "251916978", "252215430", "252358909", "252450752", "254761786", "255472200", "268735169", "274227925", "275953720", "277309441", "307080163", "310273098", "315673680", "317223781", "318020277", "318161125", "319083797", "319447921", "341833001", "348283667", "348354958", "354267977", "354284067", "355040077", "374033106", "375082731", "375442135", "375633654", "376145560", "376191960", "377310001", "383424932", "384416990", "384568405", "384572265", "385645793", "386482159", "387094233", "401961173", "404623229", "419582604", "433780712", "433836882", "434132208", "434251889", "436380339", "438456655", "438473752", "439643517", "440064552", "441005508", "455602", "126661936", "223702400", "250035185", "312672596", "313077929", "315445009", "316966872", "316966873", "340503253", "340536120", "375744673", "387094234", "433966349", "164216432", "255374631", "336300883", "346436339", "126625358", "126656559", "196105929", "316898322", "225066673", "250225058", "319436743", "329979665", "341101105", "375583185", "441079628", "441108784" ], "inchikey": "GIUYCYHIANZCFB-FJFJXFQQSA-N", "generic_name": "Fludarabine phosphate", "trade_name": "Fludara", "designated_date": "1989-04-18", "designation_status": "Designated/Approved", "marketing_approval_date": "1991-04-18", "exclusivity_end_date": "1998-04-18", "sponsor": "Berlex Laboratories, Inc.|15049 San Pablo Avenue, P.O. Box 4099|Richmond|California|94804|United States", "orphan_designation": { "original_text": "Treatment of chronic lymphocytic leukemia (CLL), including refractory CLL.", "umls": [ "C0023434", "C0278791" ], "parsed_text": [ "Chronic Lymphocytic Leukemia", "Refractory CLL" ] } } ] }, { "_id": "UMLS:C0270862", "fda_orphan_drug": [ { "pubchem_cid": 941361, "pubchem_sid": [ "7979238", "46507129", "50011452", "56365814", "85335402", "92711460", "103199419", "103928970", "126630830", "126657320", "127339817", "127339818", "127339819", "127339820", "127339821", "135000533", "144204376", "160967817", "170466037", "196108126", "250076484", "254778757", "275134410", "312228904", "313077192", "315681348", "318161135", "318240064", "319245148", "341140969", "347757569", "348284012", "349099302", "349979336", "355168581", "363666141", "375202943", "375652090", "384571720", "385781658", "386618374", "387065083", "387154176", "404616925", "404762158", "406519052", "433778801", "439358146", 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poisoning, or for use in suspected ethylene glycol ingestion.", "marketing_approval_date": "1997-12-04", "exclusivity_end_date": "2004-12-04", "sponsor": "Par Pharmaceuticals, Inc|1 Ram Ridge Road|Chestnut Ridge|New York|10977|United States", "orphan_designation": { "original_text": "Treatment of methanol or ethylene glycol poisoning.", "umls": "C0413194", "parsed_text": "Ethylene glycol poisoning" } } ] }, { "_id": "MONDO:0010735", "fda_orphan_drug": [ { "pubchem_cid": 5282199, "pubchem_sid": [ "586041", "12013741", "49997810", "53789401", "134988128", "162248733", "223441546", "312248578", "319296336", "329799745", "341147559", "347917933", "375574828", "382371232", "125307458", "318162396", "319393496", "347633400", "160808783", "163087813", "319063503", "254793068", "438477060", "319379157", "348287179" ], "inchikey": "HUCXKZBETONXFO-NJFMWZAGSA-N", "generic_name": "geranylgeranylacetone", "designated_date": "2020-06-26", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "RNR BioMedical Inc.|c/o Noa Avtalion|161 Jefferson Av|Tenefly|New Jersey|7670|United States", "orphan_designation": { "original_text": "Treatment of spinal and bulbar muscular atrophy", "umls": "C1839259", "parsed_text": "Spinal and Bulbar Muscular Atrophy" } } ] }, { "_id": "MONDO:0004946", "fda_orphan_drug": [ { "pubchem_cid": 16132283, "pubchem_sid": [ "53786928", "135231356", "135264525", "136349029", "162248084", "319557606", "341138497", "347660503", "348824618", "381970791", "387122248", "406524844", "435379280", "439496892", "439648397", "7979419", "187072139", "318162672", "341833559", "354732289", "103201090", "135651778", "224175503", "404858123", "85086321", "162224805", "255370177", "341139785", "354701767", "375744195", "4670", "46504732", "49972360", "103995578", "126674561", "160730932", "160769873", "160769884", "162248356", "251915678", "251917028", "319884269", "319885216", "341128983", "342556472", "347736994", 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"Treatment of hypoglycemia in patients with congenital hyperinsulinism (CH)", "umls": [ "C0020615", "C3888018" ], "parsed_text": [ "Hypoglycemia", "Congenital Hyperinsulinism" ] } }, { "pubchem_cid": 129012199, "pubchem_sid": [ "135264411", "135652151", "7979925", "50069637", "319360911", "319423166" ], "inchikey": "WSEVKKHALHSUMB-RYVRVIGHSA-N", "generic_name": "exendin-(9-39)", "designated_date": "2011-06-01", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "The Children's Hospital of Philadelphia|Children's Hospital of Phil. Research Institute|Abramson Research Center|Philadelphia|Pennsylvania|19104|United States", "orphan_designation": { "original_text": "Treatment of congential hyperinsulinenic hypoglycemia and other causes of hyperinsulinemic hypoglycemia in adults and children", "umls": [ "C0020615", "C3888018" ], "parsed_text": [ "Hypoglycemia", "Hyperinsulinemic Hypoglycemia" ] } } ] }, { "_id": "UMLS:C3888018", "fda_orphan_drug": [ { "pubchem_cid": 16132283, "pubchem_sid": [ "53786928", "135231356", "135264525", "136349029", "162248084", "319557606", "341138497", "347660503", "348824618", "381970791", "387122248", "406524844", "435379280", "439496892", "439648397", "7979419", "187072139", "318162672", "341833559", "354732289", "103201090", "135651778", "224175503", "404858123", "85086321", "162224805", "255370177", "341139785", "354701767", "375744195", "4670", "46504732", "49972360", "103995578", "126674561", "160730932", "160769873", "160769884", "162248356", "251915678", "251917028", "319884269", "319885216", "341128983", "342556472", "347736994", "363743236", "364230626", "404608072", "404839523", "441071562" ], "inchikey": "MASNOZXLGMXCHN-ZLPAWPGGSA-N", "generic_name": "glucagon", "designated_date": "2012-12-05", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Biodel, Inc.|100 Saw Mill Road|Danbury|Connecticut|6810|United States", "orphan_designation": { "original_text": "Prevention of hypoglycemia in the congenital hyperinsulinism population", "umls": [ "C0020615", "C3888018" ], "parsed_text": [ "Hypoglycemia", "Congenital Hyperinsulinism" ] } }, { "pubchem_cid": 126961379, "pubchem_sid": [ "336270091", "384569750", "381129638", "404719602", "404772659" ], "inchikey": "RZRMFQMNPDPAIX-AJTOSFMRSA-N", "generic_name": "dasiglucagon", "designated_date": "2017-08-10", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Zealand Pharma A/S|36 Smedeland|Glostrup|Denmark", "orphan_designation": { "original_text": "Treatment of hypoglycemia in patients with congenital hyperinsulinism (CH)", "umls": [ "C0020615", "C3888018" ], "parsed_text": [ "Hypoglycemia", "Congenital Hyperinsulinism" ] } }, { "pubchem_cid": 129012199, "pubchem_sid": [ "135264411", "135652151", "7979925", "50069637", "319360911", "319423166" ], "inchikey": "WSEVKKHALHSUMB-RYVRVIGHSA-N", "generic_name": "exendin-(9-39)", "designated_date": "2011-06-01", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "The Children's Hospital of Philadelphia|Children's Hospital of Phil. Research Institute|Abramson Research Center|Philadelphia|Pennsylvania|19104|United States", "orphan_designation": { "original_text": "Treatment of congential hyperinsulinenic hypoglycemia and other causes of hyperinsulinemic hypoglycemia in adults and children", "umls": [ "C0020615", "C3888018" ], "parsed_text": [ "Hypoglycemia", "Hyperinsulinemic Hypoglycemia" ] } } ] }, { "_id": "MONDO:0011612", "fda_orphan_drug": [ { "pubchem_cid": 61156, "pubchem_sid": [ "68914", "49856649", "78552082", "126689513", "135018160", "162188225", "162232352", "249975579", "254781271", "316964091", "318032397", "319102815", "341842762", "348284959", "349167929", "354270667", "374383800", "375110719", "383425547", "387060073", "387099169", "439437800", "441005680", "162269573", "204371687", "377879340" ], "inchikey": "HIZCTWCPHWUPFU-UHFFFAOYSA-N", "generic_name": "glyceryl tribenzoate", "designated_date": "2019-10-15", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Curyx Bio, Inc.|140 E. Ridgewood Avenue|Suite 415 South Tower|Paramus|New Jersey|7652|United States", "orphan_designation": { "original_text": "Treatment of nonketotic hyperglycinemia", "umls": "C0751748", "parsed_text": "Nonketotic Hyperglycinemia" } } ] }, { "_id": "MONDO:0005065", "fda_orphan_drug": [ { "pubchem_cid": 123133491, "pubchem_sid": 375892279, "inchikey": "GKMPTIBZVGZJDN-RJLNLWLESA-N", "generic_name": "H-(D)p-Benzoylphenylalanyl-(D)seryl-(D)tryptophanyl-(D)seryl-(D)pentafluorophenylalanyl-(D)cyclohexylalanyl-(D)arginyl-(D)arginyl-(D)arginyl-(D)glutaminyl-(D)arginyl-(D)arginine acetate salt", "designated_date": "2011-12-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "CanBas Company, Ltd.|2-2-1 Otemachi|Numazu City|Shizuoka|Japan", "orphan_designation": { "original_text": "For use in combination with cisplatin and pemetrexed for the treatment of patients with mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_cid": 5311, "pubchem_sid": [ "12015019", "26684150", "46508989", "46519184", "48426290", "50070170", "53788318", "56311712", "56312417", "56312644", "56314569", "92719823", "103174146", "124766849", "126591282", "126628079", "126649066", "131407858", "135110440", "144076182", "152258134", "160646973", "160871381", "160965729", "162011444", "162172156", "163835411", "164041944", "174560137", "178103458", "186018766", "196106493", "201758964", "223378690", "224182523", "241037723", "250134080", "251902646", "251916065", "251917413", "252158246", "252216571", "252448457", "252451337", "252543486", "254759958", "255344352", "255372419", "310281733", "312228891", "312616221", "312807988", "313075666", "313082574", "313089213", "316968004", "316972335", "318163432", "318239007", "319063436", "319451437", "319552553", "322092071", "329991738", "340521874", "346424792", "347760354", "348273796", "348283996", "349986195", "355040745", "368270137", "374030964", "375303003", "375577674", "375581398", "375638679", "376127707", "376229562", "376968025", "378132914", "381393621", "383527874", "384421795", "385641265", "386635532", "387167917", "419487788", "434131342", "434315444", "438493801", "438645259", "440770292", "440790976", "76096576", "211536333", "221672823" ], "inchikey": "WAEXFXRVDQXREF-UHFFFAOYSA-N", "generic_name": "vorinostat", "designated_date": "2004-03-17", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Merck & Co., Inc.|P. O. Box 2000, RY 32-605|Rahway|New Jersey|7065|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_cid": 135423438, "pubchem_sid": [ "99431587", "103654502", "135267499", "152258852", "160647702", "178102559", "223471427", "249738349", "249807126", "251970985", "252090009", "255344081", "277286638", "310265007", "312616403", "318163907", "319339205", "319434165", "319539392", "340521879", "341092884", "341143400", "349988262", "350079274", "350080446", "350095295", "354330903", "355040172", "375577117", "375630398", "378126786", "383524001", "384568630", "384572576", "385645764", "386482130", "387177921", "387177922", "387237164", "402420917", "405045943", "405225436", "405496926", "419592141", "433794666", "434315123", "438493298", "439666458", "440043584", "440681567", "440794461", "440801773", "440802662" ], "inchikey": "CPMDPSXJELVGJG-UHFFFAOYSA-N", "generic_name": "Nintedanib", "designated_date": "2016-12-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Boehringer Ingelheim Pharmaceuticals, Inc.|900 Ridgebury Road|P. O. Box 368|Ridgefield|Connecticut|6877|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_cid": 66558664, "pubchem_sid": [ "178103590", "210274978", "210280616", "249820378", "252160839", "312387361", "312616229", "319407144", "341200180", "347829042", "355039502", "375578110", "378138548", "384569100", "385653587", "386490045", "387207321", "433835407", "434311605", "438490560", "440702114", "440809566", "332853780", "332853781" ], "inchikey": "NSQSAUGJQHDYNO-UHFFFAOYSA-N", "generic_name": "tazemetostat", "designated_date": "2017-09-28", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Epizyme Inc.|400 Technology Square|4th Floor|Cambridge|Massachusetts|2139|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 347911395, "generic_name": "amatuximab", "designated_date": "2012-09-28", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Eisai Inc.|155 Tice Boulevard|Woodcliff Lake|New Jersey|7677|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 223366083, "generic_name": "amatuximab", "designated_date": "2012-09-28", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Eisai Inc.|155 Tice Boulevard|Woodcliff Lake|New Jersey|7677|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 160704149, "generic_name": "amatuximab", "designated_date": "2012-09-28", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Eisai Inc.|155 Tice Boulevard|Woodcliff Lake|New Jersey|7677|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 135289484, "generic_name": "amatuximab", "designated_date": "2012-09-28", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Eisai Inc.|155 Tice Boulevard|Woodcliff Lake|New Jersey|7677|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 441072526, "generic_name": "bevacizumab", "designated_date": "2016-01-27", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genentech, Inc.|1 DNA Way|South San Francisco|California|94080|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 440790697, "generic_name": "bevacizumab", "designated_date": "2016-01-27", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genentech, Inc.|1 DNA Way|South San Francisco|California|94080|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 387061165, "generic_name": "bevacizumab", "designated_date": "2016-01-27", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genentech, Inc.|1 DNA Way|South San Francisco|California|94080|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 350080043, "generic_name": "bevacizumab", "designated_date": "2016-01-27", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genentech, Inc.|1 DNA Way|South San Francisco|California|94080|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 348480209, "generic_name": "bevacizumab", "designated_date": "2016-01-27", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genentech, Inc.|1 DNA Way|South San Francisco|California|94080|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 329976976, "generic_name": "bevacizumab", "designated_date": "2016-01-27", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genentech, Inc.|1 DNA Way|South San Francisco|California|94080|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 178103377, "generic_name": "bevacizumab", "designated_date": "2016-01-27", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genentech, Inc.|1 DNA Way|South San Francisco|California|94080|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 135329020, "generic_name": "bevacizumab", "designated_date": "2016-01-27", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genentech, Inc.|1 DNA Way|South San Francisco|California|94080|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 103771315, "generic_name": "bevacizumab", "designated_date": "2016-01-27", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genentech, Inc.|1 DNA Way|South San Francisco|California|94080|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 50067187, "generic_name": "bevacizumab", "designated_date": "2016-01-27", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genentech, Inc.|1 DNA Way|South San Francisco|California|94080|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 46504473, "generic_name": "bevacizumab", "designated_date": "2016-01-27", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genentech, Inc.|1 DNA Way|South San Francisco|California|94080|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 404770335, "generic_name": "pegargiminase", "designated_date": "2014-07-21", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Polaris Group|Polaris Pharmaceuticals, Inc.|9373 Town Centre Dr., Suite 150|San Diego|California|92121|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma.", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 381127242, "generic_name": "pegargiminase", "designated_date": "2014-07-21", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Polaris Group|Polaris Pharmaceuticals, Inc.|9373 Town Centre Dr., Suite 150|San Diego|California|92121|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma.", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 194189083, "generic_name": "pegargiminase", "designated_date": "2014-07-21", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Polaris Group|Polaris Pharmaceuticals, Inc.|9373 Town Centre Dr., Suite 150|San Diego|California|92121|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma.", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 387061103, "generic_name": "nivolumab", "designated_date": "2017-08-16", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 385327549, "generic_name": "nivolumab", "designated_date": "2017-08-16", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 384647092, "generic_name": "nivolumab", "designated_date": "2017-08-16", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 354355034, "generic_name": "nivolumab", "designated_date": "2017-08-16", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 349494907, "generic_name": "nivolumab", "designated_date": "2017-08-16", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 347910393, "generic_name": "nivolumab", "designated_date": "2017-08-16", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 178103907, "generic_name": "nivolumab", "designated_date": "2017-08-16", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 160698453, "generic_name": "nivolumab", "designated_date": "2017-08-16", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } }, { "pubchem_sid": 135341610, "generic_name": "nivolumab", "designated_date": "2017-08-16", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Company|P. O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of mesothelioma", "umls": "C0025500", "parsed_text": "Mesothelioma" } } ] }, { "_id": "MONDO:0019165", "fda_orphan_drug": [ { "pubchem_cid": 126970648, "pubchem_sid": [ "50070238", "103770492", "251916712", "251917951", "277306853", "315736978", "341851362", "404621956", "439519455", "440791142", "135216749", "135610763", "312811545", "318043516", "318162669", "340470708", "385779701", "386616394", "340531560", "375749211", "386192214", "386331821", "405472875", "440019535", "7849177" ], "inchikey": "DGTVQJOTEDTFMR-VYFXDUNUSA-N", "generic_name": "Histrelin acetate", "trade_name": "Supprelin Injection", "designated_date": "1988-08-10", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of central precocious puberty.", "marketing_approval_date": "1991-12-24", "exclusivity_end_date": "1998-12-24", "sponsor": "Roberts Pharmaceutical Corp.|Meridian Center III|6 Industrial Way West|Eatontown|New Jersey|7724|United States", "orphan_designation": { "original_text": "Treatment of central precocious puberty.", "umls": "C0342543", "parsed_text": "Central Precocious Puberty" } }, { "pubchem_cid": 56927879, "pubchem_sid": [ "50070238", "103770492", "251916712", "251917951", "277306853", "315736978", "341851362", "404621956", "439519455", "440791142", "135216749", "135610763", "312811545", "318043516", "318162669", "340470708", "385779701", "386616394", "340531560", "375749211", "386192214", "386331821", "405472875", "440019535", "7849177" ], "inchikey": "BKEMVGVBBDMHKL-VYFXDUNUSA-N", "generic_name": "Histrelin acetate", "trade_name": "Supprelin Injection", "designated_date": "1988-08-10", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of central precocious puberty.", "marketing_approval_date": "1991-12-24", "exclusivity_end_date": "1998-12-24", "sponsor": "Roberts Pharmaceutical Corp.|Meridian Center III|6 Industrial Way West|Eatontown|New Jersey|7724|United States", "orphan_designation": { "original_text": "Treatment of central precocious puberty.", "umls": "C0342543", "parsed_text": "Central Precocious Puberty" } }, { "pubchem_cid": 25077993, "pubchem_sid": [ "50070238", "103770492", "251916712", "251917951", "277306853", "315736978", "341851362", "404621956", "439519455", "440791142", "135216749", "135610763", "312811545", "318043516", "318162669", "340470708", "385779701", "386616394", "340531560", "375749211", "386192214", "386331821", "405472875", "440019535", "7849177" ], "inchikey": "HHXHVIJIIXKSOE-QILQGKCVSA-N", "generic_name": "Histrelin acetate", "trade_name": "Supprelin Injection", "designated_date": "1988-08-10", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of central precocious puberty.", "marketing_approval_date": "1991-12-24", "exclusivity_end_date": "1998-12-24", "sponsor": "Roberts Pharmaceutical Corp.|Meridian Center III|6 Industrial Way West|Eatontown|New Jersey|7724|United States", "orphan_designation": { "original_text": "Treatment of central precocious puberty.", "umls": "C0342543", "parsed_text": "Central Precocious Puberty" } }, { "pubchem_cid": 25084151, "pubchem_sid": [ "50070238", "103770492", "251916712", "251917951", "277306853", "315736978", "341851362", "404621956", "439519455", "440791142", "135216749", "135610763", "312811545", "318043516", "318162669", "340470708", "385779701", "386616394", "340531560", "375749211", "386192214", "386331821", "405472875", "440019535", "7849177" ], "inchikey": "OWAUGAMNPZHEOJ-YKZVIGSYSA-N", "generic_name": "Histrelin acetate", "trade_name": "Supprelin Injection", "designated_date": "1988-08-10", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of central precocious puberty.", "marketing_approval_date": "1991-12-24", "exclusivity_end_date": "1998-12-24", "sponsor": "Roberts Pharmaceutical Corp.|Meridian Center III|6 Industrial Way West|Eatontown|New Jersey|7724|United States", "orphan_designation": { "original_text": "Treatment of central precocious puberty.", "umls": "C0342543", "parsed_text": "Central Precocious Puberty" } }, { "pubchem_cid": 657180, "pubchem_sid": [ "855512", "12013727", "53788272", "103770758", "135037705", "135610124", "223808170", "252358946", "252391197", "253659667", "254783965", "310275771", "315684154", "318044302", "319243517", "322079971", "341101974", "341149796", "341838353", "341851172", "349288158", "355176881", "375082765", "375654235", "381357327", "382371244", "384430395", "385661393", "386497899", "401961141", "402382123", "404638784", "438473471", "91148371", "312627837", "313103036", "319222177", "349978356", "354701759", "375744203", "382164531", "387195706", "387208480", "164228180", "252218577", "316967106", "316967107", "319413436", "11528673", "50065359", "318161437", "319432014", "374917907" ], "inchikey": "RGLRXNKKBLIBQS-XNHQSDQCSA-N", "generic_name": "Leuprolide acetate", "trade_name": "Lupron Injection", "designated_date": "1988-07-25", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of children with central precocious puberty", "marketing_approval_date": "1993-04-16", "exclusivity_end_date": "2000-04-16", "sponsor": "Tap Pharmaceuticals, Inc.|2355 Waukegan Road|Deerfield|Illinois|60015|United States", "orphan_designation": { "original_text": "Treatment of central precocious puberty", "umls": "C0342543", "parsed_text": "Central Precocious Puberty" } }, { "pubchem_cid": 25077993, "pubchem_sid": [ "50070238", "103770492", "251916712", "251917951", "277306853", "315736978", "341851362", "404621956", "439519455", "440791142", "135216749", "135610763", "312811545", "318043516", "318162669", "340470708", "385779701", "386616394", "340531560", "375749211", "386192214", "386331821", "405472875", "440019535", "7849177" ], "inchikey": "HHXHVIJIIXKSOE-QILQGKCVSA-N", "generic_name": 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Box 14910|Research Triangle Park|North Carolina|27709|United States", "orphan_designation": { "original_text": "Treatment of congenital ichthyosis", "umls": "C0079154", "parsed_text": "congenital ichthyosis" } }, { "pubchem_cid": 11518241, "pubchem_sid": [ "163642844", "315797970", "318464550", "319346441", "347828982", "349980450", "354341730", "355431751", "363670448", "363670533", "374883849", "375934824", "376241188", "377491545", "385728048", "386564667", "387123099", "404617000", "406849552", "406868527", "433924815", "434314646", "438492173", "439942440", "441079397" ], "inchikey": "MFBCDACCJCDGBA-UHFFFAOYSA-N", "generic_name": "trifarotene", "designated_date": "2014-06-06", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Mayne Pharma LLC.|1240 Sugg Parkway|Greenville|North Carolina|27834|United States", "orphan_designation": { "original_text": "Treatment of congenital ichthyosis", "umls": "C0079154", "parsed_text": 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"241114360", "241114382", "241121948", "241121951", "241133586", "241175826", "316963173", "364206196", "438563570", "441075050" ], "inchikey": "XSQUKJJJFZCRTK-UHFFFAOYSA-N", "generic_name": "urea", "designated_date": "2011-11-07", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Orenova Group, LLC|10 New King Street|Suite 106|White Plains|New York|10604|United States", "orphan_designation": { "original_text": "Treatment of rare congenital ichthyoses (CHILD syndrome, collodion baby, congenital ichthyosform erythroderma, Conradi-flunermann, epidermolytic hyperkeratosis, erythrokeratoderma variabilis, harlequin ichthyosis, KID syndrome, lamellar ichthyosis, Netherton syndrome, neutral lipid storage disease, Sjorgren-Larsson syndrome, trichothiodystrophy, X-linked ichthyosis).", "umls": [ "C0011606", "C0079153", "C0079154", "C0079588", "C0239849", "C0265267", "C0265962", "C0265962", "C0268238", "C1955934", "C3543867", "C3665333" ], "parsed_text": [ "Erythroderma", "Epidermolytic hyperkeratosis", "Lamellar ichthyosis", "X-linked ichthyosis", "Harlequin Ichthyosis", "Child syndrome", "Netherton Syndrome", "Netherton Syndrome", "Neutral lipid storage disease", "Trichothiodystrophy", "Collodion Baby", "Kid syndrome" ] } }, { "pubchem_cid": 5282379, "pubchem_sid": [ "3719647", "7979663", "10321605", "12013352", "46508729", "47193696", "48416136", "50044403", "53788737", "56312436", "56312437", "56312464", "56313471", "56313644", "56352887", "57288466", "57288699", "92303284", "103176635", "103914095", "103940100", "104170133", "127310152", "127310153", "127310154", "134985730", "135727087", "144075449", "162175755", "172080758", "210275182", "210280822", "223365935", "223739818", "251915655", "251917005", "252158884", "252220066", "252359484", "252391227", "252448653", "254752146", "312694748", "313081197", "315445280", "316530980", "316967059", "318161396", "319296424", "319553155", "341833909", "348276850", 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"umls": "C0079154", "parsed_text": "congenital ichthyosis" } } ] }, { "_id": "MONDO:0013024", "fda_orphan_drug": [ { "pubchem_cid": 16004692, "pubchem_sid": [ "135262332", "136023841", "152258558", "160647393", "160703277", "162202062", "163344650", "171572070", "173657394", "175427162", "178103924", "184815478", "223385903", "223701845", "252110242", "252166931", "252225701", "252451827", "255343087", "273010012", "273900173", "277425363", "310827476", "312616352", "313077413", "313079203", "315756410", "318163838", "319359736", "319470777", "319552840", "329878718", "336446249", "340487984", "340575568", "341143248", "342435774", "346408385", "347730497", "347913967", "348273535", "349060677", "349992721", "350080415", "354329137", "355040393", "373568500", "374075466", "374387382", "375588302", "375664304", "376033304", "376191619", "377310483", "378130437", "381371756", "382161039", "384568781", "384572792", "385635931", "386630109", "387108568", "402287739", "402288178", 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"378132651", "384991257", "385642091", "386478364", "387146460", "387261099", "404616494", "404638792", "405067359", "405487056", "419594761", "434310734", "438489562", "440033714", "440983677", "441105079" ], "inchikey": "QXWZQTURMXZVHJ-UHFFFAOYSA-N", "generic_name": "selexipag", "designated_date": "2018-10-02", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Janssen Research & Development LLC|1820 Chapel Ave West|Suite 300|Cherry Hill|New Jersey|8002|United States", "orphan_designation": { "original_text": "Treatment of Chronic Thromboembolic Pulmonary Hypertension", "umls": "C2363973", "parsed_text": "Chronic thromboembolic pulmonary hypertension" } }, { "pubchem_cid": 11304743, "pubchem_sid": [ "87550988", "126644888", "126680174", "135263495", "152258603", "160647437", "160682218", "164042214", "164347391", "170501515", "178101941", "185997052", "211536089", "223399697", "249821754", "251971381", "252155071", 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persistent/recurrent chronic thromboembolic pulmonary hypertension (CTEPH) WHO Group 4, after surgical treatment, or inoperable CTEPH, to improve exercise capacity and WHO functional class", "marketing_approval_date": "2013-10-08", "exclusivity_end_date": "2020-10-08", "exclusivity_protected_indication": "Treatment of adults with persistent/recurrent chronic thromboembolic pulmonary hypertension (CTEPH) WHO Group 4, after surgical treatment, or inoperable CTEPH, to improve exercise capacity and WHO functional class", "sponsor": "Bayer HealthCare Pharmaceuticals, Inc.|100 Bayer Blvd.|Whippany|New Jersey|7981|United States", "orphan_designation": { "original_text": "Treatment of chronic thromboembolic pulmonary hypertension", "umls": "C2363973", "parsed_text": "Chronic thromboembolic pulmonary hypertension" } } ] }, { "_id": "UMLS:C0085280", "fda_orphan_drug": [ { "pubchem_cid": 9831643, "pubchem_sid": [ "103469496", "249820159", "355177293", "385765900", "386602583" ], "inchikey": "STPKWKPURVSAJF-LJEWAXOPSA-N", "generic_name": "maralixibat", "designated_date": "2013-09-04", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Mirum Pharmaceuticals, Inc.|950 Tower Lane|Suite 1050|Foster City|California|94404|United States", "orphan_designation": { "original_text": "Treatment of alagille syndrome", "umls": "C0085280", "parsed_text": "Alagille Syndrome" } } ] }, { "_id": "MONDO:0005920", "fda_orphan_drug": [ { "pubchem_cid": 65329, "pubchem_sid": [ "11528737", "135022425", "162180987", "172090781", "253665632", "312694899", "319105993", "347914424", "375578027", "377311114", "438498659", "135590705", "163124581", "319345461", "160963705", "319222157", "319363938", "342526256", "366590561", "374034800", "318161535" ], "inchikey": "WESWYMRNZNDGBX-YLCXCWDSSA-N", "generic_name": "mefloquine HCL", "trade_name": "Lariam", "designated_date": "1988-04-13", "designation_status": "Designated/Approved", "approved_labeled_indication": "Prophylaxis of Plasmodium falciparum malaria which is resistant to other available drugs", "marketing_approval_date": "1989-05-03", "exclusivity_end_date": "1996-05-03", "sponsor": "Hoffmann-La Roche, Inc.|340 Kingsland Street|Nutley|New Jersey|7110|United States", "orphan_designation": { "original_text": "For use in the treatment of acute malaria due to Plasmodium falciparum and Plasmodium vivax, and for the prophylaxis of Plasmodium falciparum malaria which is resistant to other available drugs", "umls": [ "C0024535", "C2930826" ], "parsed_text": [ "Plasmodium falciparum Malaria", "Acute malaria" ] } }, { "pubchem_cid": 65329, "pubchem_sid": [ "11528737", "135022425", "162180987", "172090781", "253665632", "312694899", "319105993", "347914424", "375578027", "377311114", "438498659", "135590705", "163124581", "319345461", "160963705", "319222157", "319363938", "342526256", "366590561", "374034800", "318161535" ], "inchikey": "WESWYMRNZNDGBX-YLCXCWDSSA-N", "generic_name": "mefloquine HCL", "trade_name": "Lariam", "designated_date": "1988-04-13", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of acute malaria due to Plasmodium falciparum and Plasmodium vivax", "marketing_approval_date": "1989-05-02", "exclusivity_end_date": "1996-05-02", "sponsor": "Hoffmann-La Roche, Inc.|340 Kingsland Street|Nutley|New Jersey|7110|United States", "orphan_designation": { "original_text": "For use in the treatment of acute malaria due to Plasmodium falciparum and Plasmodium vivax, and for the prophylaxis of Plasmodium falciparum malaria which is resistant to other available drugs", "umls": [ "C0024535", "C2930826" ], "parsed_text": [ "Plasmodium falciparum Malaria", "Acute malaria" ] } } ] }, { "_id": "UMLS:C2930826", "fda_orphan_drug": [ { "pubchem_cid": 65329, "pubchem_sid": [ "11528737", "135022425", "162180987", "172090781", "253665632", "312694899", "319105993", "347914424", "375578027", "377311114", "438498659", "135590705", "163124581", "319345461", "160963705", "319222157", "319363938", "342526256", "366590561", "374034800", "318161535" ], "inchikey": "WESWYMRNZNDGBX-YLCXCWDSSA-N", "generic_name": "mefloquine HCL", "trade_name": "Lariam", "designated_date": "1988-04-13", "designation_status": "Designated/Approved", "approved_labeled_indication": "Prophylaxis of Plasmodium falciparum malaria which is resistant to other available drugs", "marketing_approval_date": "1989-05-03", "exclusivity_end_date": "1996-05-03", "sponsor": "Hoffmann-La Roche, Inc.|340 Kingsland Street|Nutley|New Jersey|7110|United States", "orphan_designation": { "original_text": "For use in the treatment of acute malaria due to Plasmodium falciparum and Plasmodium vivax, and for the prophylaxis of Plasmodium falciparum malaria which is resistant to other available drugs", "umls": [ "C0024535", "C2930826" ], "parsed_text": [ "Plasmodium falciparum Malaria", "Acute malaria" ] } }, { "pubchem_cid": 65329, "pubchem_sid": [ "11528737", "135022425", "162180987", "172090781", "253665632", "312694899", "319105993", "347914424", "375578027", "377311114", "438498659", "135590705", "163124581", "319345461", "160963705", "319222157", "319363938", "342526256", "366590561", "374034800", "318161535" ], "inchikey": "WESWYMRNZNDGBX-YLCXCWDSSA-N", "generic_name": "mefloquine HCL", "trade_name": "Lariam", "designated_date": "1988-04-13", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of acute malaria due to Plasmodium falciparum and Plasmodium vivax", "marketing_approval_date": "1989-05-02", "exclusivity_end_date": "1996-05-02", "sponsor": "Hoffmann-La Roche, Inc.|340 Kingsland Street|Nutley|New Jersey|7110|United States", "orphan_designation": { "original_text": "For use in the treatment of acute malaria due to Plasmodium falciparum and Plasmodium vivax, and for the prophylaxis of Plasmodium falciparum malaria which is resistant to other available drugs", "umls": [ "C0024535", "C2930826" ], "parsed_text": [ "Plasmodium falciparum Malaria", "Acute malaria" ] } }, { "pubchem_cid": 37393, "pubchem_sid": [ "9836", "603092", "7979519", "46506753", "48416078", "92719358", "103305814", "103994306", "126620878", "126653035", "126670922", "135029301", "160964551", "196110120", "223400586", "249967182", "252359462", "252390139", "252655909", "254783262", "273581513", "313074777", "315673727", "316966978", "318161259", "319087696", "319087697", "341142073", "348283796", "349256554", "349981736", "363350614", "375134293", "375570609", "375973209", "381359203", "384572224", "385639467", "386211343", "386633679", "387090368", "387090369", "419492975", "50003672", "134224374", "134998340", "355043294", "375685714", "384570872", "405498924", "440045580", "50003671", "85154881", "93167203", "223439899", "341825580", "415749680", "439573909", "438533166", "441072428" ], "inchikey": "FOHHNHSLJDZUGQ-UHFFFAOYSA-N", "generic_name": "Halofantrine", "trade_name": "Halfan", "designated_date": "1991-11-04", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of adults who can tolerate oral medication and who have mild to moderate malaria caused by Plasmodium falciparum or Plasmodium vivax.", "marketing_approval_date": "1992-07-24", "exclusivity_end_date": "1999-07-24", "sponsor": "SmithKline Beecham Pharmaceuticals|One Franklin Plaza|P.O. Box 7929|Philadelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "Treatment of mild to moderate acute malaria caused by susceptible strains of P. falciparum and P. vivax.", "umls": "C2930826", "parsed_text": "Acute malaria" } } ] }, { "_id": "UMLS:C2931139", "fda_orphan_drug": [ { "pubchem_cid": 4178, "pubchem_sid": [ "9429", "7979981", "46505491", "49969305", "50468461", "92729782", "103178542", "104010844", "104222091", "126656806", "126667667", "135000434", "135650603", "160963726", "162182772", "196107288", "224300994", "249911306", "273592166", "312228097", "315683626", "318161655", "319062770", "341838036", "348283066", "349994812", "355047476", "355068154", "355181512", "363593771", "375666446", "383435658", "385786983", "386268061", "386624033", "387165113", "402337528", "404617327", "404754967", "405479466", "419502473", "439374084", "439445652", "440026125", "440790273", "50468462", "93166656", "103394848", "104026219", "434124005", "50468463", "103394849", "104026220", "374917989", "341141648", "387165112", "441071712" ], "inchikey": "VLPIATFUUWWMKC-UHFFFAOYSA-N", "generic_name": "mexiletine", "designated_date": "2010-09-02", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "University of Rochester Medical Center|1351 Mt. Hope Ave.|Suite 203|Rochester|New York|14620|United States", "orphan_designation": { "original_text": "Treatment of nondystrophic myotonia", "umls": "C2931139", "parsed_text": "Nondystrophic myotonia" } } ] }, { "_id": "UMLS:C1148454", "fda_orphan_drug": [ { "pubchem_cid": 4192, "pubchem_sid": [ "9727", "7979987", "46507611", "48416279", "49689435", "49833675", "49978815", "50371480", "53789031", "93166805", "103189248", "126628994", "126644317", "126685927", "135006020", "160964028", "178100372", "210279804", "210282127", "223440338", "224544524", "241102041", "250117805", "252088595", "252352353", "275447667", "312228320", "313081519", "315673902", "318161663", "319062779", "340514832", "340514833", "340516103", "341140524", "341838472", "342421556", "348283326", "348401635", "349993618", "363592811", "368500146", "375587874", "381316813", "385785112", "386264671", "386622039", "387022014", "387083492", "387196102", "387208930", "404754970", "419506430", "433778883", "434301079", 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"sponsor": "UCB, Inc.|1950 Lake Park Drive|Building 2100|Smyrna|Georgia|30080|United States", "orphan_designation": { "original_text": "Rescue treatment of seizures in patients who require control of intermittent bouts of increased seizure activity (e.g. acute repetitive seizures, seizure clusters)", "umls": [ "C0036572", "C1148454", "C1959629" ], "parsed_text": [ "Seizures", "seizure activity", "Seizure" ] } }, { "pubchem_cid": 4192, "pubchem_sid": [ "9727", "7979987", "46507611", "48416279", "49689435", "49833675", "49978815", "50371480", "53789031", "93166805", "103189248", "126628994", "126644317", "126685927", "135006020", "160964028", "178100372", "210279804", "210282127", "223440338", "224544524", "241102041", "250117805", "252088595", "252352353", "275447667", "312228320", "313081519", "315673902", "318161663", "319062779", "340514832", "340514833", "340516103", "341140524", "341838472", "342421556", "348283326", "348401635", "349993618", "363592811", "368500146", "375587874", 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"inchikey": "VFKZTMPDYBFSTM-GUCUJZIJSA-N", "generic_name": "Mitolactol", "designated_date": "1989-01-23", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Targent, Inc..|155 Lambert Drive|Princeton|New Jersey|8540|United States", "orphan_designation": { "original_text": "Treatment of invasive carcinoma of the uterine cervix", "umls": [ "C0302592", "C1334274" ], "parsed_text": [ "Carcinoma of the Uterine Cervix", "Invasive Carcinoma" ] } } ] }, { "_id": "MONDO:0000745", "fda_orphan_drug": [ { "pubchem_cid": 1995, "pubchem_sid": [ "415872", "24890490", "51071853", "85083279", "87561793", "93576639", "126593556", "135027024", "136992199", "136996457", "160808482", "162179283", "162273445", "162760090", "163387814", "204364728", "223402904", "224829337", "249950014", "251884103", "252272402", "255415078", "275891160", "316440813", "316903423", "319061389", "321939066", "336226558", "342398339", "349519047", "354268082", 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"approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "N & N Pharmaceuticals, Inc.|2400 Research Blvd|Suite 310|Rockville|Maryland|20850|United States", "orphan_designation": { "original_text": "Treatment of neonatal herpes", "umls": "C2931185", "parsed_text": "Neonatal herpes" } } ] }, { "_id": "UMLS:C0007095", "fda_orphan_drug": [ { "pubchem_cid": 9870520, "pubchem_sid": [ "53790335", "103339635", "135302059", "319340172", "341194111", "347828609", "355426463", "385650457", "386486900", "404617123", "413049164", "434301636", "438483282", "441057066", "377642660" ], "inchikey": "YDZYKNJZCVIKPP-VWLOTQADSA-N", "generic_name": "netazepide", "designated_date": "2009-09-10", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Trio Medicines Ltd.|Pavillion View|19 New Road|Brighton|United Kingdom", "orphan_designation": { "original_text": "Treatment of gastric carcinoids", "umls": "C0007095", "parsed_text": 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"2019-10-02", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Sun Pharma Advanced Research Company Ltd.|17/B, Mahal Industrial Estate|Mahakali Caves Road|Andheri (E)|Mumbai|India", "orphan_designation": { "original_text": "Treatment of neonatal seizures", "umls": "C0159020", "parsed_text": "Neonatal seizures" } }, { "pubchem_cid": 23674889, "pubchem_sid": [ "46530538", "49857623", "78239323", "85189553", "92711019", "126631287", "126677733", "134970863", "144210591", "162177562", "252355877", "255379235", "313081868", "315672042", "318050637", "319375974", "341193521", "347919232", "355194568", "363898453", "385647532", "386483932", "53786889", "103396479", "135650817", "176317133", "318161974", "387171265", "387171266", "274621573" ], "inchikey": "WRLGYAWRGXKSKG-UHFFFAOYSA-M", "generic_name": "phenobarbital sodium", "designated_date": "2020-02-27", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Nivagen Pharmaceuticals, Inc.|3050 Fite Circle, Suite 100|Sacramento|California|95827|United States", "orphan_designation": { "original_text": "Treatment of neonatal seizures", "umls": "C0159020", "parsed_text": "Neonatal seizures" } } ] }, { "_id": "UMLS:C0039685", "fda_orphan_drug": [ { "pubchem_cid": 6041, "pubchem_sid": [ "9645", "598369", "7980308", "11533358", "46506961", "48416424", "49892965", "53787092", "56311763", "56311811", "77849473", "93166315", "103367925", "103961710", "126687263", "127259562", "127259563", "127259564", "127259565", "127259566", "127259567", "134972456", "135650819", "160963734", "223399283", "224125805", "241106148", "249911166", "275413988", "312226420", "313081876", "315674030", "318161986", "319064046", "329727346", "329727481", "340515491", "340518622", "341139157", "341837418", "348275653", "349990788", "354326248", "355176186", "363593975", "363906414", "374016238", "375204708", "375670454", "381364553", "381931620", "385633316", "386274959", "386621515", "387154632", "404755322", "406549274", "419504954", "434304443", "438486186", "438656866", "77849472", "249911497", "252355940", "387154631", "387233411", "405493239", "440039897", "77849476", "174554072", "77849474", "77849475", "77849477", "103147671", "441071824" ], "inchikey": "SONNWYBIRXJNDC-VIFPVBQESA-N", "generic_name": "phenylephrine", "designated_date": "2012-01-31", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Luitpold Pharmaceuticals, Inc.|One Luitpold Drive|P. O. Box 9001|Shirley|New York|11967|United States", "orphan_designation": { "original_text": "Treatment of Tetralogy of Fallot", "umls": "C0039685", "parsed_text": "Tetralogy of Fallot" } } ] }, { "_id": "MONDO:0015925", "fda_orphan_drug": [ { "pubchem_cid": 40632, "pubchem_sid": [ "12012763", "46500438", "50034790", "50104718", "50104719", "53787735", "78277830", "92304270", "123092043", "125307545", "126667061", "131408321", "135003538", "135684158", "136375549", "136991967", "144203776", "160809406", "162036238", "162177698", "162300359", "163088090", "163564861", "170465606", "170507527", "174555550", "175426915", "182629140", "187051833", "187072534", "204361652", "210279802", "210282125", "211534902", "223372231", "241182123", "242056874", "249807021", "251885743", "251916024", "251917372", "251971283", "252377930", "252391533", "252450504", "253668312", "254761189", "268738412", "273009199", "275388402", "277309574", "310279073", "312345031", "312616374", "312677060", "313076680", "318154583", "318163500", "318241025", "319089888", "319434697", "319539391", "319555499", "329991740", "340551687", "341142919", "341835068", "347913674", "348273655", "349104017", "349977197", "355038470", "370642381", "374017353", "375139759", "375394964", "375577914", "375586752", "375683368", "376142400", "376192427", "376220121", "377311173", "382162125", "385142590", "385646139", "386482518", "387106005", "419512072", "434303248", "438475773", "438486611", "438516941", "438596597", "440771305", "440973775", "364179720" ], "inchikey": "ISWRGOKTTBVCFA-UHFFFAOYSA-N", "generic_name": "pirfenidone", "designated_date": "2013-11-19", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genentech, Inc.|1 DNA Way|South San Francisco|California|94080|United States", "orphan_designation": { "original_text": "Treatment of systemic sclerosis (including the associated interstitial lung disease)", "umls": [ "C0036421", "C0206062" ], "parsed_text": [ "Systemic sclerosis", "Interstitial lung disease" ] } }, { "pubchem_cid": 107735, "pubchem_sid": [ "3128", "7985321", "10852056", "14709149", "50892566", "103036883", "104046194", "104097972", "104231801", "127293356", "127293357", "127293358", "127293359", "127293360", "127293361", "127293362", "127293363", "127293364", "127293365", "127293366", "127293367", "127293368", "127293369", "127293370", "127293371", "127293372", "127293373", "127293374", "127293375", "127293376", "127293377", "127293378", "135065889", "223739142", "315765911", "317230094", "319138950", "341813603", "363663697", "385651444", "386487897", "410760500", "439361701", "3324", "24844228", "46520143", "53789182", "85164948", "85267083", "103762230", "160963469", "178101511", "348274346" ], "inchikey": "LCTONWCANYUPML-UHFFFAOYSA-M", "generic_name": "Pyruvate", "designated_date": "2001-02-21", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Cellular Sciences, Inc|84 park Avenue|P.O. Box 968|Flemington|New Jersey|8822|United States", "orphan_designation": { "original_text": "Treatment of interstitial lung disease.", "umls": "C0206062", "parsed_text": "Interstitial lung disease" } }, { "pubchem_cid": 135423438, "pubchem_sid": [ "99431587", "103654502", "135267499", "152258852", "160647702", "178102559", "223471427", "249738349", "249807126", "251970985", "252090009", "255344081", "277286638", "310265007", "312616403", "318163907", "319339205", "319434165", "319539392", "340521879", "341092884", "341143400", "349988262", "350079274", "350080446", "350095295", "354330903", "355040172", "375577117", "375630398", "378126786", "383524001", "384568630", "384572576", "385645764", "386482130", "387177921", "387177922", "387237164", "402420917", "405045943", "405225436", "405496926", "419592141", "433794666", "434315123", "438493298", "439666458", "440043584", "440681567", "440794461", "440801773", "440802662" ], "inchikey": "CPMDPSXJELVGJG-UHFFFAOYSA-N", "generic_name": "nintedanib", "trade_name": "OFEV", "designated_date": "2016-07-06", "designation_status": "Designated/Approved", "approved_labeled_indication": "OFEV is indicated to slow the rate of decline in pulmonary function in patients with systemic sclerosis associated interstitial lung disease (SSc-ILD).", "marketing_approval_date": "2019-09-06", "exclusivity_end_date": "2026-09-06", "exclusivity_protected_indication": "Indicated to slow the rate of decline in pulmonary function in patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD).", "sponsor": "Boehringer Ingelheim Pharmaceuticals, Inc.|900 Ridgebury Road|P. O. Box 368|Ridgefield|Connecticut|6877|United States", "orphan_designation": { "original_text": "Treatment of systemic sclerosis (including the associated interstitial lung disease).", "umls": [ "C0036421", "C0206062" ], "parsed_text": [ "Systemic sclerosis", "Interstitial lung disease" ] } }, { "pubchem_cid": 40632, "pubchem_sid": [ "12012763", "46500438", "50034790", "50104718", "50104719", "53787735", "78277830", "92304270", "123092043", "125307545", "126667061", "131408321", "135003538", "135684158", "136375549", "136991967", "144203776", "160809406", "162036238", "162177698", "162300359", "163088090", "163564861", "170465606", "170507527", "174555550", "175426915", "182629140", "187051833", "187072534", "204361652", "210279802", "210282125", "211534902", "223372231", "241182123", "242056874", "249807021", "251885743", "251916024", "251917372", "251971283", "252377930", "252391533", "252450504", "253668312", "254761189", "268738412", "273009199", "275388402", "277309574", "310279073", "312345031", "312616374", "312677060", "313076680", "318154583", "318163500", "318241025", "319089888", "319434697", "319539391", "319555499", "329991740", "340551687", "341142919", "341835068", "347913674", "348273655", "349104017", "349977197", "355038470", "370642381", "374017353", "375139759", "375394964", "375577914", "375586752", "375683368", "376142400", "376192427", "376220121", "377311173", "382162125", "385142590", "385646139", "386482518", "387106005", "419512072", "434303248", "438475773", "438486611", "438516941", "438596597", "440771305", "440973775", "364179720" ], "inchikey": "ISWRGOKTTBVCFA-UHFFFAOYSA-N", "generic_name": "pirfenidone", "designated_date": "2020-03-11", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genentech Inc., A Member of the Roche Group|1 DNA Way|MS# 355F|South San Francisco|California|94080|United States", "orphan_designation": { "original_text": "Treatment of Unclassifiable-interstitial lung disease", "umls": "C0206062", "parsed_text": "Interstitial lung disease" } } ] }, { "_id": "UMLS:C0040557", "fda_orphan_drug": [ { "pubchem_cid": 54368, "pubchem_sid": [ "463557", "50035076", "50663483", "135012947", "160693381", "319098383", "385762588", "386599263", "596153" ], "inchikey": "IOEMETRLOWNXGW-UHFFFAOYSA-N", "generic_name": "Piritrexim isethionate", "designated_date": "1988-06-13", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Burroughs Wellcome Company|3030 Cornwallis Road|P.O. Box 12700|Research Triangle Park|North Carolina|27709|United States", "orphan_designation": { "original_text": "Treatment of infections caused by Pneumocystis carinii, Toxoplasma gondii, and Mycobacterium avium-intracellulare.", "umls": [ "C0026914", "C0040557", "C3714514" ], "parsed_text": [ "Mycobacterium avium-intracellulare", "Toxoplasma gondii", "Infections" ] } } ] }, { "_id": "UMLS:C0006434", "fda_orphan_drug": [ { "pubchem_cid": 24751, "pubchem_sid": [ "110201", "800165", "50002082", "87350387", "135020565", "251971147", "319079835", "350096139", "348287571", "439518097", "47208158", "319545334", "336269933", "347911189", "406845860", "406852363" ], "inchikey": "RVGRUAULSDPKGF-UHFFFAOYSA-N", "generic_name": "Poloxamer 188", "designated_date": "1990-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "CytRx Corporation|154 Technology Parkway|Norcross|Georgia|30092|United States", "orphan_designation": { "original_text": "Treatment of severe burns requiring hospitalization.", "umls": "C0006434", "parsed_text": "Burns" } }, { "pubchem_cid": 23668320, "pubchem_sid": [ "49869465", "134978979", "312531055", "319375012", "319070823", "319339078", "319373579" ], "inchikey": "GFJWACFSUSFUOG-ZJTJBYBXSA-M", "generic_name": "Dehydroepiandrosterone sulfate sodium", "designated_date": "1997-01-29", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Pharmadigm, Inc.|2401 Foothill Drive|Salt Lake City|Utah|84109|United States", "orphan_designation": { "original_text": "Treatment of serious burns requiring hospitalization.", "umls": "C0006434", "parsed_text": "Burns" } }, { "pubchem_sid": 379981111, "generic_name": "vibriolysin", "designated_date": "2006-06-16", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "BioMarin Pharmaceutical Inc.|105 Digital Drive|Novato|California|94949|United States", "orphan_designation": { "original_text": "Debridement of severe, deep dermal burns in hospitalized patients", "umls": "C0006434", "parsed_text": "Burns" } }, { "pubchem_sid": 347909969, "generic_name": "vibriolysin", "designated_date": "2006-06-16", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "BioMarin Pharmaceutical Inc.|105 Digital Drive|Novato|California|94949|United States", "orphan_designation": { "original_text": "Debridement of severe, deep dermal burns in hospitalized patients", "umls": "C0006434", "parsed_text": "Burns" } }, { "pubchem_sid": 135277988, "generic_name": "vibriolysin", "designated_date": "2006-06-16", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "BioMarin Pharmaceutical Inc.|105 Digital Drive|Novato|California|94949|United States", "orphan_designation": { "original_text": "Debridement of severe, deep dermal burns in hospitalized patients", "umls": "C0006434", "parsed_text": "Burns" } } ] }, { "_id": "MONDO:0015447", "fda_orphan_drug": [ { "pubchem_cid": 129073603, "pubchem_sid": [ "375588015", "375589321", "375973221", "381608874", "402382159", "428799677", "433796916", "438433353", "438790727", "440200762", "440792424", "386331594", "438433351" ], "inchikey": "GBLBJPZSROAGMF-SIYOEGHHSA-N", "generic_name": "pralsetinib", "trade_name": "Gavreto", "designated_date": "2020-05-26", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of (1) Adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy; and; (2) Adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate)", "marketing_approval_date": "2020-12-01", "exclusivity_end_date": "2027-12-01", "exclusivity_protected_indication": "Treatment of (1) Adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy; and; (2) Adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate)", "sponsor": "Blueprint Medicines Corporation|45 Sidney Street|Cambridge|Massachusetts|2139|United States", "orphan_designation": { "original_text": "Treatment of RET-fusion, RET-mutation, and TRKC-positive poorly differentiated thyroid cancer, undifferentiated or anaplastic thyroid cancer, medullary thyroid cancer, and locally advanced or metastatic follicular or papillary thyroid cancer", "umls": [ "C0238461", "C0238462", "C0238463", "C1337013" ], "parsed_text": [ "anaplastic thyroid cancer", "Thyroid cancer", "medullary", "papillary thyroid cancer", "Differentiated Thyroid Cancer" ] } }, { "pubchem_cid": 5803, "pubchem_sid": [ "46504696", "46518914", "50610406", "53788806", "103219116", "126679225", "134972266", "135651405", "144076004", "144203868", "144213850", "252543120", "254804296", "312323970", "312694876", "315681113", "318154166", "318163159", "319063791", "319461280", "319556973", "329871968", "341137219", "341163939", "347914042", "349090134", "349988451", "354269294", "355037607", "375590418", "377309481", "382161759", "383860401", "384386029", "385639343", "386633545", "387060598", "387160837", "405488619", "419484646", "434132799", "434313625", "438491722", "440035277", "441028394", "85257135", "364207340", "441075477" ], "inchikey": "UOWZUVNAGUAEQC-UHFFFAOYSA-N", "generic_name": "Tiratricol", "designated_date": "1991-08-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Laphal Laboratoires|48 Bis, Rue des Belles-Feuilles|Paris|France", "orphan_designation": { "original_text": "For use in combination with levo-thyroxine to suppress thyroid stimulating hormone in patients with well-differentiated thyroid cancer who are intolerant to adequate doses of levo-thyroxine alone.", "umls": "C1337013", "parsed_text": "Well-Differentiated Thyroid Cancer" } }, { "pubchem_cid": 134436906, "pubchem_sid": [ "381148416", "381148595", "381609315", "381744999", "386331670", "387059723", "406867341", "430057539", "433865382", "434310743", "438489574", "438645327", "438790728", "439507330", "440200949", "440702368", "440798555", "438433354", "438433356", "440792428" ], "inchikey": "XIIOFHFUYBLOLW-UHFFFAOYSA-N", "generic_name": "selpercatinib", "trade_name": "RETEVMO", "designated_date": "2019-11-21", "designation_status": "Designated/Approved", "approved_labeled_indication": "RETEVMO is indicated for the treatment of: Adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy; 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Box 8355|Wilmington|Delaware|19803|United States", "orphan_designation": { "original_text": "Adjuvant treatment of patients with stage III or stage IV differentiated thyroid cancer.", "umls": "C1337013", "parsed_text": "Differentiated Thyroid Cancer" } } ] }, { "_id": "MONDO:0006500", "fda_orphan_drug": [ { "pubchem_cid": 4946, "pubchem_sid": [ "9611", "597349", "7980417", "10525266", "26744226", "46505387", "48416484", "50023380", "53790381", "56413049", "75285225", "103164951", "134978179", "135650858", "160963916", "187072004", "223837119", "249934579", "274294624", "313077163", "315674081", "318162128", "319063213", "341139635", "341836422", "346383635", "348275406", "363592743", "363901032", "385324830", "385378959", "385785139", "386264585", "386622080", "387070977", "387070978", "404755102", "405489549", "433778949", "433780065", "439360214", "439462095", "440036207", "440791048", "75285226", "93166288", "223365931", "349978636", "387070976", "75285228", "104102155", 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"orphan_designation": { "original_text": "Prevention of infant respiratory distress syndrome associated with prematurity.", "umls": "C0035220", "parsed_text": "infant respiratory distress syndrome" } }, { "pubchem_sid": 347910369, "generic_name": "Beractant", "trade_name": "Survanta intratracheal suspension", "designated_date": "1986-02-05", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of (\"rescue\") of premature infants with RDS confirmed by x-ray and requiring mechanical ventilation", "marketing_approval_date": "1991-07-01", "exclusivity_end_date": "1998-07-01", "sponsor": "Ross Laboratories|625 Cleveland Avenue|Columbus|Ohio|43215|United States", "orphan_designation": { "original_text": "Treatment of neonatal respiratory distress syndrome", "umls": "C0035220", "parsed_text": "Neonatal Respiratory Distress Syndrome" } }, { "pubchem_sid": 135335308, "generic_name": "Beractant", "trade_name": "Survanta intratracheal suspension", 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than 1250 grams birthweight or with evidence of surfactant deficiency", "marketing_approval_date": "1991-07-02", "exclusivity_end_date": "1998-07-02", "sponsor": "Ross Laboratories|625 Cleveland Avenue|Columbus|Ohio|43215|United States", "orphan_designation": { "original_text": "Treatment of neonatal respiratory distress syndrome", "umls": "C0035220", "parsed_text": "Neonatal Respiratory Distress Syndrome" } }, { "pubchem_sid": 50070149, "generic_name": "Beractant", "trade_name": "Survanta intratracheal suspension", "designated_date": "1986-02-05", "designation_status": "Designated/Approved", "approved_labeled_indication": "Prevention of RDS (hyaline membrane disease) in premature infants less than 1250 grams birthweight or with evidence of surfactant deficiency", "marketing_approval_date": "1991-07-02", "exclusivity_end_date": "1998-07-02", "sponsor": "Ross Laboratories|625 Cleveland Avenue|Columbus|Ohio|43215|United States", "orphan_designation": { "original_text": "Treatment of 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River Road #250|Tucson|Alaska|85718|United States", "orphan_designation": { "original_text": "Treatment of Familial Adenomatous Polyposis", "umls": "C0032580", "parsed_text": "Familial Adenomatous Polyposis" } }, { "pubchem_cid": 446284, "pubchem_sid": [ "8663", "841846", "11533194", "26758663", "46506267", "53788482", "56311727", "56312089", "56312728", "56313867", "56314498", "96024753", "103582828", "134991117", "144205544", "160963508", "163688178", "178100387", "210279794", "210282117", "224483840", "254759349", "275072093", "310270318", "316405997", "316959051", "318162812", "319223590", "319555489", "348275519", "348397270", "348522370", "363901229", "378021511", "385783981", "386620840", "387107548", "404640421", "404760973", "409588443", "433775371", "440701978", "79827962", "96021247", "341140176", "347261629", "383231535", "387152113", "316966035", "319155312", "321909402", "342432676", "404786149", "340126745", "405435256", "439981925", "134980353", "135061774", "349986911" ], "inchikey": "JAZBEHYOTPTENJ-JLNKQSITSA-N", "generic_name": "eicosapentaenoic acid", "designated_date": "2011-03-08", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "S.L.A. Pharma Ltd. 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O. 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"adalimumab", "trade_name": "Humira", "designated_date": "2014-05-13", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of non-infectious intermediate, posterior, and panuveitis in adults and pediatric patients 2 years of age and older", "marketing_approval_date": "2018-09-28", "exclusivity_end_date": "2025-09-28", "exclusivity_protected_indication": "Treatment of non-infectious intermediate, posterior, and panuveitis in pediatric patients 2 years of age and older", "sponsor": "AbbVie, Inc.|1 North Waukegan Road|North Chicago|Illinois|60064|United States", "orphan_designation": { "original_text": "Treatment of non-infectious intermediate, posterior, or panuveitis, or chronic non-infectious anterior uveitis", "umls": [ "C0009450", "C0030343", "C0339317", "C0751438" ], "parsed_text": [ "Infectious", "Panuveitis", "Non-infectious anterior uveitis", "Posterior" ] } } ] }, { "_id": "UMLS:C4518725", "fda_orphan_drug": [ { "pubchem_cid": 5284616, 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"Treatment of anorexia, cachexia, or an unexplained significant weight loss in patients with a diagnosis of acquired immune deficiency syndrome.", "marketing_approval_date": "1993-09-10", "exclusivity_end_date": "2000-09-10", "sponsor": "Bristol-Myers Squibb Pharmaceutical Research Institute|2400 West Lloyd Expressway|Evansville|Indiana|47721|United States", "orphan_designation": { "original_text": "Treatment of patients with anorexia, cachexia, or significant weight loss (=/>10% of baseline body weight) and confirmed diagnosis of AIDS.", "umls": [ "C0001175", "C0006625", "C4084774" ], "parsed_text": [ "AIDS", "Cachexia", "Weight loss" ] } }, { "pubchem_cid": 16078, "pubchem_sid": [ "9186", "841255", "12013925", "46508472", "49855137", "53790103", "57389325", "78512325", "93166585", "103167825", "103920666", "134982169", "135651250", "160644679", "160963816", "175444080", "312228161", "318163419", "340514813", "340514814", "340514815", "349991117", "387021470", "387021471", 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"approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Unimed Pharmaceuticals, Inc.|901 Sawyer Rd.|Marietta|Georgia|30062|United States", "orphan_designation": { "original_text": "Treatment of weight loss in AIDS patients with HIV-associated wasting.", "umls": [ "C0006625", "C0019682", "C4084774" ], "parsed_text": [ "Wasting", "HIV", "Weight loss" ] } } ] }, { "_id": "MONDO:0005028", "fda_orphan_drug": [ { "pubchem_cid": 6435808, "pubchem_sid": [ "12014536", "50064326", "135017129", "162224110", "254759713", "315716969", "319316452", "341170610", "355058626", "385781265", "386617976", "404616030", "404770245", "406167157", "406168305", "419531929", "441038832", "135185730", "319340826", "355429656", "374355440", "405447936", "434312111", "438491244", "439994600", "50530764", "406804779" ], "inchikey": "GFFXZLZWLOBBLO-ASKVSEFXSA-N", "generic_name": "Tezacitabine", "designated_date": "2003-01-27", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Sanofi-Aventis US, Inc.|9 Great valley Parkway|Malvern|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of adenocarcinoma of the esophagus and stomach", "umls": "C0279628", "parsed_text": "adenocarcinoma of the esophagus" } } ] }, { "_id": "UMLS:C0744831", "fda_orphan_drug": [ { "pubchem_cid": 16130571, "pubchem_sid": [ "50054734", "56459441", "85335659", "135234006", "160671058", "251971434", "313075632", "318162438", "319360135", "319557613", "340470686", "341108344", "349987930", "354732282", "355036819", "375686504", "376046473", "376229513", "384566835", "422600775", "354701772", "363743246", "375744190", "46504578", "318474065", "318478064", "341200324", "349253776" ], "inchikey": "NZVYCXVTEHPMHE-ZSUJOUNUSA-N", "generic_name": "thymalfasin", "designated_date": "1991-05-03", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "SciClone Pharmaceuticals, Inc.|950 Tower Lane|Suite 900|Foster City|California|94404|United States", "orphan_designation": { "original_text": "Treatment of chronic active hepatitis B", "umls": "C0744831", "parsed_text": "Chronic active hepatitis B" } }, { "pubchem_cid": 50313, "pubchem_sid": [ "626305", "7979226", "12013388", "50059550", "78681954", "96099919", "103570568", "126591111", "126684988", "135009707", "160846590", "312343818", "312343985", "319095878", "341850818", "349980311", "355046796", "355168379", "385789677", "386626825", "404772854", "434251706", "438456351", "438477175", "440066138", "440703482", "440790253", "441005642", "517727", "78681949", "375570422", "387255474", "437412721", "160871154", "162263046", "370627518", "405498616", "440045272", "78681948", "78681950", "78681951", "78681952", "78681953", "78681955" ], "inchikey": "IPVFGAYTKQKGBM-BYPJNBLXSA-N", "generic_name": "fialuridine", "designated_date": "1992-07-24", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Oclassen Pharmaceuticals, Inc.|100 Pelican Way|San Rafael|California|94901|United States", "orphan_designation": { "original_text": "Adjunctive treatment of chronic active hepatitis B.", "umls": "C0744831", "parsed_text": "Chronic active hepatitis B" } } ] }, { "_id": "UMLS:C0022650", "fda_orphan_drug": [ { "pubchem_cid": 5483, "pubchem_sid": [ "583265", "610366", "48416637", "49973761", "75941066", "87560971", "92098527", "92714304", "103409496", "103931530", "118043432", "126624247", "126669587", "134981860", "144076409", "144205207", "162175489", "162228427", "170465217", "210279517", "210281840", "223441841", "252357562", "252453765", "254760279", "275844728", "312246002", "312598460", "312995310", "313082398", "315521003", "315674207", "316474599", "318162469", "319555055", "322075098", "332867519", "340510861", "341141763", "341835562", "346431324", "347827801", "348397573", "348481911", "349984426", "355038704", "363896125", "363899885", "374057531", "375082674", "375570227", "375639043", "377310210", "384417046", "384568176", "384571965", "385785006", "386621925", "387181596", "387181597", "404614296", "405467780", "414858523", "434132751", "434313609", "438491650", "438613555", "440014441", "440791057", "441049103", "75941068", "252088950", "381342835", "75941067", "406819142", "342552643", "441079279" ], "inchikey": "YTGJWQPHMWSCST-UHFFFAOYSA-N", "generic_name": "Tiopronin", "trade_name": "Thiola", "designated_date": "1986-01-17", "designation_status": "Designated/Approved", "approved_labeled_indication": "THIOLA is indicated, in combination with high fluid intake, alkali, and diet modification, for the prevention of cystine stone formation in adults and pediatric patients 20 kg and greater with severe homozygous cystinuria, who are not responsive to these measures alone.", "marketing_approval_date": "2019-06-28", "exclusivity_end_date": "2026-06-28", "exclusivity_protected_indication": "Indicated in combination with high fluid intake, alkali, and diet modification, for the prevention of cystine stone formation in pediatric patients between 20 kg and 9 years of age with severe homozygous cystinuria, who are not responsive to these measures alone", "sponsor": "Mission Pharmacal Company|P.O. Box 786099|San Antonio|Texas|78278|United States", "orphan_designation": { "original_text": "Prevention of cystine nephrolithiasis in patients with homozygous cystinuria.", "umls": [ "C0010691", "C0022650" ], "parsed_text": [ "Cystinuria", "Nephrolithiasis" ] } }, { "pubchem_cid": 2724354, "pubchem_sid": [ "9800", "46504680", "50048359", "76750784", "81065526", "103771056", "134973780", "135610136", "162221387", "241059553", "255370644", "312236789", "312694458", "315674141", "318162295", "319246991", "341143136", "349975619", "355178528", "355202866", "355203258", "387068439", "402382156", "404613890", "404639074", "434313106", "438489392", "438791036", "440790456", "598336", "48416560", "76750782", "103849901", "117392416", "126670172", "135056651", "160963911", "249958968", "319067694", "348283226", "387068438", "405478573", "407344867", "440025232", "76750783", "204359704", "441073219" ], "inchikey": "ACTRVOBWPAIOHC-XIXRPRMCSA-N", "generic_name": "Succimer", "designated_date": "1990-11-05", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Sanofi Winthrop, Inc.|90 Park Avenue|New York|New York|10016|United States", "orphan_designation": { "original_text": "Prevention of cystine kidney stone formation in patients with homozygous cystinuria who are prone to stone development.", "umls": [ "C0010691", "C0022650" ], "parsed_text": [ "Cystinuria", "KIDNEY STONE" ] } }, { "pubchem_cid": 5483, "pubchem_sid": [ "583265", "610366", "48416637", "49973761", "75941066", "87560971", "92098527", "92714304", "103409496", "103931530", "118043432", "126624247", "126669587", "134981860", "144076409", "144205207", "162175489", "162228427", "170465217", "210279517", "210281840", "223441841", "252357562", "252453765", "254760279", "275844728", "312246002", "312598460", "312995310", "313082398", "315521003", "315674207", "316474599", "318162469", "319555055", "322075098", "332867519", "340510861", "341141763", "341835562", "346431324", "347827801", "348397573", "348481911", "349984426", "355038704", "363896125", "363899885", "374057531", "375082674", "375570227", "375639043", "377310210", "384417046", "384568176", "384571965", "385785006", "386621925", "387181596", "387181597", "404614296", "405467780", "414858523", "434132751", "434313609", "438491650", "438613555", "440014441", "440791057", "441049103", "75941068", "252088950", "381342835", "75941067", "406819142", "342552643", "441079279" ], "inchikey": "YTGJWQPHMWSCST-UHFFFAOYSA-N", "generic_name": "Tiopronin", "trade_name": "Thiola", "designated_date": "1986-01-17", "designation_status": "Designated/Approved", "approved_labeled_indication": "Indicated for the prevention of cystine (kidney) stone formation in patients with severe homozygous cystinuria with urinary cystine greater than 500 mg/day, who are resistant to treatment with conservative measures of high fluid intake, alkali and diet modification or who have adverse reactions to d-penicillamine.", "marketing_approval_date": "1988-08-11", "exclusivity_end_date": "1995-08-11", "sponsor": "Mission Pharmacal Company|P.O. Box 786099|San Antonio|Texas|78278|United States", "orphan_designation": { "original_text": "Prevention of cystine nephrolithiasis in patients with homozygous cystinuria.", "umls": [ "C0010691", "C0022650" ], "parsed_text": [ "Cystinuria", "Nephrolithiasis" ] } }, { "pubchem_cid": 13344, "pubchem_sid": [ "50065333", "103770441", "117373240", "126674774", "127258808", "131269083", "134980566", "136349152", "162184620", "162223358", "162725308", "174531075", "223772474", "274348268", "310265041", "313081948", "316603623", "319071514", "341128587", "341142203", "349981896", "375096827", "375668794", "381352074", "385786541", "386623547", "387236913", "438793872", "439642354", "11528474", "134988749", "136349201", "355196264", "363904493", "374355434", "438472970", "76763267", "144076300", "342559499" ], "inchikey": "QEEAPRPFLLJWCF-UHFFFAOYSA-K", "generic_name": "Potassium citrate", "trade_name": "Urocit-K", "designated_date": "1984-11-01", "designation_status": "Designated/Approved", "approved_labeled_indication": "Prevention of recurring calcium conating stines associated with a deficiency in the urine.", "marketing_approval_date": "1985-08-07", "exclusivity_end_date": "1992-08-07", "sponsor": "University of Texas Health Science Center at Dallas|5323 Harry Hines Blvd|Dallas|Texas|75235|United States", "orphan_designation": { "original_text": "1. Prevention of calcium renal stones in patients with hypocitraturia 2. For avoidance of the complication of calcium stone formation in patients with uric lithiasis.", "umls": [ "C0009566", "C0022650", "C0023869", "C2673444" ], "parsed_text": [ "Complication", "renal stones", "Lithiasis", "Hypocitraturia" ] } }, { "pubchem_cid": 5483, "pubchem_sid": [ "583265", "610366", "48416637", "49973761", "75941066", "87560971", "92098527", "92714304", "103409496", "103931530", "118043432", "126624247", "126669587", "134981860", "144076409", "144205207", "162175489", "162228427", "170465217", "210279517", "210281840", "223441841", "252357562", "252453765", "254760279", "275844728", "312246002", "312598460", "312995310", "313082398", "315521003", "315674207", "316474599", "318162469", "319555055", "322075098", "332867519", "340510861", "341141763", "341835562", "346431324", "347827801", "348397573", "348481911", "349984426", "355038704", "363896125", "363899885", "374057531", "375082674", "375570227", "375639043", "377310210", "384417046", "384568176", "384571965", "385785006", "386621925", "387181596", "387181597", "404614296", "405467780", "414858523", "434132751", "434313609", "438491650", "438613555", "440014441", "440791057", "441049103", "75941068", "252088950", "381342835", "75941067", "406819142", "342552643", "441079279" ], "inchikey": "YTGJWQPHMWSCST-UHFFFAOYSA-N", "generic_name": "Tiopronin", "trade_name": "Thiola", "designated_date": "1986-01-17", "designation_status": "Designated/Approved", "approved_labeled_indication": "THIOLA EC is indicated, in combination with high fluid intake, alkali, and diet modification, for the prevention of cystine stone formation in adults and pediatric patients 20 kg and greater with severe homozygous cystinuria, who are not responsive to these measures alone.", "marketing_approval_date": "2019-06-28", "exclusivity_end_date": "2026-06-28", "sponsor": "Mission Pharmacal Company|P.O. Box 786099|San Antonio|Texas|78278|United States", "orphan_designation": { "original_text": "Prevention of cystine nephrolithiasis in patients with homozygous cystinuria.", "umls": [ "C0010691", "C0022650" ], "parsed_text": [ "Cystinuria", "Nephrolithiasis" ] } } ] }, { "_id": "UMLS:C0005779", "fda_orphan_drug": [ { "pubchem_cid": 5526, "pubchem_sid": [ "145436", "224560", "582925", "7980823", "8149799", "10321473", "46508089", "49658609", "49957672", "50219249", "87561897", "92717664", "103231281", "103913967", "117686924", "126628774", "126649567", "126653759", "126667234", "126690001", "134224929", "134354951", "134981301", "144075469", "144204051", "160963650", "162038172", "162177705", "164156942", "178103187", "184812220", "196112276", "210279267", "210281590", "222137599", "223370774", "223769484", "249897012", "251891231", "252402045", "252409021", "253653904", "254759553", "254806438", "255370733", "268734858", "274954660", "275844701", "310281093", "312236676", "312693558", "313075738", "313082425", "315681163", "316403374", "316967912", "318154169", "318162505", "318242821", "319063596", "319554744", "340515548", "340518773", "341141243", "341835596", "346420322", "347739729", "347761007", "348283002", "348397580", "348616497", "349979562", "355038906", "355155444", "362795116", "363743083", "374032331", "375570828", "375647648", "376051665", "376226240", "381410348", "382154022", "384567950", "384571656", "385633301", "386621799", "387097098", "387097099", "434131377", "434314249", "438472671", "438491588", "439592675", "439642377", "440064433", "440681881", "440790963", "440818421", "375682500", "405441773", "439988440", "441072467" ], "inchikey": "GYDJEQRTZSCIOI-UHFFFAOYSA-N", "generic_name": "tranexamic acid", "trade_name": "Cyklokapron", "designated_date": "1985-12-03", "designation_status": "Designated/Approved/Withdrawn", "approved_labeled_indication": "Treatment of patients with hemophilia for short term use (2 to 8 days) before and after tooth extraction to reduce or prevent hemorrhage abd reduce the need for replacement therapy", "marketing_approval_date": "1986-12-30", "exclusivity_end_date": "1993-12-30", "sponsor": "Pharmacia Inc.|P.O. Box 16529|Columbus|Ohio|43216|United States", "orphan_designation": { "original_text": "For use in patients with congenital coagulopathies undergoing surgical procedures, e.g. dental extractions", "umls": "C0005779", "parsed_text": "coagulopathies" } } ] }, { "_id": "MONDO:0009499", "fda_orphan_drug": [ { "pubchem_cid": 444539, "pubchem_sid": [ "3713", "98281", "607059", "10507636", "14717783", "29203962", "49955076", "53788561", "85164956", "87565486", "87565727", "103198631", "117605457", "126607060", "126677307", "134974596", "143489610", "143504134", "152223082", "152242944", "162068122", "162173393", "173128588", "174548706", "204357641", "204937003", "223821105", "250197604", "251881044", "252405011", "254831346", "255399184", "275891825", "310270273", "312225960", "316395104", "316963351", "316969810", "319223453", "319457225", "348274924", "363602855", "363901140", "374050249", "375303891", "375645777", "377493296", "377923192", "381396104", "382408039", "383469740", "384256124", "385878521", "386283426", "387168262", "404635768", "404760954", "406703569", "438470595", "438507229", "440818645", "51073550", "85084882", "160788047", "163303384", "163372525", "163802377", "252260812", "312682793", "92250589", "201501669", "348480188", "381148484" ], "inchikey": "WBYWAXJHAXSJNI-VOTSOKGWSA-N", "generic_name": "Trans-Cinnamic Acid", "designated_date": "2021-02-03", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Polaryx Therapeutics, Inc.|140 E. 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"umls": "C0079772", "parsed_text": "T-Cell Lymphoma" } }, { "pubchem_cid": 154574495, "pubchem_sid": [ "494874", "12014448", "46519185", "50062237", "53788098", "103393180", "104222481", "131408687", "163620827", "163686155", "164233439", "178103585", "184816960", "252157318", "252450287", "252473284", "275977589", "310264861", "312236778", "313077423", "318163429", "319298392", "319553162", "347713769", "347740974", "348273338", "349989406", "355039966", "381405946", "385640659", "386634915", "419582665", "438494403", "440790927", "252216224", "254759686", "255372230", "319406655", "377440328", "381183733", "384568674", "384572638", "438475532", "312624244", "319062307", "348349919", "363742771", "405447423", "439994087", "135031623", "144206460", "315683742", "136360772", "224686725", "317223454", "319112978", "319313674", "319420491", "319431911", "342600614", "342601692", "375587347", "383460094", "433841714", "438635195" ], "inchikey": "OHRURASPPZQGQM-QPJBHPOGSA-N", "generic_name": "romidepsin", "trade_name": "Istodax", "designated_date": "2004-09-30", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of cutaneous T-cell lymphoma (CTCL) in patients who have received at least one prior systemic therapy", "marketing_approval_date": "2009-11-05", "exclusivity_end_date": "2016-11-05", "sponsor": "Celgene Corporation|One Broadway|14th Floor|Cambridge|Massachusetts|2142|United States", "orphan_designation": { "original_text": "Treatment of non-Hodgkin T-cell lymphomas", "umls": "C0079772", "parsed_text": "T-Cell Lymphomas" } }, { "pubchem_cid": 129905275, "pubchem_sid": [ "494874", "12014448", "46519185", "50062237", "53788098", "103393180", "104222481", "131408687", "163620827", "163686155", "164233439", "178103585", "184816960", "252157318", "252450287", "252473284", "275977589", "310264861", "312236778", "313077423", "318163429", "319298392", "319553162", "347713769", "347740974", "348273338", "349989406", 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"2020-06-03", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Ophtho-Therapeutics, Inc.|324 S. Hyde Park Avenue|Tampa|Florida|33606|United States", "orphan_designation": { "original_text": "Treatment of vernal keratoconjunctivitis", "umls": "C0022577", "parsed_text": "Vernal keratoconjunctivitis" } } ] }, { "_id": "MONDO:0001083", "fda_orphan_drug": [ { "pubchem_cid": 6058, "pubchem_sid": [ "4821", "600739", "8145727", "11108719", "46507730", "48415835", "49966810", "56459392", "85165181", "87464908", "87464911", "87562212", "92298308", "103183110", "127338412", "127338413", "127338414", "134972777", "144206643", "160964188", "162188090", "162758643", "176318769", "193209844", "210278406", "210281443", "223751478", "249897037", "251891730", "252447700", "254761397", "274316027", "312226554", "315341088", "315673527", "318160737", "319064068", "319555392", "340506533", "341141117", "348275932", "349153437", "349993887", "355039010", "363602823", "363894901", "363901033", "374170459", "383231463", "384254986", "385788573", "386270515", "386625688", "387022742", "387159087", "405493711", "406715091", "406857227", "419521709", "434250136", "438455530", "440040369", "440791099", "441005048", "441111942", "57391518", "103158339", "404768057", "441083733" ], "inchikey": "UFULAYFCSOUIOV-UHFFFAOYSA-N", "generic_name": "Cysteamine", "designated_date": "1986-05-01", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Thoene, Jess G., M.D.|University of Michigan Medical School|300 NIB, 1192 SE|Ann Arbor|Michigan|48109|United States", "orphan_designation": { "original_text": "Treatment of nephropathic cystinosis.", "umls": "C0015624", "parsed_text": "Nephropathic cystinosis" } }, { "pubchem_cid": 6058, "pubchem_sid": [ "4821", "600739", "8145727", "11108719", "46507730", "48415835", "49966810", "56459392", "85165181", "87464908", "87464911", "87562212", "92298308", "103183110", "127338412", "127338413", "127338414", "134972777", "144206643", "160964188", "162188090", "162758643", "176318769", "193209844", "210278406", "210281443", "223751478", "249897037", "251891730", "252447700", "254761397", "274316027", "312226554", "315341088", "315673527", "318160737", "319064068", "319555392", "340506533", "341141117", "348275932", "349153437", "349993887", "355039010", "363602823", "363894901", "363901033", "374170459", "383231463", "384254986", "385788573", "386270515", "386625688", "387022742", "387159087", "405493711", "406715091", "406857227", "419521709", "434250136", "438455530", "440040369", "440791099", "441005048", "441111942", "57391518", "103158339", "404768057", "441083733" ], "inchikey": "UFULAYFCSOUIOV-UHFFFAOYSA-N", "generic_name": "Cysteamine", "trade_name": "Cystagon", "designated_date": "1991-01-25", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of nephropathic cystinosis in adults and children.", "marketing_approval_date": "1994-08-15", "exclusivity_end_date": "2001-08-15", "sponsor": "Mylan Laboratories, Inc.|781 Chestnut Ridge Road|P.O. Box 4310|Morgantown|West Virginia|26504|United States", "orphan_designation": { "original_text": "Treatment of nephropathic cystinosis.", "umls": "C0015624", "parsed_text": "Nephropathic cystinosis" } } ] }, { "_id": "UMLS:C1291601", "fda_orphan_drug": [ { "pubchem_cid": 18950, "pubchem_sid": [ "3459", "14720290", "22396012", "49747961", "53788969", "56311267", "56475044", "85164870", "103583168", "134228441", "134228449", "134958993", "175443373", "178101367", "194946299", "251973838", "253659400", "254774699", "310276046", "312225645", "315678177", "322093581", "342589039", "348274293", "349987268", "355204497", "376042568", "377796229", "378020276", "381376803", "385633527", "386619648", "387171180", "404639930", "433775074", "433776238", "433979675", "438515032", "439563517", "441049341", "2930", "99234417", "103816437", "103816438", "117585574", "134984714", "223828616", "252519341", "255368258", "316604234", "319175981", "321933713", "342526601", "347829059", "349751365", 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"UMLS:C1417244", "fda_orphan_drug": [ { "pubchem_cid": 18950, "pubchem_sid": [ "3459", "14720290", "22396012", "49747961", "53788969", "56311267", "56475044", "85164870", "103583168", "134228441", "134228449", "134958993", "175443373", "178101367", "194946299", "251973838", "253659400", "254774699", "310276046", "312225645", "315678177", "322093581", "342589039", "348274293", "349987268", "355204497", "376042568", "377796229", "378020276", "381376803", "385633527", "386619648", "387171180", "404639930", "433775074", "433776238", "433979675", "438515032", "439563517", "441049341", "2930", "99234417", "103816437", "103816438", "117585574", "134984714", "223828616", "252519341", "255368258", "316604234", "319175981", "321933713", "342526601", "347829059", "349751365", "354274828", "355203263", "363603834", "373993963", "375986792", "381331053", "383418155", "384434465", "387097288", "404737508", "404760149", "404825928", "87133", "10519500", "85087455", "163408326", "318467246", "319079830", "374168602", "405481192", "406534159", "440027851", "26759082", "126674458", "163687767", "176324233", "103825365", "117557265", "433981081", "85267543", "85267554", "164228456", "319344203", "340509673", "375590473", "386204274", "438543377" ], "inchikey": "WQZGKKKJIJFFOK-QTVWNMPRSA-N", "generic_name": "D-mannose", "designated_date": "2019-01-14", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Cerecor Inc.|1500 Liberty Ridge Drive|Suite 321|Wayne|Pennsylvania|19087|United States", "orphan_designation": { "original_text": "Treatment of Mannose Phosphate Isomerase Deficiency", "umls": [ "C1291601", "C1417244" ], "parsed_text": [ "isomerase deficiency", "mannose phosphate isomerase" ] } } ] }, { "_id": "MONDO:0019790", "fda_orphan_drug": [ { "pubchem_cid": 136675526, "pubchem_sid": [ "8149931", "49977301", "50064615", "50086896", "50106202", "50224504", "96079580", "103241627", "104170142", "135022024", "135697908", "252359371", "252402840", "254759916", "313080600", "319331846", "319331847", "355037251", "376219638", "376228113", "381382564", "381394157", "383840781", "404622018", "404839553", "434131969", "440064426", "441056653", "50224503", "92303995", "126628766", "312344549", "316899266", "316966591", "318031413", "319353482", "350086829", "350095976", "374043658", "375134207", "49977300", "53789979", "144205566", "144212314", "160964552", "170464793", "315680529", "318160748", "319362120", "319362121", "322079894", "341838939", "349988026", "385663493", "386500005", "387115919", "404639670", "441114322", "85383553", "126659538", "134996352", "144075555", "315495698", "319334576", "319378575", "319378576", "438473989", "50104441", "387065024", "163564378", "255369980", "268735387", "268738067", "274885735", "319407141", "342560733", "348777476", "368494613", "375575894", "441078404" ], "inchikey": "KSRLIXGNPXAZHD-DFPPEFKWSA-N", "generic_name": "Dantrolene sodium", "designated_date": "1987-12-14", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Norwich Eaton Pharmaceuticals|P.O. Box 191|Norwich|New York|13815|United States", "orphan_designation": { "original_text": "Treatment of the neuroleptic malignant syndrome.", "umls": "C0027849", "parsed_text": "Neuroleptic Malignant Syndrome" } }, { "pubchem_cid": 136675526, "pubchem_sid": [ "8149931", "49977301", "50064615", "50086896", "50106202", "50224504", "96079580", "103241627", "104170142", "135022024", "135697908", "252359371", "252402840", "254759916", "313080600", "319331846", "319331847", "355037251", "376219638", "376228113", "381382564", "381394157", "383840781", "404622018", "404839553", "434131969", "440064426", "441056653", "50224503", "92303995", "126628766", "312344549", "316899266", "316966591", "318031413", "319353482", "350086829", "350095976", "374043658", "375134207", "49977300", "53789979", "144205566", "144212314", "160964552", "170464793", "315680529", "318160748", "319362120", "319362121", "322079894", "341838939", "349988026", "385663493", "386500005", "387115919", "404639670", "441114322", "85383553", "126659538", "134996352", "144075555", "315495698", "319334576", "319378575", "319378576", "438473989", "50104441", "387065024", "163564378", "255369980", "268735387", "268738067", "274885735", "319407141", "342560733", "348777476", "368494613", "375575894", "441078404" ], "inchikey": "KSRLIXGNPXAZHD-DFPPEFKWSA-N", "generic_name": "Dantrolene sodium", "designated_date": "1987-12-14", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Norwich Eaton Pharmaceuticals|P.O. Box 191|Norwich|New York|13815|United States", "orphan_designation": { "original_text": "Treatment of the neuroleptic malignant syndrome.", "umls": "C0027849", "parsed_text": "Neuroleptic Malignant Syndrome" } }, { "pubchem_cid": 71563341, "pubchem_sid": [ "8149931", "49977301", "50064615", "50086896", "50106202", "50224504", "96079580", "103241627", "104170142", "135022024", "135697908", "252359371", "252402840", "254759916", "313080600", "319331846", "319331847", "355037251", "376219638", "376228113", "381382564", "381394157", "383840781", "404622018", "404839553", "434131969", "440064426", "441056653", "50224503", "92303995", "126628766", "312344549", "316899266", "316966591", "318031413", "319353482", "350086829", "350095976", "374043658", "375134207", "49977300", "53789979", "144205566", "144212314", "160964552", "170464793", "315680529", "318160748", "319362120", "319362121", "322079894", "341838939", "349988026", "385663493", "386500005", "387115919", "404639670", "441114322", "85383553", "126659538", "134996352", "144075555", "315495698", "319334576", "319378575", "319378576", "438473989", "50104441", "387065024", "163564378", "255369980", "268735387", "268738067", "274885735", "319407141", "342560733", "348777476", "368494613", "375575894", "441078404" ], "inchikey": "KSRLIXGNPXAZHD-DFPPEFKWSA-M", "generic_name": "Dantrolene sodium", "designated_date": "1987-12-14", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Norwich Eaton Pharmaceuticals|P.O. Box 191|Norwich|New York|13815|United States", "orphan_designation": { "original_text": "Treatment of the neuroleptic malignant syndrome.", "umls": "C0027849", "parsed_text": "Neuroleptic Malignant Syndrome" } }, { "pubchem_cid": 5353563, "pubchem_sid": [ "8149931", "49977301", "50064615", "50086896", "50106202", "50224504", "96079580", "103241627", "104170142", "135022024", "135697908", "252359371", "252402840", "254759916", "313080600", "319331846", "319331847", "355037251", "376219638", "376228113", "381382564", "381394157", "383840781", "404622018", "404839553", "434131969", "440064426", "441056653", "50224503", "92303995", "126628766", "312344549", "316899266", "316966591", "318031413", "319353482", "350086829", "350095976", "374043658", "375134207", "49977300", "53789979", "144205566", "144212314", "160964552", "170464793", "315680529", "318160748", "319362120", "319362121", "322079894", "341838939", "349988026", "385663493", "386500005", "387115919", "404639670", "441114322", "85383553", "126659538", "134996352", "144075555", "315495698", "319334576", "319378575", "319378576", "438473989", "50104441", "387065024", "163564378", "255369980", "268735387", "268738067", "274885735", "319407141", "342560733", "348777476", "368494613", "375575894", "441078404" ], "inchikey": "OZOMQRBLCMDCEG-CHHVJCJISA-M", "generic_name": "Dantrolene sodium", "designated_date": "1987-12-14", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Norwich Eaton Pharmaceuticals|P.O. Box 191|Norwich|New York|13815|United States", "orphan_designation": { "original_text": "Treatment of the neuroleptic malignant syndrome.", "umls": "C0027849", "parsed_text": "Neuroleptic Malignant Syndrome" } }, { "pubchem_cid": 6604728, "pubchem_sid": [ "8149931", "49977301", "50064615", "50086896", "50106202", "50224504", "96079580", "103241627", "104170142", "135022024", "135697908", "252359371", "252402840", "254759916", "313080600", "319331846", "319331847", "355037251", "376219638", "376228113", "381382564", "381394157", "383840781", "404622018", "404839553", "434131969", "440064426", "441056653", "50224503", "92303995", "126628766", "312344549", "316899266", "316966591", "318031413", "319353482", "350086829", "350095976", "374043658", "375134207", "49977300", "53789979", "144205566", "144212314", "160964552", "170464793", "315680529", "318160748", "319362120", "319362121", "322079894", "341838939", "349988026", "385663493", "386500005", "387115919", "404639670", "441114322", "85383553", "126659538", "134996352", "144075555", "315495698", "319334576", "319378575", "319378576", "438473989", "50104441", "387065024", "163564378", "255369980", "268735387", "268738067", "274885735", "319407141", "342560733", "348777476", "368494613", "375575894", "441078404" ], "inchikey": "OZOMQRBLCMDCEG-VIZOYTHASA-M", "generic_name": "Dantrolene sodium", "designated_date": "1987-12-14", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Norwich Eaton Pharmaceuticals|P.O. Box 191|Norwich|New York|13815|United States", "orphan_designation": { "original_text": "Treatment of the neuroleptic malignant syndrome.", "umls": "C0027849", "parsed_text": "Neuroleptic Malignant Syndrome" } }, { "pubchem_cid": 23707296, "pubchem_sid": [ "8149931", "49977301", "50064615", "50086896", "50106202", "50224504", "96079580", "103241627", "104170142", "135022024", "135697908", "252359371", "252402840", "254759916", "313080600", "319331846", "319331847", "355037251", "376219638", "376228113", "381382564", "381394157", "383840781", "404622018", "404839553", "434131969", "440064426", "441056653", "50224503", "92303995", "126628766", "312344549", "316899266", "316966591", "318031413", "319353482", "350086829", "350095976", "374043658", "375134207", "49977300", "53789979", "144205566", "144212314", "160964552", "170464793", "315680529", "318160748", "319362120", "319362121", "322079894", "341838939", "349988026", "385663493", "386500005", "387115919", "404639670", "441114322", "85383553", "126659538", "134996352", "144075555", "315495698", "319334576", "319378575", "319378576", "438473989", "50104441", "387065024", "163564378", "255369980", "268735387", "268738067", "274885735", "319407141", "342560733", "348777476", "368494613", "375575894", "441078404" ], "inchikey": "KSRLIXGNPXAZHD-HAZZGOGXSA-N", "generic_name": "Dantrolene sodium", "designated_date": "1987-12-14", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Norwich Eaton Pharmaceuticals|P.O. Box 191|Norwich|New York|13815|United States", "orphan_designation": { "original_text": "Treatment of the neuroleptic malignant syndrome.", "umls": "C0027849", "parsed_text": "Neuroleptic Malignant Syndrome" } }, { "pubchem_cid": 16219293, "pubchem_sid": [ "8149931", "49977301", "50064615", "50086896", "50106202", "50224504", "96079580", "103241627", "104170142", "135022024", "135697908", "252359371", "252402840", "254759916", "313080600", "319331846", "319331847", "355037251", "376219638", "376228113", "381382564", "381394157", "383840781", "404622018", "404839553", "434131969", "440064426", "441056653", "50224503", "92303995", "126628766", "312344549", "316899266", "316966591", "318031413", "319353482", "350086829", "350095976", "374043658", "375134207", "49977300", "53789979", "144205566", "144212314", "160964552", "170464793", "315680529", "318160748", "319362120", "319362121", "322079894", "341838939", "349988026", "385663493", "386500005", "387115919", "404639670", "441114322", "85383553", "126659538", "134996352", "144075555", "315495698", "319334576", "319378575", "319378576", "438473989", "50104441", "387065024", "163564378", "255369980", "268735387", "268738067", "274885735", "319407141", "342560733", "348777476", "368494613", "375575894", "441078404" ], "inchikey": "BDZCLIOURDFSFQ-HAZZGOGXSA-N", "generic_name": "Dantrolene sodium", "designated_date": "1987-12-14", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Norwich Eaton Pharmaceuticals|P.O. Box 191|Norwich|New York|13815|United States", "orphan_designation": { "original_text": "Treatment of the neuroleptic malignant syndrome.", "umls": "C0027849", "parsed_text": "Neuroleptic Malignant Syndrome" } }, { "pubchem_cid": 9568637, "pubchem_sid": [ "8149931", "49977301", "50064615", "50086896", "50106202", "50224504", "96079580", "103241627", "104170142", "135022024", "135697908", "252359371", "252402840", "254759916", "313080600", "319331846", "319331847", "355037251", "376219638", "376228113", "381382564", "381394157", "383840781", "404622018", "404839553", "434131969", "440064426", "441056653", "50224503", "92303995", "126628766", "312344549", "316899266", "316966591", "318031413", "319353482", "350086829", "350095976", "374043658", "375134207", "49977300", "53789979", "144205566", "144212314", "160964552", "170464793", "315680529", "318160748", "319362120", "319362121", "322079894", "341838939", "349988026", "385663493", "386500005", "387115919", "404639670", "441114322", "85383553", "126659538", "134996352", "144075555", "315495698", "319334576", "319378575", "319378576", "438473989", "50104441", "387065024", "163564378", "255369980", "268735387", "268738067", "274885735", "319407141", "342560733", "348777476", "368494613", "375575894", "441078404" ], "inchikey": "LTWQNYPDAUSXBC-CDJGKPBYSA-L", "generic_name": "Dantrolene sodium", "designated_date": "1987-12-14", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Norwich Eaton Pharmaceuticals|P.O. Box 191|Norwich|New York|13815|United States", "orphan_designation": { "original_text": "Treatment of the neuroleptic malignant syndrome.", "umls": "C0027849", "parsed_text": "Neuroleptic Malignant Syndrome" } }, { "pubchem_cid": 32731, "pubchem_sid": [ "8149931", "49977301", "50064615", "50086896", "50106202", "50224504", "96079580", "103241627", "104170142", "135022024", "135697908", "252359371", "252402840", "254759916", "313080600", "319331846", "319331847", "355037251", "376219638", "376228113", "381382564", "381394157", "383840781", "404622018", "404839553", "434131969", "440064426", "441056653", "50224503", "92303995", "126628766", "312344549", "316899266", "316966591", "318031413", "319353482", "350086829", "350095976", "374043658", "375134207", "49977300", "53789979", "144205566", "144212314", "160964552", "170464793", "315680529", "318160748", "319362120", "319362121", "322079894", "341838939", "349988026", "385663493", "386500005", "387115919", "404639670", "441114322", "85383553", "126659538", "134996352", "144075555", "315495698", "319334576", "319378575", "319378576", "438473989", "50104441", "387065024", "163564378", "255369980", "268735387", "268738067", "274885735", "319407141", "342560733", "348777476", "368494613", "375575894", "441078404" ], "inchikey": "KSRLIXGNPXAZHD-UHFFFAOYSA-M", "generic_name": "Dantrolene sodium", "designated_date": "1987-12-14", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Norwich Eaton Pharmaceuticals|P.O. Box 191|Norwich|New York|13815|United States", "orphan_designation": { "original_text": "Treatment of the neuroleptic malignant syndrome.", "umls": "C0027849", "parsed_text": "Neuroleptic Malignant Syndrome" } }, { "pubchem_cid": 13271066, "pubchem_sid": [ "8149931", "49977301", "50064615", "50086896", "50106202", "50224504", "96079580", "103241627", "104170142", "135022024", "135697908", "252359371", "252402840", "254759916", "313080600", "319331846", "319331847", "355037251", "376219638", "376228113", "381382564", "381394157", "383840781", "404622018", "404839553", "434131969", "440064426", "441056653", "50224503", "92303995", "126628766", "312344549", "316899266", "316966591", "318031413", "319353482", "350086829", "350095976", "374043658", "375134207", "49977300", "53789979", "144205566", "144212314", "160964552", "170464793", "315680529", "318160748", "319362120", "319362121", "322079894", "341838939", "349988026", "385663493", "386500005", "387115919", "404639670", "441114322", "85383553", "126659538", "134996352", "144075555", "315495698", "319334576", "319378575", "319378576", "438473989", "50104441", "387065024", "163564378", "255369980", "268735387", "268738067", "274885735", "319407141", "342560733", "348777476", "368494613", "375575894", "441078404" ], "inchikey": "KSRLIXGNPXAZHD-HAZZGOGXSA-M", "generic_name": "Dantrolene sodium", "designated_date": "1987-12-14", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Norwich Eaton Pharmaceuticals|P.O. Box 191|Norwich|New York|13815|United States", "orphan_designation": { "original_text": "Treatment of the neuroleptic malignant syndrome.", "umls": "C0027849", "parsed_text": "Neuroleptic Malignant Syndrome" } }, { "pubchem_cid": 71563341, "pubchem_sid": [ "8149931", "49977301", "50064615", "50086896", "50106202", "50224504", "96079580", "103241627", "104170142", "135022024", "135697908", "252359371", "252402840", "254759916", "313080600", "319331846", "319331847", "355037251", "376219638", "376228113", "381382564", "381394157", "383840781", "404622018", "404839553", "434131969", "440064426", "441056653", "50224503", "92303995", "126628766", "312344549", "316899266", "316966591", "318031413", "319353482", "350086829", "350095976", "374043658", "375134207", "49977300", "53789979", "144205566", "144212314", "160964552", "170464793", "315680529", "318160748", "319362120", "319362121", "322079894", "341838939", "349988026", "385663493", "386500005", "387115919", "404639670", "441114322", "85383553", "126659538", "134996352", "144075555", "315495698", "319334576", "319378575", "319378576", "438473989", "50104441", "387065024", "163564378", "255369980", "268735387", "268738067", "274885735", "319407141", "342560733", "348777476", "368494613", "375575894", "441078404" ], "inchikey": "KSRLIXGNPXAZHD-DFPPEFKWSA-M", "generic_name": "Dantrolene sodium", "designated_date": "1987-12-14", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Norwich Eaton Pharmaceuticals|P.O. Box 191|Norwich|New York|13815|United States", "orphan_designation": { "original_text": "Treatment of the neuroleptic malignant syndrome.", "umls": "C0027849", "parsed_text": "Neuroleptic Malignant Syndrome" } }, { "pubchem_cid": 13271066, "pubchem_sid": [ "8149931", "49977301", "50064615", "50086896", "50106202", "50224504", "96079580", "103241627", "104170142", "135022024", "135697908", "252359371", "252402840", "254759916", "313080600", "319331846", "319331847", "355037251", "376219638", "376228113", "381382564", "381394157", "383840781", "404622018", "404839553", "434131969", "440064426", "441056653", "50224503", "92303995", "126628766", "312344549", "316899266", "316966591", "318031413", "319353482", "350086829", "350095976", "374043658", "375134207", "49977300", "53789979", "144205566", "144212314", "160964552", "170464793", "315680529", "318160748", "319362120", "319362121", "322079894", "341838939", "349988026", "385663493", "386500005", "387115919", "404639670", "441114322", "85383553", "126659538", "134996352", "144075555", "315495698", "319334576", "319378575", "319378576", "438473989", "50104441", "387065024", "163564378", "255369980", "268735387", "268738067", "274885735", "319407141", "342560733", "348777476", "368494613", "375575894", "441078404" ], "inchikey": "KSRLIXGNPXAZHD-HAZZGOGXSA-M", "generic_name": "Dantrolene sodium", "designated_date": "1987-12-14", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Norwich Eaton Pharmaceuticals|P.O. Box 191|Norwich|New York|13815|United States", "orphan_designation": { "original_text": "Treatment of the neuroleptic malignant syndrome.", "umls": "C0027849", "parsed_text": "Neuroleptic Malignant Syndrome" } } ] }, { "_id": "MONDO:0011895", "fda_orphan_drug": [ { "pubchem_cid": 59868, "pubchem_sid": [ "103554261", "135263898", "163621042", "163686371", "185974307", "274869867", "315707237", "318170845", "319101965", "319553106", "341191413", "346441121", "348524597", "355039791", "375341979", "375575625", "378132549", "381417108", "382152900", "385632648", "386625224", "387023080", "387087780", "404617075", "404772570", "405107799", "406549742", "419510994", "434251041", "438453923", "440771401", "313046726", "405254081", "405435427", "439982096", "57309747", "254762885", "376229608" ], "inchikey": "FASDKYOPVNHBLU-SSDOTTSWSA-N", "generic_name": "Dexpramipexole", "designated_date": "2019-04-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Knopp Biosciences LLC|2100 Wharton Street|Suite 615|Pittsburgh|Pennsylvania|15203|United States", "orphan_designation": { "original_text": "Treatment of hypereosinophilic syndrome (HES)", "umls": [ "C0206141", "C1540912" ], "parsed_text": [ "Hypereosinophilic syndrome", "Hypereosinophilic syndrome" ] } }, { "pubchem_cid": 123596, "pubchem_sid": [ "533344", "11528615", "12015012", "46386639", "46530584", "49681833", "49831794", "57288506", "74523529", "92717786", "99431918", "103619406", "126622001", "126653682", "127324695", "127324696", "127324697", "127324698", "127324699", "127324700", "127324701", "127324702", "127324703", "127324704", "134964410", "135078796", "135697621", "136340399", "143495161", "144205237", "152238597", "162011507", "163383485", "164202603", "165236990", "170465393", "172080548", "187072367", "196111564", "207186225", "211535627", "223367738", "223371936", "224173486", "249833369", "251915620", "251916970", "252215575", "252266054", 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O. Box 8355|1800 Concord Pike|Wilmington|Delaware|19803|United States", "orphan_designation": { "original_text": "Treatment of hypereosinophilic syndrome", "umls": "C1540912", "parsed_text": "Hypereosinophilic syndrome" } }, { "pubchem_sid": 402355846, "generic_name": "benralizumab", "designated_date": "2019-02-01", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "AstraZeneca Pharmaceuticals LP|P. O. Box 8355|1800 Concord Pike|Wilmington|Delaware|19803|United States", "orphan_designation": { "original_text": "Treatment of hypereosinophilic syndrome", "umls": "C1540912", "parsed_text": "Hypereosinophilic syndrome" } }, { "pubchem_sid": 347911271, "generic_name": "benralizumab", "designated_date": "2019-02-01", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "AstraZeneca Pharmaceuticals LP|P. O. 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Box 331|Westbury|New York|11590|United States", "orphan_designation": { "original_text": "Treatment of mitochondrial cytopathies.", "umls": "C0162670", "parsed_text": "mitochondrial cytopathies" } }, { "pubchem_cid": 3686, "pubchem_sid": [ "12012810", "26719872", "46386682", "49681642", "49957336", "56313750", "78556875", "92710538", "103060386", "103556242", "126631778", "126658121", "126665893", "135003257", "135698082", "136990616", "143492810", "144075938", "144205417", "152242914", "160871272", "162038194", "162173185", "170465611", "172080219", "187072683", "196109651", "210024006", "210280241", "210282221", "223377118", "223441547", "223701090", "249866551", "250133395", "251960540", "252156937", "252218535", "252350841", "252448661", "253663492", "254761339", "255474572", "268736344", "274896695", "275956092", "277309071", "310275130", "312692157", "312695136", "313076962", "313081144", "315678276", "316962814", "318045949", "318154402", "318161318", "318166205", "318240733", 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Hammerstrasse|Liestal|Basel-Landschaft|Switzerland", "orphan_designation": { "original_text": "Treatment of mitochondrial myopathy, encephalopathy, lactic acidosis with stroke-like episodes syndrome (MELAS)", "umls": [ "C0001125", "C0039082", "C0085584", "C0162670", "C0162671", "C1857287" ], "parsed_text": [ "LACTIC ACIDOSIS", "Syndrome", "Encephalopathy", "MITOCHONDRIAL MYOPATHY", "MELAS", "Stroke-like episodes" ] } }, { "pubchem_cid": 10917, "pubchem_sid": [ "3612", "841764", "8145671", "46505864", "48416167", "49867038", "53788226", "77300350", "85165027", "91146345", "93166761", "103332887", "103944816", "134975930", "144075870", "160963928", "241079878", "249738337", "252359342", "268735121", "312694995", "315673807", "318160504", "319069091", "340508211", "347912969", "348274226", "348316454", "349991268", "349995052", "355038682", "375134178", "376226610", "387061010", "387138300", "404618973", "433780388", "434132152", "438644624", "440064641", "10321817", "77300349", "310269393", "387196303", "387209312", "434298347", "438460199", "438473460", "438473461", "77300351", "77300352", "77300353", "77300354", "77300355" ], "inchikey": "PHIQHXFUZVPYII-ZCFIWIBFSA-N", "generic_name": "Levocarnitine", "designated_date": "1997-04-07", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Leadiant Biosciences, Inc.|9841 Washingtonian Boulevard|Suite 500|Gaithersburg|Maryland|20878|United States", "orphan_designation": { "original_text": "Treatment of zidovudine-induced mitochondrial myopathy.", "umls": "C0162670", "parsed_text": "MITOCHONDRIAL MYOPATHY" } } ] }, { "_id": "UMLS:C0030200", "fda_orphan_drug": [ { "pubchem_cid": 53395169, "pubchem_sid": [ "160671112", "162173271", "241110367", "252359482", "319360808", "341117487", "341832813", "349159527", "49962762", "126665099", "316966743", "341236623", "349978209", "426222646", "85335660", "135029501", "318160943", "423379501", "274629388", "318042908", "322079912", "340470684", "342577569", "364208222", "404771269", "406167832", "406852978" ], "inchikey": "JYSJVJJVLNYRKL-UHFFFAOYSA-N", "generic_name": "Elcatonin", "designated_date": "1995-09-25", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Innapharma, Inc.|10 Mountainview Road|Upper Saddle River|New Jersey|7458|United States", "orphan_designation": { "original_text": "Intrathecal treatment of intractable pain.", "umls": "C0030200", "parsed_text": "intractable pain" } }, { "pubchem_cid": 134688669, "pubchem_sid": [ "53790944", "53801085", "74974821", "103949314", "135062055", "223440846", "241182163", "242084271", "273942988", "319308454", "341146393", "349133261", "349980118", "381323662", "384443082", "385782409", "386619163", "387086177", "404615268", "404770295", "434304737", "438487828", "7980510", "49688359", "49878789", "74974822", "74974823", "241100095", "310279643", "316539718", "319137114", "319311872", "341106292", "347660387", "355043403", "387086176", "74974819", "85089156", "255386135", "319294806", "381975343", "11371", "74974820", "318155738", "382162670", "386297926", "415966910", "103592451", "160845722", "162223540", "319403390", "74974818", "162228033", "313369291", "342599926", "363682317", "375131204", "384404901", "384404902", "384404903", "384404904", "384404905", "384404906", "438534956" ], "inchikey": "DSDNAKHZNJAGHN-WLJYXNHNSA-N", "generic_name": "resiniferatoxin", "designated_date": "2003-05-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Sorrento Theraeputics, Inc.|9380 Judicial Drive|San Diego|California|92121|United States", "orphan_designation": { "original_text": "Treatment of intractable pain at end-stage disease", "umls": "C0030200", "parsed_text": "intractable pain" } }, { "pubchem_cid": 44564218, "pubchem_sid": [ "53790944", "53801085", "74974821", "103949314", "135062055", "223440846", "241182163", "242084271", "273942988", "319308454", "341146393", "349133261", "349980118", "381323662", "384443082", "385782409", "386619163", "387086177", "404615268", "404770295", "434304737", "438487828", "7980510", "49688359", "49878789", "74974822", "74974823", "241100095", "310279643", "316539718", "319137114", "319311872", "341106292", "347660387", "355043403", "387086176", "74974819", "85089156", "255386135", "319294806", "381975343", "11371", "74974820", "318155738", "382162670", "386297926", "415966910", "103592451", "160845722", "162223540", "319403390", "74974818", "162228033", "313369291", "342599926", "363682317", "375131204", "384404901", "384404902", "384404903", "384404904", "384404905", "384404906", "438534956" ], "inchikey": "DSDNAKHZNJAGHN-GGIHZKEUSA-N", "generic_name": "resiniferatoxin", "designated_date": "2003-05-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Sorrento Theraeputics, Inc.|9380 Judicial Drive|San Diego|California|92121|United States", "orphan_designation": { "original_text": "Treatment of intractable pain at end-stage disease", "umls": "C0030200", "parsed_text": "intractable pain" } }, { "pubchem_cid": 2733497, "pubchem_sid": [ "53790944", "53801085", "74974821", "103949314", "135062055", "223440846", "241182163", "242084271", "273942988", "319308454", "341146393", "349133261", "349980118", "381323662", "384443082", "385782409", "386619163", "387086177", "404615268", "404770295", "434304737", "438487828", "7980510", "49688359", "49878789", "74974822", "74974823", "241100095", "310279643", "316539718", "319137114", "319311872", "341106292", "347660387", "355043403", "387086176", "74974819", "85089156", "255386135", "319294806", "381975343", "11371", "74974820", "318155738", "382162670", "386297926", "415966910", "103592451", "160845722", "162223540", "319403390", "74974818", "162228033", "313369291", "342599926", "363682317", "375131204", "384404901", "384404902", "384404903", "384404904", "384404905", "384404906", "438534956" ], "inchikey": "DSDNAKHZNJAGHN-YOHYOAFPSA-N", "generic_name": "resiniferatoxin", "designated_date": "2003-05-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Sorrento Theraeputics, Inc.|9380 Judicial Drive|San Diego|California|92121|United States", "orphan_designation": { "original_text": "Treatment of intractable pain at end-stage disease", "umls": "C0030200", "parsed_text": "intractable pain" } }, { "pubchem_cid": 5148108, "pubchem_sid": [ "53790944", "53801085", "74974821", "103949314", "135062055", "223440846", "241182163", "242084271", "273942988", "319308454", "341146393", "349133261", "349980118", "381323662", "384443082", "385782409", "386619163", "387086177", "404615268", "404770295", "434304737", "438487828", "7980510", "49688359", "49878789", "74974822", "74974823", "241100095", "310279643", "316539718", "319137114", "319311872", "341106292", "347660387", "355043403", "387086176", "74974819", "85089156", "255386135", "319294806", "381975343", "11371", "74974820", "318155738", "382162670", "386297926", "415966910", "103592451", "160845722", "162223540", "319403390", "74974818", "162228033", "313369291", "342599926", "363682317", "375131204", "384404901", "384404902", "384404903", "384404904", "384404905", "384404906", "438534956" ], "inchikey": "DSDNAKHZNJAGHN-UHFFFAOYSA-N", "generic_name": "resiniferatoxin", "designated_date": "2003-05-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Sorrento Theraeputics, Inc.|9380 Judicial Drive|San Diego|California|92121|United States", "orphan_designation": { "original_text": "Treatment of intractable pain at end-stage disease", "umls": "C0030200", "parsed_text": "intractable pain" } }, { "pubchem_cid": 6324660, "pubchem_sid": [ "53790944", "53801085", "74974821", "103949314", "135062055", "223440846", "241182163", "242084271", "273942988", "319308454", "341146393", "349133261", "349980118", "381323662", "384443082", "385782409", "386619163", "387086177", "404615268", "404770295", "434304737", "438487828", "7980510", "49688359", "49878789", "74974822", "74974823", "241100095", "310279643", "316539718", "319137114", "319311872", "341106292", "347660387", "355043403", "387086176", "74974819", "85089156", "255386135", "319294806", "381975343", "11371", "74974820", "318155738", "382162670", "386297926", "415966910", "103592451", "160845722", "162223540", "319403390", "74974818", "162228033", "313369291", "342599926", "363682317", "375131204", "384404901", "384404902", "384404903", "384404904", "384404905", "384404906", "438534956" ], "inchikey": "DSDNAKHZNJAGHN-VQBCBPMFSA-N", "generic_name": "resiniferatoxin", "designated_date": "2003-05-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Sorrento Theraeputics, Inc.|9380 Judicial Drive|San Diego|California|92121|United States", "orphan_designation": { "original_text": "Treatment of intractable pain at end-stage disease", "umls": "C0030200", "parsed_text": "intractable pain" } }, { "pubchem_cid": 104826, "pubchem_sid": [ "53790944", "53801085", "74974821", "103949314", "135062055", "223440846", "241182163", "242084271", "273942988", "319308454", "341146393", "349133261", "349980118", "381323662", "384443082", "385782409", "386619163", "387086177", "404615268", "404770295", "434304737", "438487828", "7980510", "49688359", "49878789", "74974822", "74974823", "241100095", "310279643", "316539718", "319137114", "319311872", "341106292", "347660387", "355043403", "387086176", "74974819", "85089156", "255386135", "319294806", "381975343", "11371", "74974820", "318155738", "382162670", "386297926", "415966910", "103592451", "160845722", "162223540", "319403390", "74974818", "162228033", "313369291", "342599926", "363682317", "375131204", "384404901", "384404902", "384404903", "384404904", "384404905", "384404906", "438534956" ], "inchikey": "DSDNAKHZNJAGHN-IHCAYWNCSA-N", "generic_name": "resiniferatoxin", "designated_date": "2003-05-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Sorrento Theraeputics, Inc.|9380 Judicial Drive|San Diego|California|92121|United States", "orphan_designation": { "original_text": "Treatment of intractable pain at end-stage disease", "umls": "C0030200", "parsed_text": "intractable pain" } }, { "pubchem_cid": 5702546, "pubchem_sid": [ "53790944", "53801085", "74974821", "103949314", "135062055", "223440846", "241182163", "242084271", "273942988", "319308454", "341146393", "349133261", "349980118", "381323662", "384443082", "385782409", "386619163", "387086177", "404615268", "404770295", "434304737", "438487828", "7980510", "49688359", "49878789", "74974822", "74974823", "241100095", "310279643", "316539718", "319137114", "319311872", "341106292", "347660387", "355043403", "387086176", "74974819", "85089156", "255386135", "319294806", "381975343", "11371", "74974820", "318155738", "382162670", "386297926", "415966910", "103592451", "160845722", "162223540", "319403390", "74974818", "162228033", "313369291", "342599926", "363682317", "375131204", "384404901", "384404902", "384404903", "384404904", "384404905", "384404906", "438534956" ], "inchikey": "DSDNAKHZNJAGHN-MXTYGGKSSA-N", "generic_name": "resiniferatoxin", "designated_date": "2003-05-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Sorrento Theraeputics, Inc.|9380 Judicial Drive|San Diego|California|92121|United States", "orphan_designation": { "original_text": "Treatment of intractable pain at end-stage disease", "umls": "C0030200", "parsed_text": "intractable pain" } }, { "pubchem_cid": 5284604, "pubchem_sid": [ "10219", "81989", "841905", "46505296", "48416368", "50523562", "103250876", "103916122", "126629458", "134223676", "134971543", "160964525", "162224633", "178103672", "210279774", "210282097", "223440572", "223822210", "252355643", "274086604", "312228703", "313081792", "315673979", "318161882", "319297069", "340514712", "340514713", "341145304", "348283771", "349301950", "349976884", "363669561", "382164649", "385785143", "386622084", "387161065", "419493221", "433778918", "439388693", "440790873", "50523561", "250115866", "387161066", "387196442", "387209500", "49993133", "50523560", "117478587", "164232052", "342565921", "441078824" ], "inchikey": "UQCNKQCJZOAFTQ-ISWURRPUSA-N", "generic_name": "Oxymorphone", "designated_date": "1985-03-19", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "DuPont Merck Pharmaceutical Company|P.O. Box 80027|Wilmington|Delaware|19880|United States", "orphan_designation": { "original_text": "For relief of severe intractable pain in narcotic-tolerant patients.", "umls": "C0030200", "parsed_text": "intractable pain" } } ] }, { "_id": "UMLS:C1412063", "fda_orphan_drug": [ { "pubchem_cid": 25221720, "pubchem_sid": [ "160698340", "163643180", "315711258", "319382873", "329727336", "329727466", "341138483", "341662032", "347828823", "355036683", "378132327", "385649777", "386486216", "439428492", "439556358", "439576675", "335987508", "375758325" ], "inchikey": "WJIGGYYSZBWCGC-MRXNPFEDSA-N", "generic_name": "emixustat", "designated_date": "2017-01-04", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Acucela Inc.|1301 2nd Avenue|Suite 4200|Seattle|Washington|98101|United States", "orphan_designation": { "original_text": "Treatment of Stargardt disease (STGD)", "umls": [ "C1412063", "C1855465" ], "parsed_text": [ "Stargardt disease", "STGD" ] } }, { "pubchem_cid": 213054, "pubchem_sid": [ "53013250", "135188011", "160681924", "341678923", "347242797", "347831669", "347832078", "349991547", "355036220", "385738106", "386574734", "439364272", "405476274", "440022933", "53013247", "53013248", "53013249", "275292501" ], "inchikey": "PWILYDZRJORZDR-MISYRCLQSA-N", "generic_name": "soraprazan", "designated_date": "2017-12-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Katairo GmbH|21 Lederstra\u07e5|Kusterdingen|Baden-Wrttemberg|Germany", "orphan_designation": { "original_text": "Treatment of Stargardt Disease (StD)", "umls": [ "C0036916", "C1412063" ], "parsed_text": [ "STD", "Stargardt disease" ] } } ] }, { "_id": "MONDO:0019353", "fda_orphan_drug": [ { "pubchem_cid": 25221720, "pubchem_sid": [ "160698340", "163643180", "315711258", "319382873", "329727336", "329727466", "341138483", "341662032", "347828823", "355036683", "378132327", "385649777", "386486216", "439428492", "439556358", "439576675", "335987508", "375758325" ], "inchikey": "WJIGGYYSZBWCGC-MRXNPFEDSA-N", "generic_name": "emixustat", "designated_date": "2017-01-04", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Acucela Inc.|1301 2nd Avenue|Suite 4200|Seattle|Washington|98101|United States", "orphan_designation": { "original_text": "Treatment of Stargardt disease (STGD)", "umls": [ "C1412063", "C1855465" ], "parsed_text": [ "Stargardt disease", "STGD" ] } } ] }, { "_id": "MONDO:0005878", "fda_orphan_drug": [ { "pubchem_cid": 6918632, "pubchem_sid": [ "12015634", "124489064", "135263175", "160703346", "160778542", "162224783", "223444021", "275120955", "315726240", "319332960", "341424257", "385641818", "386478089", "404620748", "404770626", "419501487", "434250837", "438455164", "439572168", "440703890", "335988606", "335988854", "341138471", "355048706", "433909670", "375566397", "387269353", "405458359", "440005021" ], "inchikey": "ZMQMTKVVAMWKNY-YSXLEBCMSA-N", "generic_name": "emodepside", "designated_date": "2016-09-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bayer HealthCare Pharmaceuticals, Inc.|100 Bayer Boulevard|P. O. Box 915|Whippany|New Jersey|7981|United States", "orphan_designation": { "original_text": "Treatment of onchocerhiasis (\"river blindness\") caused by the filarial nematode onchocerca volvulus", "umls": "C0029002", "parsed_text": "River Blindness" } } ] }, { "_id": "UMLS:C1140680", "fda_orphan_drug": [ { "pubchem_cid": 135565889, "pubchem_sid": [ "347828157", "381370483", "385737949", "386574576", "404650926", "162169437", "319349222", "349796666", "319537886" ], "inchikey": "YZSIZVRFVQKMJU-RDGPPVDQSA-N", "generic_name": "etarfolatide", "designated_date": "2000-02-16", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Endocyte, Inc.|3000 Kent Ave.|Suite A1-100|West Lafayette|Indiana|47906|United States", "orphan_designation": { "original_text": "For the identification of ovarian carcinomas", "umls": "C1140680", "parsed_text": "ovarian carcinomas" } } ] }, { "_id": "MONDO:0016693", "fda_orphan_drug": [ { "pubchem_cid": 6442177, 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"orphan_designation": { "original_text": "Amelioration of recurrent attacks of acute intermittent porphyria (AIP) temporarily related to the menstrual cycle in susceptible women and similar symptoms which occur in other patients with AIP, porphyria variegata and hereditary coproporphyria.", "umls": [ "C0162531", "C0162532", "C0162565", "C1279945", "C1457887" ], "parsed_text": [ "Hereditary Coproporphyria", "Porphyria Variegata", "Acute intermittent porphyria", "AIP", "Symptoms" ] } }, { "pubchem_sid": 406530486, "generic_name": "Hemin", "trade_name": "Panhematin", "designated_date": "1984-03-16", "designation_status": "Designated/Approved", "marketing_approval_date": "1983-07-20", "exclusivity_end_date": "1990-07-20", "sponsor": "Abbott Laboratories|Diagnostics Division|Abbott Park|Illinois|60064|United States", "orphan_designation": { "original_text": "Amelioration of recurrent attacks of acute intermittent porphyria (AIP) temporarily related to the menstrual cycle in susceptible 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"C1279945", "C1457887" ], "parsed_text": [ "Hereditary Coproporphyria", "Porphyria Variegata", "Acute intermittent porphyria", "AIP", "Symptoms" ] } }, { "pubchem_sid": 404847608, "generic_name": "Hemin", "trade_name": "Panhematin", "designated_date": "1984-03-16", "designation_status": "Designated/Approved", "marketing_approval_date": "1983-07-20", "exclusivity_end_date": "1990-07-20", "sponsor": "Abbott Laboratories|Diagnostics Division|Abbott Park|Illinois|60064|United States", "orphan_designation": { "original_text": "Amelioration of recurrent attacks of acute intermittent porphyria (AIP) temporarily related to the menstrual cycle in susceptible women and similar symptoms which occur in other patients with AIP, porphyria variegata and hereditary coproporphyria.", "umls": [ "C0162531", "C0162532", "C0162565", "C1279945", "C1457887" ], "parsed_text": [ "Hereditary Coproporphyria", "Porphyria Variegata", "Acute intermittent porphyria", "AIP", "Symptoms" ] } }, { "pubchem_sid": 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"parsed_text": [ "Hereditary Coproporphyria", "Porphyria Variegata", "Acute intermittent porphyria", "AIP", "Symptoms" ] } }, { "pubchem_sid": 376126324, "generic_name": "Hemin", "trade_name": "Panhematin", "designated_date": "1984-03-16", "designation_status": "Designated/Approved", "marketing_approval_date": "1983-07-20", "exclusivity_end_date": "1990-07-20", "sponsor": "Abbott Laboratories|Diagnostics Division|Abbott Park|Illinois|60064|United States", "orphan_designation": { "original_text": "Amelioration of recurrent attacks of acute intermittent porphyria (AIP) temporarily related to the menstrual cycle in susceptible women and similar symptoms which occur in other patients with AIP, porphyria variegata and hereditary coproporphyria.", "umls": [ "C0162531", "C0162532", "C0162565", "C1279945", "C1457887" ], "parsed_text": [ "Hereditary Coproporphyria", "Porphyria Variegata", "Acute intermittent porphyria", "AIP", "Symptoms" ] } }, { "pubchem_sid": 375682001, "generic_name": "Hemin", "trade_name": "Panhematin", "designated_date": "1984-03-16", "designation_status": "Designated/Approved", "marketing_approval_date": "1983-07-20", "exclusivity_end_date": "1990-07-20", "sponsor": "Abbott Laboratories|Diagnostics Division|Abbott Park|Illinois|60064|United States", "orphan_designation": { "original_text": "Amelioration of recurrent attacks of acute intermittent porphyria (AIP) temporarily related to the menstrual cycle in susceptible women and similar symptoms which occur in other patients with AIP, porphyria variegata and hereditary coproporphyria.", "umls": [ "C0162531", "C0162532", "C0162565", "C1279945", "C1457887" ], "parsed_text": [ "Hereditary Coproporphyria", "Porphyria Variegata", "Acute intermittent porphyria", "AIP", "Symptoms" ] } }, { "pubchem_sid": 375568326, "generic_name": "Hemin", "trade_name": "Panhematin", "designated_date": "1984-03-16", "designation_status": "Designated/Approved", "marketing_approval_date": "1983-07-20", 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"orphan_designation": { "original_text": "Amelioration of recurrent attacks of acute intermittent porphyria (AIP) temporarily related to the menstrual cycle in susceptible women and similar symptoms which occur in other patients with AIP, porphyria variegata and hereditary coproporphyria.", "umls": [ "C0162531", "C0162532", "C0162565", "C1279945", "C1457887" ], "parsed_text": [ "Hereditary Coproporphyria", "Porphyria Variegata", "Acute intermittent porphyria", "AIP", "Symptoms" ] } }, { "pubchem_sid": 49969817, "generic_name": "Hemin", "trade_name": "Panhematin", "designated_date": "1984-03-16", "designation_status": "Designated/Approved", "marketing_approval_date": "1983-07-20", "exclusivity_end_date": "1990-07-20", "sponsor": "Abbott Laboratories|Diagnostics Division|Abbott Park|Illinois|60064|United States", "orphan_designation": { "original_text": "Amelioration of recurrent attacks of acute intermittent porphyria (AIP) temporarily related to the menstrual cycle in susceptible women 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variegata and hereditary coproporphyria.", "umls": [ "C0162531", "C0162532", "C0162565", "C1279945", "C1457887" ], "parsed_text": [ "Hereditary Coproporphyria", "Porphyria Variegata", "Acute intermittent porphyria", "AIP", "Symptoms" ] } }, { "pubchem_sid": 127294174, "generic_name": "Hemin", "trade_name": "Panhematin", "designated_date": "1984-03-16", "designation_status": "Designated/Approved", "marketing_approval_date": "1983-07-20", "exclusivity_end_date": "1990-07-20", "sponsor": "Abbott Laboratories|Diagnostics Division|Abbott Park|Illinois|60064|United States", "orphan_designation": { "original_text": "Amelioration of recurrent attacks of acute intermittent porphyria (AIP) temporarily related to the menstrual cycle in susceptible women and similar symptoms which occur in other patients with AIP, porphyria variegata and hereditary coproporphyria.", "umls": [ "C0162531", "C0162532", "C0162565", "C1279945", "C1457887" ], "parsed_text": [ "Hereditary Coproporphyria", "Porphyria 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"1984-03-16", "designation_status": "Designated/Approved", "marketing_approval_date": "1983-07-20", "exclusivity_end_date": "1990-07-20", "sponsor": "Abbott Laboratories|Diagnostics Division|Abbott Park|Illinois|60064|United States", "orphan_designation": { "original_text": "Amelioration of recurrent attacks of acute intermittent porphyria (AIP) temporarily related to the menstrual cycle in susceptible women and similar symptoms which occur in other patients with AIP, porphyria variegata and hereditary coproporphyria.", "umls": [ "C0162531", "C0162532", "C0162565", "C1279945", "C1457887" ], "parsed_text": [ "Hereditary Coproporphyria", "Porphyria Variegata", "Acute intermittent porphyria", "AIP", "Symptoms" ] } }, { "pubchem_sid": 438459028, "generic_name": "Hemin", "trade_name": "Panhematin", "designated_date": "1984-03-16", "designation_status": "Designated/Approved", "marketing_approval_date": "1983-07-20", "exclusivity_end_date": "1990-07-20", "sponsor": "Abbott Laboratories|Diagnostics Division|Abbott Park|Illinois|60064|United States", "orphan_designation": { "original_text": "Amelioration of recurrent attacks of acute intermittent porphyria (AIP) temporarily related to the menstrual cycle in susceptible women and similar symptoms which occur in other patients with AIP, porphyria variegata and hereditary coproporphyria.", "umls": [ "C0162531", "C0162532", "C0162565", "C1279945", "C1457887" ], "parsed_text": [ "Hereditary Coproporphyria", "Porphyria Variegata", "Acute intermittent porphyria", "AIP", "Symptoms" ] } }, { "pubchem_sid": 434252965, "generic_name": "Hemin", "trade_name": "Panhematin", "designated_date": "1984-03-16", "designation_status": "Designated/Approved", "marketing_approval_date": "1983-07-20", "exclusivity_end_date": "1990-07-20", "sponsor": "Abbott Laboratories|Diagnostics Division|Abbott Park|Illinois|60064|United States", "orphan_designation": { "original_text": "Amelioration of recurrent attacks of acute intermittent porphyria (AIP) temporarily related to the menstrual cycle in susceptible women and similar symptoms which occur in other patients with AIP, porphyria variegata and hereditary coproporphyria.", "umls": [ "C0162531", "C0162532", "C0162565", "C1279945", "C1457887" ], "parsed_text": [ "Hereditary Coproporphyria", "Porphyria Variegata", "Acute intermittent porphyria", "AIP", "Symptoms" ] } }, { "pubchem_sid": 433705116, "generic_name": "Hemin", "trade_name": "Panhematin", "designated_date": "1984-03-16", "designation_status": "Designated/Approved", "marketing_approval_date": "1983-07-20", "exclusivity_end_date": "1990-07-20", "sponsor": "Abbott Laboratories|Diagnostics Division|Abbott Park|Illinois|60064|United States", "orphan_designation": { "original_text": "Amelioration of recurrent attacks of acute intermittent porphyria (AIP) temporarily related to the menstrual cycle in susceptible women and similar symptoms which occur in other patients with AIP, porphyria variegata and hereditary coproporphyria.", "umls": [ "C0162531", "C0162532", "C0162565", "C1279945", "C1457887" ], "parsed_text": [ "Hereditary Coproporphyria", "Porphyria Variegata", "Acute intermittent porphyria", "AIP", "Symptoms" ] } }, { "pubchem_sid": 405109240, "generic_name": "Hemin", "trade_name": "Panhematin", "designated_date": "1984-03-16", "designation_status": "Designated/Approved", "marketing_approval_date": "1983-07-20", "exclusivity_end_date": "1990-07-20", "sponsor": "Abbott Laboratories|Diagnostics Division|Abbott Park|Illinois|60064|United States", "orphan_designation": { "original_text": "Amelioration of recurrent attacks of acute intermittent porphyria (AIP) temporarily related to the menstrual cycle in susceptible women and similar symptoms which occur in other patients with AIP, porphyria variegata and hereditary coproporphyria.", "umls": [ "C0162531", "C0162532", "C0162565", "C1279945", "C1457887" ], "parsed_text": [ "Hereditary Coproporphyria", "Porphyria Variegata", "Acute intermittent porphyria", "AIP", "Symptoms" ] } }, { "pubchem_sid": 404620137, "generic_name": "Hemin", "trade_name": "Panhematin", "designated_date": "1984-03-16", "designation_status": "Designated/Approved", "marketing_approval_date": "1983-07-20", "exclusivity_end_date": "1990-07-20", "sponsor": "Abbott Laboratories|Diagnostics Division|Abbott Park|Illinois|60064|United States", "orphan_designation": { "original_text": "Amelioration of recurrent attacks of acute intermittent porphyria (AIP) temporarily related to the menstrual cycle in susceptible women and similar symptoms which occur in other patients with AIP, porphyria variegata and hereditary coproporphyria.", "umls": [ "C0162531", "C0162532", "C0162565", "C1279945", "C1457887" ], "parsed_text": [ "Hereditary Coproporphyria", "Porphyria Variegata", "Acute intermittent porphyria", "AIP", "Symptoms" ] } }, { "pubchem_sid": 387076540, "generic_name": "Hemin", "trade_name": "Panhematin", "designated_date": "1984-03-16", 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coproporphyria.", "umls": [ "C0162531", "C0162532", "C0162565", "C1279945", "C1457887" ], "parsed_text": [ "Hereditary Coproporphyria", "Porphyria Variegata", "Acute intermittent porphyria", "AIP", "Symptoms" ] } }, { "pubchem_sid": 319359663, "generic_name": "Hemin", "trade_name": "Panhematin", "designated_date": "1984-03-16", "designation_status": "Designated/Approved", "marketing_approval_date": "1983-07-20", "exclusivity_end_date": "1990-07-20", "sponsor": "Abbott Laboratories|Diagnostics Division|Abbott Park|Illinois|60064|United States", "orphan_designation": { "original_text": "Amelioration of recurrent attacks of acute intermittent porphyria (AIP) temporarily related to the menstrual cycle in susceptible women and similar symptoms which occur in other patients with AIP, porphyria variegata and hereditary coproporphyria.", "umls": [ "C0162531", "C0162532", "C0162565", "C1279945", "C1457887" ], "parsed_text": [ "Hereditary Coproporphyria", "Porphyria Variegata", "Acute intermittent porphyria", "AIP", "Symptoms" ] } }, { "pubchem_sid": 319225729, "generic_name": "Hemin", "trade_name": "Panhematin", "designated_date": "1984-03-16", "designation_status": "Designated/Approved", "marketing_approval_date": "1983-07-20", "exclusivity_end_date": "1990-07-20", "sponsor": "Abbott Laboratories|Diagnostics Division|Abbott Park|Illinois|60064|United States", "orphan_designation": { "original_text": "Amelioration of recurrent attacks of acute intermittent porphyria (AIP) temporarily related to the menstrual cycle in susceptible women and similar symptoms which occur in other patients with AIP, porphyria variegata and hereditary coproporphyria.", "umls": [ "C0162531", "C0162532", "C0162565", "C1279945", "C1457887" ], "parsed_text": [ "Hereditary Coproporphyria", "Porphyria Variegata", "Acute intermittent porphyria", "AIP", "Symptoms" ] } }, { "pubchem_sid": 87570469, "generic_name": "Hemin", "trade_name": "Panhematin", "designated_date": "1984-03-16", 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"sponsor": "CSL Behring L.L.C.|1020 First Ave.|PO Box 61501|King of Prussia|Pennsylvania|19406|United States", "orphan_designation": { "original_text": "For slowing the progression of emphysema in alpha1-antitrypsin deficient patients.", "umls": [ "C0034067", "C0242656" ], "parsed_text": [ "Emphysema", "Progression" ] } } ] }, { "_id": "MONDO:0013282", "fda_orphan_drug": [ { "pubchem_cid": 24847767, "pubchem_sid": [ "92711079", "126631224", "126657602", "144206148", "196110024", "249716772", "318241043", "440701464", "124965876", "162176361", "254787121", "310274713", "341139334", "375637121", "376010541", "385782869", "386619650", "387114074", "438651501", "53790595", "178101652", "252215555", "316962692", "126673760", "223434885", "310264899", "599262", "50065366", "51273928", "381970662", "382370671", "386289793", "405509800", "406543781", "439649926", "440056456", "3801", "841303", "56352864", "57393329", "124499697", "134989201", "176259447", "242549671", "255371160", "312226369", 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O. 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"144211711", "160791995", "162241433", "162278510", "162760662", "163808839", "176263557", "204360026", "251866151", "252156763", "252308341", "274954425", "315677688", "316404242", "332869019", "340124157", "341172422", "346549223", "354330009", "355045761", "355202580", "363905647", "374059876", "381385079", "382158424", "384257116", "385738493", "386575122", "387121130", "404571935", "404575260", "404619012", "434311840", "438490420", "438656970", "440974818", "441124748", "322095394", "341835343", "348903889", "53788498", "318160826", "91147426", "441082593" ], "inchikey": "LUPNKHXLFSSUGS-UHFFFAOYSA-M", "generic_name": "Sodium dichloroacetate", "designated_date": "1997-12-31", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Questcor Pharmaceuticals, Inc.|3260 Whipple Rd.|Union City|California|94587|United States", "orphan_designation": { "original_text": "Treatment of congenital lactic acidosis.", "umls": 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Box 100277|Gainesville|Florida|32610|United States", "orphan_designation": { "original_text": "Treatment of congenital lactic acidosis", "umls": "C4025276", "parsed_text": "Congenital lactic acidosis" } } ] }, { "_id": "MONDO:0002474", "fda_orphan_drug": [ { "pubchem_cid": 5311454, "pubchem_sid": [ "7980667", "12012842", "26719721", "46386912", "49681794", "50003116", "50296460", "53787806", "126687172", "135001137", "144207171", "160682657", "162248724", "172080465", "178102119", "223434582", "250056868", "251916521", "251917868", "252356786", "275388690", "277306975", "277331022", "310265035", "310280302", "315684210", "318019958", "318168574", "329882812", "341105466", "341143036", "341849023", "347831766", "347832176", "349977259", "350087314", "355036190", "374086099", "375578773", "382161577", "382370941", "384249379", "385782972", "386619753", "387102705", "387102706", "404639474", "406530907", "406867580", "409311006", "434310878", "438475273", "438489325", "438604631", 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"approved_labeled_indication": "Treatment of nighttime sleep disturbances in Smith-Magenis Syndrome (SMS) in pediatric patients 3 to 15 years of age", "marketing_approval_date": "2020-12-01", "exclusivity_end_date": "2027-12-01", "exclusivity_protected_indication": "Treatment of nighttime sleep disturbances in Smith-Magenis Syndrome (SMS) in pediatric patients 3 to 15 years of age", "sponsor": "Vanda Pharmaceuticals, Inc.|9605 Medical Center Drive|Rockville|Maryland|20850|United States", "orphan_designation": { "original_text": "Treatment of the sleep disorder in Smith-Magenis syndrome associated with diurnal melatonin secretion.", "umls": [ "C0795864", "C0851578" ], "parsed_text": [ "Smith-Magenis syndrome", "Sleep disorder" ] } } ] }, { "_id": "MONDO:0004981", "fda_orphan_drug": [ { "pubchem_cid": 54718618, "pubchem_sid": [ "135262852", "160681942", "275049376", "315795994", "319400650", "341430450", "347260904", "347828992", "349982069", "355427891", "375659975", "378134571", 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"1989-12-13", "exclusivity_end_date": "1996-12-13", "sponsor": "Pharmacia & Upjohn AB|Lindhagensgatan 133|Stockholm|Sweden", "orphan_designation": { "original_text": "For the treatment of patients with hereditary antithrombin III deficiency in connection with surgical or obstetrical procedures or when they suffer from thromboembolism.", "umls": [ "C0040038", "C2586031" ], "parsed_text": [ "Thromboembolism", "Hereditary antithrombin III deficiency" ] } }, { "pubchem_sid": 135346995, "generic_name": "Antithrombin III (human)", "trade_name": "ATnativ", "designated_date": "1985-02-08", "designation_status": "Designated/Approved", "marketing_approval_date": "1989-12-13", "exclusivity_end_date": "1996-12-13", "sponsor": "Pharmacia & Upjohn AB|Lindhagensgatan 133|Stockholm|Sweden", "orphan_designation": { "original_text": "For the treatment of patients with hereditary antithrombin III deficiency in connection with surgical or obstetrical procedures or when they suffer from thromboembolism.", "umls": [ "C0040038", "C2586031" ], "parsed_text": [ "Thromboembolism", "Hereditary antithrombin III deficiency" ] } } ] }, { "_id": "UMLS:C0206580", "fda_orphan_drug": [ { "pubchem_cid": 16124688, "pubchem_sid": [ "57304412", "135195316", "163645737", "252225037", "254793603", "254803431", "312322470", "318154843", "319360010", "341857729", "347828336", "349982440", "374263507", "382165751", "385638403", "386632597", "426222546", "438447936", "439573622", "375997097", "386635866", "405507156", "419497989", "434311946", "438490873", "440053812", "126679961", "316906184", "342562550", "349333842" ], "inchikey": "CSKDFZIMJXRJGH-VWLPUNTISA-N", "generic_name": "tecovirimat", "designated_date": "2010-09-29", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "SIGA Technologies, Inc.|4575 SW Research Way|Corvallis|Oregon|97333|United States", "orphan_designation": { "original_text": "Treatment of orthopoxvirus infections.", "umls": [ 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Box 4914|7 Powder Horn Srive|Warren|New Jersey|7059|United States", "orphan_designation": { "original_text": "Treatment of the clinical manifestations of mycobacterial infection caused by Mycobacterium tuberculosis and non-tuberculous mycobacteria.", "umls": [ "C0026918", "C0041296", "C1265234" ], "parsed_text": [ "Mycobacterial infection", "Infection caused by Mycobacterium tuberculosis", "Non-Tuberculous Mycobacteria" ] } } ] }, { "_id": "UMLS:C0231170", "fda_orphan_drug": [ { "pubchem_cid": 6167, "pubchem_sid": [ "9794", "67636", "596079", "8136890", "8149269", "10321652", "12012587", "17389095", "17389096", "29203973", "48423165", "48424983", "48424984", "49718107", "49854497", "50085974", "50104821", "50104822", "53788545", "56310804", "56313821", "56463595", "87565511", "92303527", "103177124", "103914140", "103926740", "126590037", "127310169", "127310170", "127310171", "127310172", "127310173", "127310174", "127310175", "127310176", "127310177", "134972467", "135650118", "135697921", "144074972", "144204498", "144208745", "144210899", "160964667", "163564264", "163614540", "163621028", "163686358", "170465011", "174530179", "185995519", "223933846", "250134562", "252151491", "252159234", "252216674", "252306537", "252359151", "268734454", "274950639", "275133753", "277309149", "312343609", "312344435", "312616065", "315444479", "315674752", "316395119", "316542915", "317227658", "318152237", "318160703", "318465192", "319064177", "319553643", "322071378", "329585673", "340126148", "340516094", "340520562", "341139228", "341832155", "349211944", "349983323", "354267701", "355039804", "363592773", "363894934", "363898641", "368520997", "375082967", "375578400", "375668451", "376219524", "376227621", "377008820", "381336731", "381361383", "384251764", "384568052", "384571801", "386264877", "387102517", "404638659", "404641332", "404755351", "406663475", "419506983", "433780598", "433967999", "434131599", "434145536", "438454166", "438615159", "440062286", "440063970", "440791096", "7636362", "10526229", "10531711", "46505639", "49671357", "56366847", "56394843", "85084935", "92718043", "126628622", "126653546", "162180601", "163813746", "176254650", "184021980", "196106469", "204939830", "211535142", "241108777", "241182364", "252402159", "254761541", "273002773", "310270453", "312695359", "313075595", "315493146", "318164972", "319061906", "348283902", "350089698", "354268355", "354283773", "363604547", "375581611", "376192534", "381118117", "382163479", "387060913", "387102519", "404754680", "405496121", "419491752", "438474038", "438482863", "440042779", "26746574", "76281009", "341827232", "204377240", "342457047", "50071452", "187051827", "206246221", "255386486", "318695474", "441075648" ], "inchikey": "IAKHMKGGTNLKSZ-INIZCTEOSA-N", "generic_name": "Colchicine", "designated_date": "1985-12-09", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Pharmacontrol Corporation|661 Palisade Ave., P.O. 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intermittent porphyria", "AIP", "Symptoms" ] } }, { "pubchem_sid": 438459028, "generic_name": "Hemin", "trade_name": "Panhematin", "designated_date": "1984-03-16", "designation_status": "Designated/Approved", "marketing_approval_date": "1983-07-20", "exclusivity_end_date": "1990-07-20", "sponsor": "Abbott Laboratories|Diagnostics Division|Abbott Park|Illinois|60064|United States", "orphan_designation": { "original_text": "Amelioration of recurrent attacks of acute intermittent porphyria (AIP) temporarily related to the menstrual cycle in susceptible women and similar symptoms which occur in other patients with AIP, porphyria variegata and hereditary coproporphyria.", "umls": [ "C0162531", "C0162532", "C0162565", "C1279945", "C1457887" ], "parsed_text": [ "Hereditary Coproporphyria", "Porphyria Variegata", "Acute intermittent porphyria", "AIP", "Symptoms" ] } }, { "pubchem_sid": 434252965, "generic_name": "Hemin", "trade_name": "Panhematin", "designated_date": "1984-03-16", 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intermittent porphyria", "AIP", "Symptoms" ] } }, { "pubchem_sid": 386623978, "generic_name": "Hemin", "trade_name": "Panhematin", "designated_date": "1984-03-16", "designation_status": "Designated/Approved", "marketing_approval_date": "1983-07-20", "exclusivity_end_date": "1990-07-20", "sponsor": "Abbott Laboratories|Diagnostics Division|Abbott Park|Illinois|60064|United States", "orphan_designation": { "original_text": "Amelioration of recurrent attacks of acute intermittent porphyria (AIP) temporarily related to the menstrual cycle in susceptible women and similar symptoms which occur in other patients with AIP, porphyria variegata and hereditary coproporphyria.", "umls": [ "C0162531", "C0162532", "C0162565", "C1279945", "C1457887" ], "parsed_text": [ "Hereditary Coproporphyria", "Porphyria Variegata", "Acute intermittent porphyria", "AIP", "Symptoms" ] } }, { "pubchem_sid": 385786934, "generic_name": "Hemin", "trade_name": "Panhematin", "designated_date": "1984-03-16", 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"exclusivity_protected_indication": "treatment of pediatric patients 2 years of age up to patients 8 years of age with confirmed Fabry disease", "sponsor": "Genzyme Corporation|50 Binney Street|Cambridge|Massachusetts|2142|United States", "orphan_designation": { "original_text": "Treatment of Fabry's disease.", "umls": "C0002986", "parsed_text": "Fabry's Disease" } }, { "pubchem_sid": 135311478, "generic_name": "Alpha-galactosidase A", "designated_date": "1990-07-20", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Desnick, Robert J. M.D.|The Mount Sinai School Of Medicine|Fifth Avenue at 100th Street, Box 1203|New York|New York|10029|United States", "orphan_designation": { "original_text": "Treatment of Fabry's disease.", "umls": "C0002986", "parsed_text": "Fabry's Disease" } }, { "pubchem_sid": 46508538, "generic_name": "Alpha-galactosidase A", "designated_date": "1990-07-20", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Desnick, Robert J. M.D.|The Mount Sinai School Of Medicine|Fifth Avenue at 100th Street, Box 1203|New York|New York|10029|United States", "orphan_designation": { "original_text": "Treatment of Fabry's disease.", "umls": "C0002986", "parsed_text": "Fabry's Disease" } }, { "pubchem_sid": 135311478, "generic_name": "Alpha-galactosidase A", "designated_date": "1998-06-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Shire Human Genetic Therapies, Inc.|700 Main Street|Cambridge|Massachusetts|2139|United States", "orphan_designation": { "original_text": "Long-term enzyme replacement therapy for the treatment of Fabry disease", "umls": "C0002986", "parsed_text": "Fabry Disease" } }, { "pubchem_sid": 46508538, "generic_name": "Alpha-galactosidase A", "designated_date": "1998-06-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Shire Human Genetic Therapies, Inc.|700 Main Street|Cambridge|Massachusetts|2139|United States", "orphan_designation": { "original_text": "Long-term enzyme replacement therapy for the treatment of Fabry disease", "umls": "C0002986", "parsed_text": "Fabry Disease" } }, { "pubchem_sid": 135311478, "generic_name": "Alpha-galactosidase A", "designated_date": "1991-06-17", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "David Calhoun, Ph.D.|Department of Chemistry|Convent Avenue & 138th Street|New York|New York|10031|United States", "orphan_designation": { "original_text": "Treatment of alpha-galactosidase A deficiency (Fabry's disease).", "umls": "C0002986", "parsed_text": "alpha-Galactosidase A Deficiency" } }, { "pubchem_sid": 46508538, "generic_name": "Alpha-galactosidase A", "designated_date": "1991-06-17", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "David Calhoun, Ph.D.|Department of Chemistry|Convent Avenue & 138th Street|New York|New York|10031|United States", "orphan_designation": { "original_text": "Treatment of alpha-galactosidase A deficiency (Fabry's disease).", "umls": "C0002986", "parsed_text": "alpha-Galactosidase A Deficiency" } } ] }, { "_id": "UMLS:C0027854", "fda_orphan_drug": [ { "pubchem_cid": 51634, "pubchem_sid": [ "596124", "12014416", "46506350", "46518432", "50017245", "50139361", "79091966", "103277564", "126649082", "134223536", "135011418", "144204381", "160963765", "170464950", "178101543", "210275570", "210281218", "249582554", "252225017", "254761691", "310269139", "312228126", "313077529", "315681349", "318026089", "318161667", "318243546", "319096716", "319553242", "329997201", "341142752", "347258598", "348038449", "348283098", "348397351", "349276013", "349987729", "355039164", "378134334", "381358004", "382164368", "385787584", "386624662", "387161172", "419500971", "434301210", "438462968", "440790850", "79091968", "375587988", "387161171", 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"designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Rain Therapeutics, Inc.|8000 Jarvis Rd., Suite 204|Newark|California|94560|United States", "orphan_designation": { "original_text": "Treatment of liposarcoma", "umls": "C0023827", "parsed_text": "liposarcoma" } } ] }, { "_id": "UMLS:C1368683", "fda_orphan_drug": [ { "pubchem_cid": 46937352, "pubchem_sid": [ "295371160", "404719197" ], "inchikey": "DGVCEXQFNYYRQI-BTJKTKAUSA-N", "generic_name": "milciclib maleate", "designated_date": "2012-09-20", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Tiziana Life Sciences PLC|18 South Street|W1K 1DG|London|United Kingdom", "orphan_designation": { "original_text": "Treatment of thymic epithelial tumors.", "umls": "C1368683", "parsed_text": "epithelial tumors" } } ] }, { "_id": "UMLS:C0595939", "fda_orphan_drug": [ { "pubchem_cid": 5282381, "pubchem_sid": [ "7980004", 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"346442721", "347271293", "349539049", "375204165", "375575654", "404639067", "441076086" ], "inchikey": "OJLOPKGSLYJEMD-URPKTTJQSA-N", "generic_name": "misoprostol", "designated_date": "2005-01-10", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Gynuity Health Projects, LLC|15 East 26th Street|New York|New York|10010|United States", "orphan_designation": { "original_text": "Treatment of intrauterine fetal death not accompanied by complete expulsion of the products of conception in the second and third trimesters of pregnancy.", "umls": "C0595939", "parsed_text": "Intrauterine Fetal Death" } } ] }, { "_id": "UMLS:C4551761", "fda_orphan_drug": [ { "pubchem_cid": 4236, "pubchem_sid": [ "7980013", "12014011", "26719679", "46386605", "46504648", "48416288", "49834664", "50065072", "56313688", "56313766", "81065631", "92714071", "93166872", "103458965", "104043335", "126592891", "126624419", "126655680", "126685090", "131407184", 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"generic_name": "Modafinil", "trade_name": "Provigil", "designated_date": "1993-03-15", "designation_status": "Designated/Approved", "approved_labeled_indication": "Improve wakefulness in patients with excessive daytime sleepiness associated with narcolepsy.", "marketing_approval_date": "1998-12-24", "exclusivity_end_date": "2005-12-24", "sponsor": "Cephalon, Inc.|145 Brandywine Parkway|West Chester|Pennsylvania|19380|United States", "orphan_designation": { "original_text": "Treatment of excessive daytime sleepiness in narcolepsy.", "umls": [ "C0027404", "C4551761" ], "parsed_text": [ "Narcolepsy", "Excessive daytime sleepiness" ] } } ] }, { "_id": "UMLS:C1414313", "fda_orphan_drug": [ { "pubchem_cid": 117909640, "pubchem_sid": 348351320, "inchikey": "IDRGFNPZDVBSSE-UHFFFAOYSA-N", "generic_name": "N-(3-(2-(2,3-difluoro-4-(4-(2-hydroxyethyl)piperazin-1-yl)phenylamino)quinazolin-8-yl)phenyl)acrylamide", "designated_date": "2017-09-05", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Checkpoint Therapeutics, Inc.|2 Gansevoort Street|9th Floor|New York|New York|10014|United States", "orphan_designation": { "original_text": "Treatment of epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC)", "umls": [ "C0007131", "C1414313", "C3539721" ], "parsed_text": [ "Non-Small Cell Lung Cancer", "EPIDERMAL GROWTH FACTOR RECEPTOR", "Non-Small Cell Lung Cancer" ] } }, { "pubchem_cid": 71496458, "pubchem_sid": [ "210275341", "210280989", "223366052", "223370460", "252151380", "252160935", "310265210", "312367127", "312531017", "312616285", "313065691", "318163960", "319412253", "340125552", "350095312", "355039279", "375577402", "385645111", "386048307", "386481457", "387247835", "402287505", "405430332", "438789238", "439977008", "440801090", "440816354", "332853784" ], "inchikey": "DUYJMQONPNNFPI-UHFFFAOYSA-N", "generic_name": "osimertinib", "trade_name": "Tagrisso", "designated_date": "2014-09-04", "designation_status": "Designated/Approved", "approved_labeled_indication": "adjuvant therapy after tumor resection in adult patients with non-small cell lung cancer (NSCLC) whose tumors have epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R mutations, as detected by an FDA-approved test", "marketing_approval_date": "2020-12-18", "exclusivity_end_date": "2027-12-18", "exclusivity_protected_indication": "adjuvant therapy after tumor resection in adult patients with non-small cell lung cancer (NSCLC) whose tumors have epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R mutations, as detected by an FDA-approved test", "sponsor": "AstraZeneca Pharmaceuticals LP|P. O. Box 8355|1800 Concord Pike|Wilmington|Delaware|19803|United States", "orphan_designation": { "original_text": "Treatment of epidermal growth factor receptor mutation-positive non-small cell lung cancer", "umls": [ "C1414313", "C3539721" ], "parsed_text": [ "EPIDERMAL GROWTH FACTOR RECEPTOR", "Non-Small Cell Lung Cancer" ] } }, { "pubchem_cid": 71496458, "pubchem_sid": [ "210275341", "210280989", "223366052", "223370460", "252151380", "252160935", "310265210", "312367127", "312531017", "312616285", "313065691", "318163960", "319412253", "340125552", "350095312", "355039279", "375577402", "385645111", "386048307", "386481457", "387247835", "402287505", "405430332", "438789238", "439977008", "440801090", "440816354", "332853784" ], "inchikey": "DUYJMQONPNNFPI-UHFFFAOYSA-N", "generic_name": "osimertinib", "trade_name": "Tagrisso", "designated_date": "2014-09-04", "designation_status": "Designated/Approved", "approved_labeled_indication": "For treatment of patients with metastatic epidermal growth factor receptor (EGFR) T790M mutation-positive non-small cell lung cancer (NSCLC), as detected by an FDA-approved test, who have progressed on or after EGFR TKI therapy", "marketing_approval_date": "2015-11-13", "exclusivity_end_date": "2022-11-13", "exclusivity_protected_indication": "For treatment of patients with metastatic epidermal growth factor receptor (EGFR) T790M mutation-positive non-small cell lung cancer (NSCLC), as detected by an FDA-approved test, who have progressed on or after EGFR TKI therapy", "sponsor": "AstraZeneca Pharmaceuticals LP|P. O. Box 8355|1800 Concord Pike|Wilmington|Delaware|19803|United States", "orphan_designation": { "original_text": "Treatment of epidermal growth factor receptor mutation-positive non-small cell lung cancer", "umls": [ "C1414313", "C3539721" ], "parsed_text": [ "EPIDERMAL GROWTH FACTOR RECEPTOR", "Non-Small Cell Lung Cancer" ] } }, { "pubchem_cid": 71496458, "pubchem_sid": [ "210275341", "210280989", "223366052", "223370460", "252151380", "252160935", "310265210", "312367127", "312531017", "312616285", "313065691", "318163960", "319412253", "340125552", "350095312", "355039279", "375577402", "385645111", "386048307", "386481457", "387247835", "402287505", "405430332", "438789238", "439977008", "440801090", "440816354", "332853784" ], "inchikey": "DUYJMQONPNNFPI-UHFFFAOYSA-N", "generic_name": "osimertinib", "trade_name": "Tagrisso", "designated_date": "2014-09-04", "designation_status": "Designated/Approved", "approved_labeled_indication": "TAGRISSO is indicated for the first-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R mutations, as detected by an FDA-approved test", "marketing_approval_date": "2018-04-18", "exclusivity_end_date": "2025-04-18", "exclusivity_protected_indication": "TAGRISSO is indicated for the first-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R mutations, as detected by an FDA-approved test", "sponsor": "AstraZeneca Pharmaceuticals LP|P. O. Box 8355|1800 Concord Pike|Wilmington|Delaware|19803|United States", "orphan_designation": { "original_text": "Treatment of epidermal growth factor receptor mutation-positive non-small cell lung cancer", "umls": [ "C1414313", "C3539721" ], "parsed_text": [ "EPIDERMAL GROWTH FACTOR RECEPTOR", "Non-Small Cell Lung Cancer" ] } }, { "pubchem_cid": 57335384, "pubchem_sid": [ "172650914", "210275306", "210280955", "249565649", "255343085", "312468158", "312616269", "319403453", "329881447", "335969319", "340125553", "341191034", "347828240", "355039294", "375577399", "375590692", "375776007", "376033414", "377019806", "378132084", "384569121", "385652487", "386488942", "387235883", "434315026", "438493257", "440802669" ], "inchikey": "HUFOZJXAKZVRNJ-UHFFFAOYSA-N", "generic_name": "rociletinib", "designated_date": "2013-05-14", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Clovis Oncology, Inc.|5500 Flatiron Parkway|Suite 100|Boulder|Colorado|80301|United States", "orphan_designation": { "original_text": "Treatment of non-small cell lung cancer and mutations in the epidermal growth factor receptor", "umls": [ "C1414313", "C3539721" ], "parsed_text": [ "EPIDERMAL GROWTH FACTOR RECEPTOR", "Non-Small Cell Lung Cancer" ] } } ] }, { "_id": "MONDO:0019542", "fda_orphan_drug": [ { "pubchem_cid": 12035, "pubchem_sid": [ "8144902", "12013391", "48413148", "48422870", "49856254", "53790587", "56310757", "56311342", "56311343", "56311724", "56314540", "92714687", "99443235", "126623942", "126655263", "134978062", "136348796", "160967850", "174478612", "184818743", "196106914", "223741118", "249926182", "275228731", "318160103", "319070271", "321939574", "342398324", "347912801", "348275440", "349974913", "387141259", "404834430", "433777488", "409483", "85083256", "124994937", "125321798", "315683346", "341139678", "386304089", "387141261", "127268235", "127268236", "223710691" ], "inchikey": 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acute liver failure or acute on chronic liver disase", "umls": [ "C0019151", "C0162557", "C0220994" ], "parsed_text": [ "Hepatic Encephalopathy", "Acute liver failure", "Hyperammonemia" ] } }, { "pubchem_sid": 178101605, "generic_name": "cardiotrophin-1", "designated_date": "2011-09-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Digna Biotech, S.L.|Calloe ORENSE No. 85|2nd Floor|Madrid|Spain", "orphan_designation": { "original_text": "Treatment of acute liver failure", "umls": "C0162557", "parsed_text": "Acute liver failure" } } ] }, { "_id": "MONDO:0010161", "fda_orphan_drug": [ { "pubchem_cid": 115355, "pubchem_sid": [ "46507380", "49957875", "50139362", "76271421", "93167147", "103425313", "103987675", "123051096", "126666671", "134223796", "135072534", "136340345", "152344367", "160963695", "162011463", "163372627", "178103440", "187072003", "204420341", "207180286", "210279764", "210282087", "223375405", 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del Nogal|Suite 120|Carlsbad|California|92011|United States", "orphan_designation": { "original_text": "Use as an antidote in the management of systemic monochloroacetic acid poisoning", "umls": "C4707283", "parsed_text": "Systemic monochloroacetic acid poisoning" } } ] }, { "_id": "UMLS:C0007134", "fda_orphan_drug": [ { "pubchem_cid": 216239, "pubchem_sid": [ "12015507", "14720365", "46505329", "46516901", "50069824", "50100118", "53787819", "56312334", "56312336", "56312338", "56312517", "56312519", "56312521", "56394953", "75518900", "91148447", "92718861", "93581025", "103420820", "126592980", "126621268", "126648470", "126667052", "127301466", "127301467", "127301468", "127301469", "127301470", "135135413", "136988877", "160963744", "162187510", "164193922", "164205279", "165238004", "170498763", "178102337", "187072864", "196106195", "210274662", "210280294", "211536173", "223372989", "223702425", "223738908", "251890354", "251916684", "251917923", "251971230", "252216331", "252451842", "252543354", "254760598", "255370630", "273004163", "274520591", "274955142", "277309430", "310280203", "312228110", "312616194", "312809921", "313077268", "313082226", "315446230", "315678528", "316541656", "318162269", "318238766", "319209219", "319477227", "319552704", "322090670", "340516981", "340521809", "341841046", "347914265", "348283078", "348947944", "349994517", "350080667", "354281168", "355039702", "355181865", "355426538", "374034089", "375081381", "375577411", "375581801", "375651413", "376229618", "377309919", "378135535", "381328559", "381756449", "382159450", "383460570", "384570851", "385790473", "385842156", "386269288", "386627634", "387060553", "387124229", "402287699", "402289189", "402419945", "405045804", "405068296", "405424602", "406689441", "433841866", "438517445", "439459500", "440774553", "440790374", "440816345", "537765", "341141089", "438580978", "75518901", "75518902", "163644328", "318696011", "319433682", "434122845", "438546799", "441079974" ], "inchikey": "MLDQJTXFUGDVEO-UHFFFAOYSA-N", "generic_name": "Sorafenib", "trade_name": "Nexavar", "designated_date": "2004-10-08", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of patients with advanced renal cell carcinoma", "marketing_approval_date": "2005-12-20", "exclusivity_end_date": "2012-12-20", "sponsor": "Bayer Pharmaceutical Corporation|400 Morgan Lane|West Haven|Connecticut|6516|United States", "orphan_designation": { "original_text": "Treatment of renal cell carcinoma.", "umls": "C0007134", "parsed_text": "Renal Cell Carcinoma" } }, { "pubchem_cid": 154827473, "pubchem_sid": [ "12014940", "56313948", "103771165", "131346349", "135105386", "144206068", "152258857", "160647706", "160967858", "162172159", "174527830", "175437917", "178102515", "204359550", "252158731", "254760066", "310280528", "312311726", "312616372", "315678400", "316541660", "318153060", "318163458", "332864831", "341100968", "341142655", "341854972", 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"348353832", "354265908", "364196441", "375562992", "422710142", "426405759", "433865533", "438637079", "441080312" ], "inchikey": "CBPNZQVSJQDFBE-RODMVNQKSA-N", "generic_name": "Temsirolimus", "trade_name": "Torisel", "designated_date": "2004-12-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of advanced renal cell carcinoma", "marketing_approval_date": "2007-05-30", "exclusivity_end_date": "2014-05-30", "sponsor": "Wyeth Pharmaceuticals, Inc.|P. O. Box 8299|Phildelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "Treatment of renal cell carcinoma", "umls": "C0007134", "parsed_text": "Renal Cell Carcinoma" } }, { "pubchem_cid": 154575198, "pubchem_sid": [ "12014940", "56313948", "103771165", "131346349", "135105386", "144206068", "152258857", "160647706", "160967858", "162172159", "174527830", "175437917", "178102515", "204359550", "252158731", "254760066", "310280528", "312311726", "312616372", "315678400", "316541660", "318153060", "318163458", "332864831", "341100968", "341142655", "341854972", "354335686", "375577136", "384568322", "384572156", "385638415", "386632609", "406722223", "419480166", "434315187", "438475185", "438493358", "440786213", "53788684", "126666958", "252216390", "273316948", "313082308", "319332855", "329891082", "329978376", "349986356", "381391551", "382156887", "436380077", "440790951", "441016339", "17396179", "99443243", "223444871", "312228938", "332852674", "348284052", "426257242", "126617270", "196112710", "251971410", "313075773", "316900193", "318242896", "319166069", "387078114", "163123459", "319411484", "340502971", "347235644", "405460216", "440006878", "50070177", "162249026", "319340263", "319424078", "348814616", "355040167", "375664784", "377461485", "381183588", "381940665", "406531701", "426260554", "520036", "50883861", "162011848", "223434992", "252160375", "312811417", "319363586", "319379126", "319397925", "319399230", "319407906", "319409097", "319413543", "319413592", "319416085", "319417630", "319417970", "342547077", "348353832", "354265908", "364196441", "375562992", "422710142", "426405759", "433865533", "438637079", "441080312" ], "inchikey": "CBPNZQVSJQDFBE-NUTFMQKQSA-N", "generic_name": "Temsirolimus", "trade_name": "Torisel", "designated_date": "2004-12-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of advanced renal cell carcinoma", "marketing_approval_date": "2007-05-30", "exclusivity_end_date": "2014-05-30", "sponsor": "Wyeth Pharmaceuticals, Inc.|P. O. Box 8299|Phildelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "Treatment of renal cell carcinoma", "umls": "C0007134", "parsed_text": "Renal Cell Carcinoma" } }, { "pubchem_cid": 152772378, "pubchem_sid": [ "12014940", "56313948", "103771165", "131346349", "135105386", "144206068", "152258857", "160647706", "160967858", "162172159", "174527830", "175437917", "178102515", "204359550", "252158731", "254760066", "310280528", "312311726", "312616372", "315678400", "316541660", "318153060", "318163458", "332864831", "341100968", "341142655", "341854972", "354335686", "375577136", "384568322", "384572156", "385638415", "386632609", "406722223", "419480166", "434315187", "438475185", "438493358", "440786213", "53788684", "126666958", "252216390", "273316948", "313082308", "319332855", "329891082", "329978376", "349986356", "381391551", "382156887", "436380077", "440790951", "441016339", "17396179", "99443243", "223444871", "312228938", "332852674", "348284052", "426257242", "126617270", "196112710", "251971410", "313075773", "316900193", "318242896", "319166069", "387078114", "163123459", "319411484", "340502971", "347235644", "405460216", "440006878", "50070177", "162249026", "319340263", "319424078", "348814616", "355040167", "375664784", "377461485", "381183588", "381940665", "406531701", "426260554", "520036", "50883861", "162011848", "223434992", "252160375", "312811417", "319363586", "319379126", "319397925", "319399230", "319407906", "319409097", "319413543", "319413592", "319416085", "319417630", "319417970", "342547077", "348353832", "354265908", "364196441", "375562992", "422710142", "426405759", "433865533", "438637079", "441080312" ], "inchikey": "CBPNZQVSJQDFBE-LGVPMJLXSA-N", "generic_name": "Temsirolimus", "trade_name": "Torisel", "designated_date": "2004-12-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of advanced renal cell carcinoma", "marketing_approval_date": "2007-05-30", "exclusivity_end_date": "2014-05-30", "sponsor": "Wyeth Pharmaceuticals, Inc.|P. O. Box 8299|Phildelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "Treatment of renal cell carcinoma", "umls": "C0007134", "parsed_text": "Renal Cell Carcinoma" } }, { "pubchem_cid": 137295275, "pubchem_sid": [ "12014940", "56313948", "103771165", "131346349", "135105386", "144206068", "152258857", "160647706", "160967858", "162172159", "174527830", "175437917", "178102515", "204359550", "252158731", "254760066", "310280528", "312311726", "312616372", "315678400", "316541660", "318153060", "318163458", "332864831", "341100968", "341142655", "341854972", "354335686", "375577136", "384568322", "384572156", "385638415", "386632609", "406722223", "419480166", "434315187", "438475185", "438493358", "440786213", "53788684", "126666958", "252216390", "273316948", "313082308", "319332855", "329891082", "329978376", "349986356", "381391551", "382156887", "436380077", "440790951", "441016339", "17396179", "99443243", "223444871", "312228938", "332852674", "348284052", "426257242", "126617270", "196112710", "251971410", "313075773", "316900193", "318242896", "319166069", "387078114", "163123459", "319411484", "340502971", "347235644", "405460216", "440006878", "50070177", "162249026", "319340263", "319424078", "348814616", "355040167", "375664784", "377461485", "381183588", "381940665", "406531701", "426260554", "520036", "50883861", "162011848", "223434992", "252160375", "312811417", "319363586", "319379126", "319397925", "319399230", "319407906", "319409097", "319413543", "319413592", "319416085", "319417630", "319417970", "342547077", "348353832", "354265908", "364196441", "375562992", "422710142", "426405759", "433865533", "438637079", "441080312" ], "inchikey": "CBPNZQVSJQDFBE-HELDCPHESA-N", "generic_name": "Temsirolimus", "trade_name": "Torisel", "designated_date": "2004-12-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of advanced renal cell carcinoma", "marketing_approval_date": "2007-05-30", "exclusivity_end_date": "2014-05-30", "sponsor": "Wyeth Pharmaceuticals, Inc.|P. O. Box 8299|Phildelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "Treatment of renal cell carcinoma", "umls": "C0007134", "parsed_text": "Renal Cell Carcinoma" } }, { "pubchem_cid": 132274070, "pubchem_sid": [ "12014940", "56313948", "103771165", "131346349", "135105386", "144206068", "152258857", "160647706", "160967858", "162172159", "174527830", "175437917", "178102515", "204359550", "252158731", "254760066", "310280528", "312311726", "312616372", "315678400", "316541660", "318153060", "318163458", "332864831", "341100968", "341142655", "341854972", "354335686", "375577136", "384568322", "384572156", "385638415", "386632609", "406722223", "419480166", "434315187", "438475185", "438493358", "440786213", "53788684", "126666958", "252216390", "273316948", "313082308", "319332855", "329891082", "329978376", "349986356", "381391551", "382156887", "436380077", "440790951", "441016339", "17396179", "99443243", "223444871", "312228938", "332852674", "348284052", "426257242", "126617270", "196112710", "251971410", "313075773", "316900193", "318242896", "319166069", "387078114", "163123459", "319411484", "340502971", "347235644", "405460216", "440006878", "50070177", "162249026", "319340263", "319424078", "348814616", "355040167", "375664784", "377461485", "381183588", "381940665", "406531701", "426260554", "520036", "50883861", "162011848", "223434992", "252160375", "312811417", "319363586", "319379126", "319397925", "319399230", "319407906", "319409097", "319413543", "319413592", "319416085", "319417630", "319417970", "342547077", "348353832", "354265908", "364196441", "375562992", "422710142", "426405759", "433865533", "438637079", "441080312" ], "inchikey": "CBPNZQVSJQDFBE-VYCIWTNRSA-N", "generic_name": "Temsirolimus", "trade_name": "Torisel", "designated_date": "2004-12-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of advanced renal cell carcinoma", "marketing_approval_date": "2007-05-30", "exclusivity_end_date": "2014-05-30", "sponsor": "Wyeth Pharmaceuticals, Inc.|P. O. Box 8299|Phildelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "Treatment of renal cell carcinoma", "umls": "C0007134", "parsed_text": "Renal Cell Carcinoma" } }, { "pubchem_cid": 86287409, "pubchem_sid": [ "12014940", "56313948", "103771165", "131346349", "135105386", "144206068", "152258857", "160647706", "160967858", "162172159", "174527830", "175437917", "178102515", "204359550", "252158731", "254760066", "310280528", "312311726", "312616372", "315678400", "316541660", "318153060", "318163458", "332864831", "341100968", "341142655", "341854972", "354335686", "375577136", "384568322", "384572156", "385638415", "386632609", "406722223", "419480166", "434315187", "438475185", "438493358", "440786213", "53788684", "126666958", "252216390", "273316948", "313082308", "319332855", "329891082", "329978376", "349986356", "381391551", "382156887", "436380077", "440790951", "441016339", "17396179", "99443243", "223444871", "312228938", "332852674", "348284052", "426257242", "126617270", "196112710", "251971410", "313075773", "316900193", "318242896", "319166069", "387078114", "163123459", "319411484", "340502971", "347235644", "405460216", "440006878", "50070177", "162249026", "319340263", "319424078", "348814616", "355040167", "375664784", "377461485", "381183588", "381940665", "406531701", "426260554", "520036", "50883861", "162011848", "223434992", "252160375", "312811417", "319363586", "319379126", "319397925", "319399230", "319407906", "319409097", "319413543", "319413592", "319416085", "319417630", "319417970", "342547077", "348353832", "354265908", "364196441", "375562992", "422710142", "426405759", "433865533", "438637079", "441080312" ], "inchikey": "CBPNZQVSJQDFBE-QWRHTZMXSA-N", "generic_name": "Temsirolimus", "trade_name": "Torisel", "designated_date": "2004-12-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of advanced renal cell carcinoma", "marketing_approval_date": "2007-05-30", "exclusivity_end_date": "2014-05-30", "sponsor": "Wyeth Pharmaceuticals, Inc.|P. O. Box 8299|Phildelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "Treatment of renal cell carcinoma", "umls": "C0007134", "parsed_text": "Renal Cell Carcinoma" } }, { "pubchem_cid": 71296107, "pubchem_sid": [ "12014940", "56313948", "103771165", "131346349", "135105386", "144206068", "152258857", "160647706", "160967858", "162172159", "174527830", "175437917", "178102515", "204359550", "252158731", "254760066", "310280528", "312311726", "312616372", "315678400", "316541660", "318153060", "318163458", "332864831", "341100968", "341142655", "341854972", "354335686", "375577136", "384568322", "384572156", "385638415", "386632609", "406722223", "419480166", "434315187", "438475185", "438493358", "440786213", "53788684", "126666958", "252216390", "273316948", "313082308", "319332855", "329891082", "329978376", "349986356", "381391551", "382156887", "436380077", "440790951", "441016339", "17396179", "99443243", "223444871", "312228938", "332852674", "348284052", "426257242", "126617270", "196112710", "251971410", "313075773", "316900193", "318242896", "319166069", "387078114", "163123459", "319411484", "340502971", "347235644", "405460216", "440006878", "50070177", "162249026", "319340263", "319424078", "348814616", "355040167", "375664784", "377461485", "381183588", "381940665", "406531701", "426260554", "520036", "50883861", "162011848", "223434992", "252160375", "312811417", "319363586", "319379126", "319397925", "319399230", "319407906", "319409097", "319413543", "319413592", "319416085", "319417630", "319417970", "342547077", "348353832", "354265908", "364196441", "375562992", "422710142", "426405759", "433865533", "438637079", "441080312" ], "inchikey": "CBPNZQVSJQDFBE-DZCFMHCPSA-N", "generic_name": "Temsirolimus", "trade_name": "Torisel", "designated_date": "2004-12-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of advanced renal cell carcinoma", "marketing_approval_date": "2007-05-30", "exclusivity_end_date": "2014-05-30", "sponsor": "Wyeth Pharmaceuticals, Inc.|P. O. Box 8299|Phildelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "Treatment of renal cell carcinoma", "umls": "C0007134", "parsed_text": "Renal Cell Carcinoma" } }, { "pubchem_cid": 54608508, "pubchem_sid": [ "12014940", "56313948", "103771165", "131346349", "135105386", "144206068", "152258857", "160647706", "160967858", "162172159", "174527830", "175437917", "178102515", "204359550", "252158731", "254760066", "310280528", "312311726", "312616372", "315678400", "316541660", "318153060", "318163458", "332864831", "341100968", "341142655", "341854972", "354335686", "375577136", "384568322", "384572156", "385638415", "386632609", "406722223", "419480166", "434315187", "438475185", "438493358", "440786213", "53788684", "126666958", "252216390", "273316948", "313082308", "319332855", "329891082", "329978376", "349986356", "381391551", "382156887", "436380077", "440790951", "441016339", "17396179", "99443243", "223444871", "312228938", "332852674", "348284052", "426257242", "126617270", "196112710", "251971410", "313075773", "316900193", "318242896", "319166069", "387078114", "163123459", "319411484", "340502971", "347235644", "405460216", "440006878", "50070177", "162249026", "319340263", "319424078", "348814616", "355040167", "375664784", "377461485", "381183588", "381940665", "406531701", "426260554", "520036", "50883861", "162011848", "223434992", "252160375", "312811417", "319363586", "319379126", "319397925", "319399230", "319407906", "319409097", "319413543", "319413592", "319416085", "319417630", "319417970", "342547077", "348353832", "354265908", "364196441", "375562992", "422710142", "426405759", "433865533", "438637079", "441080312" ], "inchikey": "CBPNZQVSJQDFBE-VBSVFFBFSA-N", "generic_name": "Temsirolimus", "trade_name": "Torisel", "designated_date": "2004-12-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of advanced renal cell carcinoma", "marketing_approval_date": "2007-05-30", "exclusivity_end_date": "2014-05-30", "sponsor": "Wyeth Pharmaceuticals, Inc.|P. O. Box 8299|Phildelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "Treatment of renal cell carcinoma", "umls": "C0007134", "parsed_text": "Renal Cell Carcinoma" } }, { "pubchem_cid": 9876533, "pubchem_sid": [ "12014940", "56313948", "103771165", "131346349", "135105386", "144206068", "152258857", "160647706", "160967858", "162172159", "174527830", "175437917", "178102515", "204359550", "252158731", "254760066", "310280528", "312311726", "312616372", "315678400", "316541660", "318153060", "318163458", "332864831", "341100968", "341142655", "341854972", "354335686", "375577136", "384568322", "384572156", "385638415", "386632609", "406722223", "419480166", "434315187", "438475185", "438493358", "440786213", "53788684", "126666958", "252216390", "273316948", "313082308", "319332855", "329891082", "329978376", "349986356", "381391551", "382156887", "436380077", "440790951", "441016339", "17396179", "99443243", "223444871", "312228938", "332852674", "348284052", "426257242", "126617270", "196112710", "251971410", "313075773", "316900193", "318242896", "319166069", "387078114", "163123459", "319411484", "340502971", "347235644", "405460216", "440006878", "50070177", "162249026", "319340263", "319424078", "348814616", "355040167", "375664784", "377461485", "381183588", "381940665", "406531701", "426260554", "520036", "50883861", "162011848", "223434992", "252160375", "312811417", "319363586", "319379126", "319397925", "319399230", "319407906", "319409097", "319413543", "319413592", "319416085", "319417630", "319417970", "342547077", "348353832", "354265908", "364196441", "375562992", "422710142", "426405759", "433865533", "438637079", "441080312" ], "inchikey": "CBPNZQVSJQDFBE-MDHHOXSCSA-N", "generic_name": "Temsirolimus", "trade_name": "Torisel", "designated_date": "2004-12-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of advanced renal cell carcinoma", "marketing_approval_date": "2007-05-30", "exclusivity_end_date": "2014-05-30", "sponsor": "Wyeth Pharmaceuticals, Inc.|P. O. Box 8299|Phildelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "Treatment of renal cell carcinoma", "umls": "C0007134", "parsed_text": "Renal Cell Carcinoma" } }, { "pubchem_cid": 145712265, "pubchem_sid": [ "12014940", "56313948", "103771165", "131346349", "135105386", "144206068", "152258857", "160647706", "160967858", "162172159", "174527830", "175437917", "178102515", "204359550", "252158731", "254760066", "310280528", "312311726", "312616372", "315678400", "316541660", "318153060", "318163458", "332864831", "341100968", "341142655", "341854972", "354335686", "375577136", "384568322", "384572156", "385638415", "386632609", "406722223", "419480166", "434315187", "438475185", "438493358", "440786213", "53788684", "126666958", "252216390", "273316948", "313082308", "319332855", "329891082", "329978376", "349986356", "381391551", "382156887", "436380077", "440790951", "441016339", "17396179", "99443243", "223444871", "312228938", "332852674", "348284052", "426257242", "126617270", "196112710", "251971410", "313075773", "316900193", "318242896", "319166069", "387078114", "163123459", "319411484", "340502971", "347235644", "405460216", "440006878", "50070177", "162249026", "319340263", "319424078", "348814616", "355040167", "375664784", "377461485", "381183588", "381940665", "406531701", "426260554", "520036", "50883861", "162011848", "223434992", "252160375", "312811417", "319363586", "319379126", "319397925", "319399230", "319407906", "319409097", "319413543", "319413592", "319416085", "319417630", "319417970", "342547077", "348353832", "354265908", "364196441", "375562992", "422710142", "426405759", "433865533", "438637079", "441080312" ], "inchikey": "CBPNZQVSJQDFBE-GOFCWFHQSA-N", "generic_name": "Temsirolimus", "trade_name": "Torisel", "designated_date": "2004-12-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of advanced renal cell carcinoma", "marketing_approval_date": "2007-05-30", "exclusivity_end_date": "2014-05-30", "sponsor": "Wyeth Pharmaceuticals, Inc.|P. O. Box 8299|Phildelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "Treatment of renal cell carcinoma", "umls": "C0007134", "parsed_text": "Renal Cell Carcinoma" } }, { "pubchem_cid": 71312019, "pubchem_sid": [ "12014940", "56313948", "103771165", "131346349", "135105386", "144206068", "152258857", "160647706", "160967858", "162172159", "174527830", "175437917", "178102515", "204359550", "252158731", "254760066", "310280528", "312311726", "312616372", "315678400", "316541660", "318153060", "318163458", "332864831", "341100968", "341142655", "341854972", "354335686", "375577136", "384568322", "384572156", "385638415", "386632609", "406722223", "419480166", "434315187", "438475185", "438493358", "440786213", "53788684", "126666958", "252216390", "273316948", "313082308", "319332855", "329891082", "329978376", "349986356", "381391551", "382156887", "436380077", "440790951", "441016339", "17396179", "99443243", "223444871", "312228938", "332852674", "348284052", "426257242", "126617270", "196112710", "251971410", "313075773", "316900193", "318242896", "319166069", "387078114", "163123459", "319411484", "340502971", "347235644", "405460216", "440006878", "50070177", "162249026", "319340263", "319424078", "348814616", "355040167", "375664784", "377461485", "381183588", "381940665", "406531701", "426260554", "520036", "50883861", "162011848", "223434992", "252160375", "312811417", "319363586", "319379126", "319397925", "319399230", "319407906", "319409097", "319413543", "319413592", "319416085", "319417630", "319417970", "342547077", "348353832", "354265908", "364196441", "375562992", "422710142", "426405759", "433865533", "438637079", "441080312" ], "inchikey": "CBPNZQVSJQDFBE-KFZNZFKNSA-N", "generic_name": "Temsirolimus", "trade_name": "Torisel", "designated_date": "2004-12-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of advanced renal cell carcinoma", "marketing_approval_date": "2007-05-30", "exclusivity_end_date": "2014-05-30", "sponsor": "Wyeth Pharmaceuticals, Inc.|P. O. Box 8299|Phildelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "Treatment of renal cell carcinoma", "umls": "C0007134", "parsed_text": "Renal Cell Carcinoma" } }, { "pubchem_cid": 24847874, "pubchem_sid": [ "12014940", "56313948", "103771165", "131346349", "135105386", "144206068", "152258857", "160647706", "160967858", "162172159", "174527830", "175437917", "178102515", "204359550", "252158731", "254760066", "310280528", "312311726", "312616372", "315678400", "316541660", "318153060", "318163458", "332864831", "341100968", "341142655", "341854972", "354335686", "375577136", "384568322", "384572156", "385638415", "386632609", "406722223", "419480166", "434315187", "438475185", "438493358", "440786213", "53788684", "126666958", "252216390", "273316948", "313082308", "319332855", "329891082", "329978376", "349986356", "381391551", "382156887", "436380077", "440790951", "441016339", "17396179", "99443243", "223444871", "312228938", "332852674", "348284052", "426257242", "126617270", "196112710", "251971410", "313075773", "316900193", "318242896", "319166069", "387078114", "163123459", "319411484", "340502971", "347235644", "405460216", "440006878", "50070177", "162249026", "319340263", "319424078", "348814616", "355040167", "375664784", "377461485", "381183588", "381940665", "406531701", "426260554", "520036", "50883861", "162011848", "223434992", "252160375", "312811417", "319363586", "319379126", "319397925", "319399230", "319407906", "319409097", "319413543", "319413592", "319416085", "319417630", "319417970", "342547077", "348353832", "354265908", "364196441", "375562992", "422710142", "426405759", "433865533", "438637079", "441080312" ], "inchikey": "CBPNZQVSJQDFBE-ZJEVVSOISA-N", "generic_name": "Temsirolimus", "trade_name": "Torisel", "designated_date": "2004-12-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of advanced renal cell carcinoma", "marketing_approval_date": "2007-05-30", "exclusivity_end_date": "2014-05-30", "sponsor": "Wyeth Pharmaceuticals, Inc.|P. O. Box 8299|Phildelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "Treatment of renal cell carcinoma", "umls": "C0007134", "parsed_text": "Renal Cell Carcinoma" } }, { "pubchem_cid": 5938194, "pubchem_sid": [ "12014940", "56313948", "103771165", "131346349", "135105386", "144206068", "152258857", "160647706", "160967858", "162172159", "174527830", "175437917", "178102515", "204359550", "252158731", "254760066", "310280528", "312311726", "312616372", "315678400", "316541660", "318153060", "318163458", "332864831", "341100968", "341142655", "341854972", "354335686", "375577136", "384568322", "384572156", "385638415", "386632609", "406722223", "419480166", "434315187", "438475185", "438493358", "440786213", "53788684", "126666958", "252216390", "273316948", "313082308", "319332855", "329891082", "329978376", "349986356", "381391551", "382156887", "436380077", "440790951", "441016339", "17396179", "99443243", "223444871", "312228938", "332852674", "348284052", "426257242", "126617270", "196112710", "251971410", "313075773", "316900193", "318242896", "319166069", "387078114", "163123459", "319411484", "340502971", "347235644", "405460216", "440006878", "50070177", "162249026", "319340263", "319424078", "348814616", "355040167", "375664784", "377461485", "381183588", "381940665", "406531701", "426260554", "520036", "50883861", "162011848", "223434992", "252160375", "312811417", "319363586", "319379126", "319397925", "319399230", "319407906", "319409097", "319413543", "319413592", "319416085", "319417630", "319417970", "342547077", "348353832", "354265908", "364196441", "375562992", "422710142", "426405759", "433865533", "438637079", "441080312" ], "inchikey": "CBPNZQVSJQDFBE-DYFDTRMFSA-N", "generic_name": "Temsirolimus", "trade_name": "Torisel", "designated_date": "2004-12-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of advanced renal cell carcinoma", "marketing_approval_date": "2007-05-30", "exclusivity_end_date": "2014-05-30", "sponsor": "Wyeth Pharmaceuticals, Inc.|P. O. Box 8299|Phildelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "Treatment of renal cell carcinoma", "umls": "C0007134", "parsed_text": "Renal Cell Carcinoma" } }, { "pubchem_cid": 129317846, "pubchem_sid": [ "12014940", "56313948", "103771165", "131346349", "135105386", "144206068", "152258857", "160647706", "160967858", "162172159", "174527830", "175437917", "178102515", "204359550", "252158731", "254760066", "310280528", "312311726", "312616372", "315678400", "316541660", "318153060", "318163458", "332864831", "341100968", "341142655", "341854972", "354335686", "375577136", "384568322", "384572156", "385638415", "386632609", "406722223", "419480166", "434315187", "438475185", "438493358", "440786213", "53788684", "126666958", "252216390", "273316948", "313082308", "319332855", "329891082", "329978376", "349986356", "381391551", "382156887", "436380077", "440790951", "441016339", "17396179", "99443243", "223444871", "312228938", "332852674", "348284052", "426257242", "126617270", "196112710", "251971410", "313075773", "316900193", "318242896", "319166069", "387078114", "163123459", "319411484", "340502971", "347235644", "405460216", "440006878", "50070177", "162249026", "319340263", "319424078", "348814616", "355040167", "375664784", "377461485", "381183588", "381940665", "406531701", "426260554", "520036", "50883861", "162011848", "223434992", "252160375", "312811417", "319363586", "319379126", "319397925", "319399230", "319407906", "319409097", "319413543", "319413592", "319416085", "319417630", "319417970", "342547077", "348353832", "354265908", "364196441", "375562992", "422710142", "426405759", "433865533", "438637079", "441080312" ], "inchikey": "CBPNZQVSJQDFBE-UITOJQKMSA-N", "generic_name": "Temsirolimus", "trade_name": "Torisel", "designated_date": "2004-12-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of advanced renal cell carcinoma", "marketing_approval_date": "2007-05-30", "exclusivity_end_date": "2014-05-30", "sponsor": "Wyeth Pharmaceuticals, Inc.|P. O. Box 8299|Phildelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "Treatment of renal cell carcinoma", "umls": "C0007134", "parsed_text": "Renal Cell Carcinoma" } }, { "pubchem_cid": 71433778, "pubchem_sid": [ "12014940", "56313948", "103771165", "131346349", "135105386", "144206068", "152258857", "160647706", "160967858", "162172159", "174527830", "175437917", "178102515", "204359550", "252158731", "254760066", "310280528", "312311726", "312616372", "315678400", "316541660", "318153060", "318163458", "332864831", "341100968", "341142655", "341854972", "354335686", "375577136", "384568322", "384572156", "385638415", "386632609", "406722223", "419480166", "434315187", "438475185", "438493358", "440786213", "53788684", "126666958", "252216390", "273316948", "313082308", "319332855", "329891082", "329978376", "349986356", "381391551", "382156887", "436380077", "440790951", "441016339", "17396179", "99443243", "223444871", "312228938", "332852674", "348284052", "426257242", "126617270", "196112710", "251971410", "313075773", "316900193", "318242896", "319166069", "387078114", "163123459", "319411484", "340502971", "347235644", "405460216", "440006878", "50070177", "162249026", "319340263", "319424078", "348814616", "355040167", "375664784", "377461485", "381183588", "381940665", "406531701", "426260554", "520036", "50883861", "162011848", "223434992", "252160375", "312811417", "319363586", "319379126", "319397925", "319399230", "319407906", "319409097", "319413543", "319413592", "319416085", "319417630", "319417970", "342547077", "348353832", "354265908", "364196441", "375562992", "422710142", "426405759", "433865533", "438637079", "441080312" ], "inchikey": "CBPNZQVSJQDFBE-AQHKCTBXSA-N", "generic_name": "Temsirolimus", "trade_name": "Torisel", "designated_date": "2004-12-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of advanced renal cell carcinoma", "marketing_approval_date": "2007-05-30", "exclusivity_end_date": "2014-05-30", "sponsor": "Wyeth Pharmaceuticals, Inc.|P. O. Box 8299|Phildelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "Treatment of renal cell carcinoma", "umls": "C0007134", "parsed_text": "Renal Cell Carcinoma" } }, { "pubchem_cid": 53486199, "pubchem_sid": [ "12014940", "56313948", "103771165", "131346349", "135105386", "144206068", "152258857", "160647706", "160967858", "162172159", "174527830", "175437917", "178102515", "204359550", "252158731", "254760066", "310280528", "312311726", "312616372", "315678400", "316541660", "318153060", "318163458", "332864831", "341100968", "341142655", "341854972", "354335686", "375577136", "384568322", "384572156", "385638415", "386632609", "406722223", "419480166", "434315187", "438475185", "438493358", "440786213", "53788684", "126666958", "252216390", "273316948", "313082308", "319332855", "329891082", "329978376", "349986356", "381391551", "382156887", "436380077", "440790951", "441016339", "17396179", "99443243", "223444871", "312228938", "332852674", "348284052", "426257242", "126617270", "196112710", "251971410", "313075773", "316900193", "318242896", "319166069", "387078114", "163123459", "319411484", "340502971", "347235644", "405460216", "440006878", "50070177", "162249026", "319340263", "319424078", "348814616", "355040167", "375664784", "377461485", "381183588", "381940665", "406531701", "426260554", "520036", "50883861", "162011848", "223434992", "252160375", "312811417", "319363586", "319379126", "319397925", "319399230", "319407906", "319409097", "319413543", "319413592", "319416085", "319417630", "319417970", "342547077", "348353832", "354265908", "364196441", "375562992", "422710142", "426405759", "433865533", "438637079", "441080312" ], "inchikey": "CBPNZQVSJQDFBE-FAYWIXGWSA-N", "generic_name": "Temsirolimus", "trade_name": "Torisel", "designated_date": "2004-12-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of advanced renal cell carcinoma", "marketing_approval_date": "2007-05-30", "exclusivity_end_date": "2014-05-30", "sponsor": "Wyeth Pharmaceuticals, Inc.|P. O. Box 8299|Phildelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "Treatment of renal cell carcinoma", "umls": "C0007134", "parsed_text": "Renal Cell Carcinoma" } }, { "pubchem_cid": 148191, "pubchem_sid": [ "12014940", "56313948", "103771165", "131346349", "135105386", "144206068", "152258857", "160647706", "160967858", "162172159", "174527830", "175437917", "178102515", "204359550", "252158731", "254760066", "310280528", "312311726", "312616372", "315678400", "316541660", "318153060", "318163458", "332864831", "341100968", "341142655", "341854972", "354335686", "375577136", "384568322", "384572156", "385638415", "386632609", "406722223", "419480166", "434315187", "438475185", "438493358", "440786213", "53788684", "126666958", "252216390", "273316948", "313082308", "319332855", "329891082", "329978376", "349986356", "381391551", "382156887", "436380077", "440790951", "441016339", "17396179", "99443243", "223444871", "312228938", "332852674", "348284052", "426257242", "126617270", "196112710", "251971410", "313075773", "316900193", "318242896", "319166069", "387078114", "163123459", "319411484", "340502971", "347235644", "405460216", "440006878", "50070177", "162249026", "319340263", "319424078", "348814616", "355040167", "375664784", "377461485", "381183588", "381940665", "406531701", "426260554", "520036", "50883861", "162011848", "223434992", "252160375", "312811417", "319363586", "319379126", "319397925", "319399230", "319407906", "319409097", "319413543", "319413592", "319416085", "319417630", "319417970", "342547077", "348353832", "354265908", "364196441", "375562992", "422710142", "426405759", "433865533", "438637079", "441080312" ], "inchikey": "CBPNZQVSJQDFBE-UHFFFAOYSA-N", "generic_name": "Temsirolimus", "trade_name": "Torisel", "designated_date": "2004-12-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of advanced renal cell carcinoma", "marketing_approval_date": "2007-05-30", "exclusivity_end_date": "2014-05-30", "sponsor": "Wyeth Pharmaceuticals, Inc.|P. O. Box 8299|Phildelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "Treatment of renal cell carcinoma", "umls": "C0007134", "parsed_text": "Renal Cell Carcinoma" } }, { "pubchem_cid": 23724530, "pubchem_sid": [ "12014940", "56313948", "103771165", "131346349", "135105386", "144206068", "152258857", "160647706", "160967858", "162172159", "174527830", "175437917", "178102515", "204359550", "252158731", "254760066", "310280528", "312311726", "312616372", "315678400", "316541660", "318153060", "318163458", "332864831", "341100968", "341142655", "341854972", "354335686", "375577136", "384568322", "384572156", "385638415", "386632609", "406722223", "419480166", "434315187", "438475185", "438493358", "440786213", "53788684", "126666958", "252216390", "273316948", "313082308", "319332855", "329891082", "329978376", "349986356", "381391551", "382156887", "436380077", "440790951", "441016339", "17396179", "99443243", "223444871", "312228938", "332852674", "348284052", "426257242", "126617270", "196112710", "251971410", "313075773", "316900193", "318242896", "319166069", "387078114", "163123459", "319411484", "340502971", "347235644", "405460216", "440006878", "50070177", "162249026", "319340263", "319424078", "348814616", "355040167", "375664784", "377461485", "381183588", "381940665", "406531701", "426260554", "520036", "50883861", "162011848", "223434992", "252160375", "312811417", "319363586", "319379126", "319397925", "319399230", "319407906", "319409097", "319413543", "319413592", "319416085", "319417630", "319417970", "342547077", "348353832", "354265908", "364196441", "375562992", "422710142", "426405759", "433865533", "438637079", "441080312" ], "inchikey": "CBPNZQVSJQDFBE-HGVVHKDOSA-N", "generic_name": "Temsirolimus", "trade_name": "Torisel", "designated_date": "2004-12-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of advanced renal cell carcinoma", "marketing_approval_date": "2007-05-30", "exclusivity_end_date": "2014-05-30", "sponsor": "Wyeth Pharmaceuticals, Inc.|P. O. Box 8299|Phildelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "Treatment of renal cell carcinoma", "umls": "C0007134", "parsed_text": "Renal Cell Carcinoma" } }, { "pubchem_cid": 18293306, "pubchem_sid": [ "12014940", "56313948", "103771165", "131346349", "135105386", "144206068", "152258857", "160647706", "160967858", "162172159", "174527830", "175437917", "178102515", "204359550", "252158731", "254760066", "310280528", "312311726", "312616372", "315678400", "316541660", "318153060", "318163458", "332864831", "341100968", "341142655", "341854972", "354335686", "375577136", "384568322", "384572156", "385638415", "386632609", "406722223", "419480166", "434315187", "438475185", "438493358", "440786213", "53788684", "126666958", "252216390", "273316948", "313082308", "319332855", "329891082", "329978376", "349986356", "381391551", "382156887", "436380077", "440790951", "441016339", "17396179", "99443243", "223444871", "312228938", "332852674", "348284052", "426257242", "126617270", "196112710", "251971410", "313075773", "316900193", "318242896", "319166069", "387078114", "163123459", "319411484", "340502971", "347235644", "405460216", "440006878", "50070177", "162249026", "319340263", "319424078", "348814616", "355040167", "375664784", "377461485", "381183588", "381940665", "406531701", "426260554", "520036", "50883861", "162011848", "223434992", "252160375", "312811417", "319363586", "319379126", "319397925", "319399230", "319407906", "319409097", "319413543", "319413592", "319416085", "319417630", "319417970", "342547077", "348353832", "354265908", "364196441", "375562992", "422710142", "426405759", "433865533", "438637079", "441080312" ], "inchikey": "CBPNZQVSJQDFBE-PXVOFZQNSA-N", "generic_name": "Temsirolimus", "trade_name": "Torisel", "designated_date": "2004-12-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of advanced renal cell carcinoma", "marketing_approval_date": "2007-05-30", "exclusivity_end_date": "2014-05-30", "sponsor": "Wyeth Pharmaceuticals, Inc.|P. O. Box 8299|Phildelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "Treatment of renal cell carcinoma", "umls": "C0007134", "parsed_text": "Renal Cell Carcinoma" } }, { "pubchem_cid": 6918289, "pubchem_sid": [ "12014940", "56313948", "103771165", "131346349", "135105386", "144206068", "152258857", "160647706", "160967858", "162172159", "174527830", "175437917", "178102515", "204359550", "252158731", "254760066", "310280528", "312311726", "312616372", "315678400", "316541660", "318153060", "318163458", "332864831", "341100968", "341142655", "341854972", "354335686", "375577136", "384568322", "384572156", "385638415", "386632609", "406722223", "419480166", "434315187", "438475185", "438493358", "440786213", "53788684", "126666958", "252216390", "273316948", "313082308", "319332855", "329891082", "329978376", "349986356", "381391551", "382156887", "436380077", "440790951", "441016339", "17396179", "99443243", "223444871", "312228938", "332852674", "348284052", "426257242", "126617270", "196112710", "251971410", "313075773", "316900193", "318242896", "319166069", "387078114", "163123459", "319411484", "340502971", "347235644", "405460216", "440006878", "50070177", "162249026", "319340263", "319424078", "348814616", "355040167", "375664784", "377461485", "381183588", "381940665", "406531701", "426260554", "520036", "50883861", "162011848", "223434992", "252160375", "312811417", "319363586", "319379126", "319397925", "319399230", "319407906", "319409097", "319413543", "319413592", "319416085", "319417630", "319417970", "342547077", "348353832", "354265908", "364196441", "375562992", "422710142", "426405759", "433865533", "438637079", "441080312" ], "inchikey": "CBPNZQVSJQDFBE-FUXHJELOSA-N", "generic_name": "Temsirolimus", "trade_name": "Torisel", "designated_date": "2004-12-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of advanced renal cell carcinoma", "marketing_approval_date": "2007-05-30", "exclusivity_end_date": "2014-05-30", "sponsor": "Wyeth Pharmaceuticals, Inc.|P. O. Box 8299|Phildelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "Treatment of renal cell carcinoma", "umls": "C0007134", "parsed_text": "Renal Cell Carcinoma" } }, { "pubchem_cid": 148189, "pubchem_sid": [ "50046948", "74930025", "85334769", "103347250", "134223672", "135105384", "162222173", "163359859", "175427030", "252215604", "315726859", "316967053", "318161375", "319166068", "341244788", "348805614", "349974527", "355060186", "385790357", "386627518", "406810577", "419525873", "434297003", "438460214", "74930026", "254760054", "347760442", "74930024", "74930027", "74930028", "441076479" ], "inchikey": "NICJCIQSJJKZAH-AWEZNQCLSA-N", "generic_name": "irofulven", "designated_date": "1999-07-27", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Eisai, Inc.|300 Tice Blvd|Woodcliff Lake|New Jersey|7677|United States", "orphan_designation": { "original_text": "Treatment of renal cell carcinoma.", "umls": "C0007134", "parsed_text": "Renal Cell Carcinoma" } }, { "pubchem_cid": 323, "pubchem_sid": [ "8144", "74390", "585009", "587971", "603928", "7886722", "8025192", "8144283", "10537828", "11533015", "14716586", "17389959", "24698238", "24771116", "29203983", "46509134", "48413487", "48415819", "48423170", "48424987", "49693401", "49856031", "51073612", "53790693", "56312206", "56314271", "56422867", "85084963", "85148751", "85164923", "87565523", "90944856", "90944862", "90944869", "103164854", "103825471", "117588942", "117595185", "126678379", "127262255", "127262256", "127262257", "127262258", "127262259", "127262260", "127262261", "127262262", "127262263", "127262264", "127262265", "127262266", "127262267", "127262268", "127262269", "127262270", "127262271", "127262272", "127262273", "127262274", "127262275", "127262276", "127262277", "127262278", "127262279", "127262280", "127262281", "127262282", "127262283", "127262284", "127262285", "127262286", "127262287", 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Approved for Orphan Indication", "sponsor": "Heidelberg Pharma AG|101 Schriesheimer Stra\u07e5|Ladenburg|Baden-Wrttemberg|Germany", "orphan_designation": { "original_text": "Treatment of renal cell carcinoma.", "umls": "C0007134", "parsed_text": "Renal Cell Carcinoma" } }, { "pubchem_sid": 249565668, "generic_name": "Girentuximab", "designated_date": "2001-03-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Heidelberg Pharma AG|101 Schriesheimer Stra\u07e5|Ladenburg|Baden-Wrttemberg|Germany", "orphan_designation": { "original_text": "Treatment of renal cell carcinoma.", "umls": "C0007134", "parsed_text": "Renal Cell Carcinoma" } }, { "pubchem_sid": 160666627, "generic_name": "Girentuximab", "designated_date": "2001-03-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Heidelberg Pharma AG|101 Schriesheimer Stra\u07e5|Ladenburg|Baden-Wrttemberg|Germany", 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O. Box 368|900 Ridgebury Road|Ridgefield|Connecticut|6877|United States", "orphan_designation": { "original_text": "To improve pulmonary function in conjunction with standard therapy in the management of patients with cystic fibrosis", "umls": [ "C0010674", "C1547996" ], "parsed_text": [ "Cystic Fibrosis", "Pulmonary Function" ] } }, { "pubchem_cid": 7059524, "pubchem_sid": [ "604404", "26683847", "49836678", "49973715", "53789767", "57264288", "103258901", "135060321", "162228241", "223746147", "241100231", "241181769", "252469295", "274050275", "310278048", "312226797", "319137130", "347238591", "348276343", "355201223", "375131249", "375671520", "376220031", "385017167", "419569864", "438475357", "439592477", "26759267", "56310915", "162092488", "163687584", "315678231", "341161564", "385640789", "386294839", "386635052", "50106655", "50106656", "92304111", "163564771", "268738355", "312344941", "585107", "332853587", "348267250", "438554011" ], "inchikey": 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"urea", "designated_date": "2011-11-07", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Orenova Group, LLC|10 New King Street|Suite 106|White Plains|New York|10604|United States", "orphan_designation": { "original_text": "Treatment of rare congenital ichthyoses (CHILD syndrome, collodion baby, congenital ichthyosform erythroderma, Conradi-flunermann, epidermolytic hyperkeratosis, erythrokeratoderma variabilis, harlequin ichthyosis, KID syndrome, lamellar ichthyosis, Netherton syndrome, neutral lipid storage disease, Sjorgren-Larsson syndrome, trichothiodystrophy, X-linked ichthyosis).", "umls": [ "C0011606", "C0079153", "C0079154", "C0079588", "C0239849", "C0265267", "C0265962", "C0265962", "C0268238", "C1955934", "C3543867", "C3665333" ], "parsed_text": [ "Erythroderma", "Epidermolytic hyperkeratosis", "Lamellar ichthyosis", "X-linked ichthyosis", "Harlequin Ichthyosis", "Child syndrome", "Netherton Syndrome", "Netherton Syndrome", "Neutral lipid storage disease", "Trichothiodystrophy", "Collodion Baby", "Kid syndrome" ] } } ] }, { "_id": "UMLS:C0079153", "fda_orphan_drug": [ { "pubchem_cid": 1176, "pubchem_sid": [ "3274", "3386", "92014", "584817", "589705", "599346", "855226", "7891021", "8007365", "8008401", "8144973", "8149579", "10527796", "11110202", "11110234", "14709415", "17389689", "17435981", "17435984", "17436160", "24424275", "24424281", "38671673", "38671682", "46508687", "48414618", "48416732", "48424272", "48425726", "49640652", "49655242", "49855531", "53788128", "56269912", "56314413", "56436181", "85090292", "85090294", "85090299", "85281224", "87544414", "87561630", "92297522", "93578211", "99226750", "99226755", "103258138", "104179569", "104296984", "117526279", "121270351", "121270605", "123055040", "124391969", "125165283", "125307123", "126677730", "126723148", "126723153", "131465355", "131500181", "131500183", "134220905", "134970701", "136348432", "144075273", "144088965", 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"C0079153", "C0079154", "C0079588", "C0239849", "C0265267", "C0265962", "C0265962", "C0268238", "C1955934", "C3543867", "C3665333" ], "parsed_text": [ "Erythroderma", "Epidermolytic hyperkeratosis", "Lamellar ichthyosis", "X-linked ichthyosis", "Harlequin Ichthyosis", "Child syndrome", "Netherton Syndrome", "Netherton Syndrome", "Neutral lipid storage disease", "Trichothiodystrophy", "Collodion Baby", "Kid syndrome" ] } } ] }, { "_id": "MONDO:0010622", "fda_orphan_drug": [ { "pubchem_cid": 1176, "pubchem_sid": [ "3274", "3386", "92014", "584817", "589705", "599346", "855226", "7891021", "8007365", "8008401", "8144973", "8149579", "10527796", "11110202", "11110234", "14709415", "17389689", "17435981", "17435984", "17436160", "24424275", "24424281", "38671673", "38671682", "46508687", "48414618", "48416732", "48424272", "48425726", "49640652", "49655242", "49855531", "53788128", "56269912", "56314413", "56436181", "85090292", "85090294", "85090299", "85281224", "87544414", "87561630", 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congenital ichthyosform erythroderma, Conradi-flunermann, epidermolytic hyperkeratosis, erythrokeratoderma variabilis, harlequin ichthyosis, KID syndrome, lamellar ichthyosis, Netherton syndrome, neutral lipid storage disease, Sjorgren-Larsson syndrome, trichothiodystrophy, X-linked ichthyosis).", "umls": [ "C0011606", "C0079153", "C0079154", "C0079588", "C0239849", "C0265267", "C0265962", "C0265962", "C0268238", "C1955934", "C3543867", "C3665333" ], "parsed_text": [ "Erythroderma", "Epidermolytic hyperkeratosis", "Lamellar ichthyosis", "X-linked ichthyosis", "Harlequin Ichthyosis", "Child syndrome", "Netherton Syndrome", "Netherton Syndrome", "Neutral lipid storage disease", "Trichothiodystrophy", "Collodion Baby", "Kid syndrome" ] } } ] }, { "_id": "MONDO:0009443", "fda_orphan_drug": [ { "pubchem_cid": 1176, "pubchem_sid": [ "3274", "3386", "92014", "584817", "589705", "599346", "855226", "7891021", "8007365", "8008401", "8144973", "8149579", "10527796", "11110202", "11110234", 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"2011-11-07", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Orenova Group, LLC|10 New King Street|Suite 106|White Plains|New York|10604|United States", "orphan_designation": { "original_text": "Treatment of rare congenital ichthyoses (CHILD syndrome, collodion baby, congenital ichthyosform erythroderma, Conradi-flunermann, epidermolytic hyperkeratosis, erythrokeratoderma variabilis, harlequin ichthyosis, KID syndrome, lamellar ichthyosis, Netherton syndrome, neutral lipid storage disease, Sjorgren-Larsson syndrome, trichothiodystrophy, X-linked ichthyosis).", "umls": [ "C0011606", "C0079153", "C0079154", "C0079588", "C0239849", "C0265267", "C0265962", "C0265962", "C0268238", "C1955934", "C3543867", "C3665333" ], "parsed_text": [ "Erythroderma", "Epidermolytic hyperkeratosis", "Lamellar ichthyosis", "X-linked ichthyosis", "Harlequin Ichthyosis", "Child syndrome", "Netherton Syndrome", "Netherton Syndrome", "Neutral lipid storage disease", "Trichothiodystrophy", "Collodion Baby", "Kid syndrome" ] } } ] }, { "_id": "MONDO:0010621", "fda_orphan_drug": [ { "pubchem_cid": 1176, "pubchem_sid": [ "3274", "3386", "92014", "584817", "589705", "599346", "855226", "7891021", "8007365", "8008401", "8144973", "8149579", "10527796", "11110202", "11110234", "14709415", "17389689", "17435981", "17435984", "17436160", "24424275", "24424281", "38671673", "38671682", "46508687", "48414618", "48416732", "48424272", "48425726", "49640652", "49655242", "49855531", "53788128", "56269912", "56314413", "56436181", "85090292", "85090294", "85090299", "85281224", "87544414", "87561630", "92297522", "93578211", "99226750", "99226755", "103258138", "104179569", "104296984", "117526279", "121270351", "121270605", "123055040", "124391969", "125165283", "125307123", "126677730", "126723148", "126723153", "131465355", "131500181", "131500183", "134220905", "134970701", "136348432", "144075273", "144088965", "144187553", "144187559", 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"C0079588", "C0239849", "C0265267", "C0265962", "C0265962", "C0268238", "C1955934", "C3543867", "C3665333" ], "parsed_text": [ "Erythroderma", "Epidermolytic hyperkeratosis", "Lamellar ichthyosis", "X-linked ichthyosis", "Harlequin Ichthyosis", "Child syndrome", "Netherton Syndrome", "Netherton Syndrome", "Neutral lipid storage disease", "Trichothiodystrophy", "Collodion Baby", "Kid syndrome" ] } } ] }, { "_id": "UMLS:C0268238", "fda_orphan_drug": [ { "pubchem_cid": 1176, "pubchem_sid": [ "3274", "3386", "92014", "584817", "589705", "599346", "855226", "7891021", "8007365", "8008401", "8144973", "8149579", "10527796", "11110202", "11110234", "14709415", "17389689", "17435981", "17435984", "17436160", "24424275", "24424281", "38671673", "38671682", "46508687", "48414618", "48416732", "48424272", "48425726", "49640652", "49655242", "49855531", "53788128", "56269912", "56314413", "56436181", "85090292", "85090294", "85090299", "85281224", "87544414", "87561630", "92297522", "93578211", 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"Trichothiodystrophy", "Collodion Baby", "Kid syndrome" ] } } ] }, { "_id": "UMLS:C3543867", "fda_orphan_drug": [ { "pubchem_cid": 1176, "pubchem_sid": [ "3274", "3386", "92014", "584817", "589705", "599346", "855226", "7891021", "8007365", "8008401", "8144973", "8149579", "10527796", "11110202", "11110234", "14709415", "17389689", "17435981", "17435984", "17436160", "24424275", "24424281", "38671673", "38671682", "46508687", "48414618", "48416732", "48424272", "48425726", "49640652", "49655242", "49855531", "53788128", "56269912", "56314413", "56436181", "85090292", "85090294", "85090299", "85281224", "87544414", "87561630", "92297522", "93578211", "99226750", "99226755", "103258138", "104179569", "104296984", "117526279", "121270351", "121270605", "123055040", "124391969", "125165283", "125307123", "126677730", "126723148", "126723153", "131465355", "131500181", "131500183", "134220905", "134970701", "136348432", "144075273", "144088965", "144187553", "144187559", "144204587", 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"C0239849", "C0265267", "C0265962", "C0265962", "C0268238", "C1955934", "C3543867", "C3665333" ], "parsed_text": [ "Erythroderma", "Epidermolytic hyperkeratosis", "Lamellar ichthyosis", "X-linked ichthyosis", "Harlequin Ichthyosis", "Child syndrome", "Netherton Syndrome", "Netherton Syndrome", "Neutral lipid storage disease", "Trichothiodystrophy", "Collodion Baby", "Kid syndrome" ] } } ] }, { "_id": "UMLS:C3665333", "fda_orphan_drug": [ { "pubchem_cid": 1176, "pubchem_sid": [ "3274", "3386", "92014", "584817", "589705", "599346", "855226", "7891021", "8007365", "8008401", "8144973", "8149579", "10527796", "11110202", "11110234", "14709415", "17389689", "17435981", "17435984", "17436160", "24424275", "24424281", "38671673", "38671682", "46508687", "48414618", "48416732", "48424272", "48425726", "49640652", "49655242", "49855531", "53788128", "56269912", "56314413", "56436181", "85090292", "85090294", "85090299", "85281224", "87544414", "87561630", "92297522", "93578211", "99226750", 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Conradi-flunermann, epidermolytic hyperkeratosis, erythrokeratoderma variabilis, harlequin ichthyosis, KID syndrome, lamellar ichthyosis, Netherton syndrome, neutral lipid storage disease, Sjorgren-Larsson syndrome, trichothiodystrophy, X-linked ichthyosis).", "umls": [ "C0011606", "C0079153", "C0079154", "C0079588", "C0239849", "C0265267", "C0265962", "C0265962", "C0268238", "C1955934", "C3543867", "C3665333" ], "parsed_text": [ "Erythroderma", "Epidermolytic hyperkeratosis", "Lamellar ichthyosis", "X-linked ichthyosis", "Harlequin Ichthyosis", "Child syndrome", "Netherton Syndrome", "Netherton Syndrome", "Neutral lipid storage disease", "Trichothiodystrophy", "Collodion Baby", "Kid syndrome" ] } } ] }, { "_id": "MONDO:0006823", "fda_orphan_drug": [ { "pubchem_cid": 73759969, "pubchem_sid": [ "50305517", "319104121", "319104122", "341137140", "349977047", "355169217", "385782102", "386618838", "405514332", "440060988", "440790969", "135360587", "252543140", "319416343", "319416344", 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"385782102", "386618838", "405514332", "440060988", "440790969", "135360587", "252543140", "319416343", "319416344", "341114284", "347660804", "375580919", "135020747", "176262070", "319416063", "7980866", "46507385", "103771339", "135358620", "135651747", "316906349", "342552139", "349512679", "438543441" ], "inchikey": "ZDRRIRUAESZNIH-BZGUUIOASA-N", "generic_name": "Urofollitropin", "designated_date": "1997-12-05", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "EMD Serono, Inc.|One Technology Place|Rockland|Massachusetts|2370|United States", "orphan_designation": { "original_text": "For the initiation and re-initiation of spermatogenesis in adult males with reproductive failure due to hypothalamic or pituitary dysfunction, hypogonadotropic hypogonadism.", "umls": [ "C0022735", "C0281947", "C3671958" ], "parsed_text": [ "Hypogonadotropic Hypogonadism", "pituitary dysfunction", "Reproductive failure" ] } }, { "pubchem_cid": 62819, "pubchem_sid": [ "50305517", "319104121", "319104122", "341137140", "349977047", "355169217", "385782102", "386618838", "405514332", "440060988", "440790969", "135360587", "252543140", "319416343", "319416344", "341114284", "347660804", "375580919", "135020747", "176262070", "319416063", "7980866", "46507385", "103771339", "135358620", "135651747", "316906349", "342552139", "349512679", "438543441" ], "inchikey": "ZDRRIRUAESZNIH-UHFFFAOYSA-N", "generic_name": "Urofollitropin", "designated_date": "1997-12-05", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "EMD Serono, Inc.|One Technology Place|Rockland|Massachusetts|2370|United States", "orphan_designation": { "original_text": "For the initiation and re-initiation of spermatogenesis in adult males with reproductive failure due to hypothalamic or pituitary dysfunction, hypogonadotropic hypogonadism.", "umls": [ "C0022735", "C0281947", "C3671958" ], "parsed_text": [ "Hypogonadotropic Hypogonadism", "pituitary dysfunction", "Reproductive failure" ] } }, { "pubchem_sid": 441076374, "generic_name": "Somatropin", "designated_date": "1987-09-01", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Novo Nordisk Pharmaceuticals|100 Overlook Center|Suite 200|Princeton|New Jersey|8540|United States", "orphan_designation": { "original_text": "Adjunct for the induction of ovulation in women with infertility due to hypogonadotropic hypogonadism or bilateral tubal occlusion or unexplained infertility, who are undergoing in vivo or in vitro fertilization procedures.", "umls": [ "C0021359", "C0022735", "C0404585" ], "parsed_text": [ "Infertility", "Hypogonadotropic Hypogonadism", "Unexplained infertility" ] } }, { "pubchem_sid": 178101641, "generic_name": "Somatropin", "designated_date": "1987-09-01", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Novo Nordisk Pharmaceuticals|100 Overlook Center|Suite 200|Princeton|New Jersey|8540|United States", "orphan_designation": { "original_text": "Adjunct for the induction of ovulation in women with infertility due to hypogonadotropic hypogonadism or bilateral tubal occlusion or unexplained infertility, who are undergoing in vivo or in vitro fertilization procedures.", "umls": [ "C0021359", "C0022735", "C0404585" ], "parsed_text": [ "Infertility", "Hypogonadotropic Hypogonadism", "Unexplained infertility" ] } }, { "pubchem_sid": 135274454, "generic_name": "Somatropin", "designated_date": "1987-09-01", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Novo Nordisk Pharmaceuticals|100 Overlook Center|Suite 200|Princeton|New Jersey|8540|United States", "orphan_designation": { "original_text": "Adjunct for the induction of ovulation in women with infertility due to hypogonadotropic hypogonadism or bilateral tubal occlusion or unexplained infertility, who are undergoing in vivo or in vitro fertilization procedures.", "umls": [ "C0021359", "C0022735", "C0404585" ], "parsed_text": [ "Infertility", "Hypogonadotropic Hypogonadism", "Unexplained infertility" ] } }, { "pubchem_sid": 103771423, "generic_name": "Somatropin", "designated_date": "1987-09-01", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Novo Nordisk Pharmaceuticals|100 Overlook Center|Suite 200|Princeton|New Jersey|8540|United States", "orphan_designation": { "original_text": "Adjunct for the induction of ovulation in women with infertility due to hypogonadotropic hypogonadism or bilateral tubal occlusion or unexplained infertility, who are undergoing in vivo or in vitro fertilization procedures.", "umls": [ "C0021359", "C0022735", "C0404585" ], "parsed_text": [ "Infertility", "Hypogonadotropic Hypogonadism", "Unexplained infertility" ] } }, { "pubchem_sid": 50046577, "generic_name": "Somatropin", "designated_date": "1987-09-01", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Novo Nordisk Pharmaceuticals|100 Overlook Center|Suite 200|Princeton|New Jersey|8540|United States", "orphan_designation": { "original_text": "Adjunct for the induction of ovulation in women with infertility due to hypogonadotropic hypogonadism or bilateral tubal occlusion or unexplained infertility, who are undergoing in vivo or in vitro fertilization procedures.", "umls": [ "C0021359", "C0022735", "C0404585" ], "parsed_text": [ "Infertility", "Hypogonadotropic Hypogonadism", "Unexplained infertility" ] } }, { "pubchem_sid": 46506010, "generic_name": "Somatropin", "designated_date": "1987-09-01", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Novo Nordisk Pharmaceuticals|100 Overlook Center|Suite 200|Princeton|New Jersey|8540|United States", "orphan_designation": { "original_text": "Adjunct for the induction of ovulation in women with infertility due to hypogonadotropic hypogonadism or bilateral tubal occlusion or unexplained infertility, who are undergoing in vivo or in vitro fertilization procedures.", "umls": [ "C0021359", "C0022735", "C0404585" ], "parsed_text": [ "Infertility", "Hypogonadotropic Hypogonadism", "Unexplained infertility" ] } } ] }, { "_id": "UMLS:C0281947", "fda_orphan_drug": [ { "pubchem_cid": 73759969, "pubchem_sid": [ "50305517", "319104121", "319104122", "341137140", "349977047", "355169217", "385782102", "386618838", "405514332", "440060988", "440790969", "135360587", "252543140", "319416343", "319416344", "341114284", "347660804", "375580919", "135020747", "176262070", "319416063", "7980866", "46507385", "103771339", "135358620", "135651747", "316906349", "342552139", "349512679", "438543441" ], "inchikey": "ZDRRIRUAESZNIH-JLSAMJFVSA-N", "generic_name": "Urofollitropin", "designated_date": "1997-12-05", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "EMD Serono, Inc.|One Technology Place|Rockland|Massachusetts|2370|United States", "orphan_designation": { "original_text": "For the initiation and re-initiation of spermatogenesis in adult males with reproductive failure due to hypothalamic or pituitary dysfunction, hypogonadotropic hypogonadism.", "umls": [ "C0022735", "C0281947", "C3671958" ], "parsed_text": [ "Hypogonadotropic Hypogonadism", "pituitary dysfunction", "Reproductive failure" ] } }, { "pubchem_cid": 75409771, "pubchem_sid": [ "50305517", "319104121", "319104122", "341137140", "349977047", "355169217", "385782102", "386618838", "405514332", "440060988", "440790969", "135360587", "252543140", "319416343", "319416344", "341114284", "347660804", "375580919", "135020747", "176262070", "319416063", "7980866", 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Hypogonadism", "pituitary dysfunction", "Reproductive failure" ] } }, { "pubchem_cid": 75409771, "pubchem_sid": [ "50305517", "319104121", "319104122", "341137140", "349977047", "355169217", "385782102", "386618838", "405514332", "440060988", "440790969", "135360587", "252543140", "319416343", "319416344", "341114284", "347660804", "375580919", "135020747", "176262070", "319416063", "7980866", "46507385", "103771339", "135358620", "135651747", "316906349", "342552139", "349512679", "438543441" ], "inchikey": "ZDRRIRUAESZNIH-BZGUUIOASA-N", "generic_name": "Urofollitropin", "designated_date": "1997-12-05", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "EMD Serono, Inc.|One Technology Place|Rockland|Massachusetts|2370|United States", "orphan_designation": { "original_text": "For the initiation and re-initiation of spermatogenesis in adult males with reproductive failure due to hypothalamic or pituitary dysfunction, hypogonadotropic hypogonadism.", "umls": [ "C0022735", "C0281947", "C3671958" ], "parsed_text": [ "Hypogonadotropic Hypogonadism", "pituitary dysfunction", "Reproductive failure" ] } }, { "pubchem_cid": 62819, "pubchem_sid": [ "50305517", "319104121", "319104122", "341137140", "349977047", "355169217", "385782102", "386618838", "405514332", "440060988", "440790969", "135360587", "252543140", "319416343", "319416344", "341114284", "347660804", "375580919", "135020747", "176262070", "319416063", "7980866", "46507385", "103771339", "135358620", "135651747", "316906349", "342552139", "349512679", "438543441" ], "inchikey": "ZDRRIRUAESZNIH-UHFFFAOYSA-N", "generic_name": "Urofollitropin", "designated_date": "1997-12-05", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "EMD Serono, Inc.|One Technology Place|Rockland|Massachusetts|2370|United States", "orphan_designation": { "original_text": "For the initiation and re-initiation of spermatogenesis in adult males with reproductive failure due to hypothalamic or pituitary dysfunction, hypogonadotropic hypogonadism.", "umls": [ "C0022735", "C0281947", "C3671958" ], "parsed_text": [ "Hypogonadotropic Hypogonadism", "pituitary dysfunction", "Reproductive failure" ] } } ] }, { "_id": "MONDO:0003749", "fda_orphan_drug": [ { "pubchem_cid": 133566183, "pubchem_sid": [ "46506923", "50472841", "135028044", "252157050", "316539782", "319332765", "319557371", "341148489", "354274927", "355036733", "363904445", "384442019", "434313605", "438492765", "50064081", "50472842", "136350435", "316967973", "319111466", "335987027", "363743247", "375611274", "375758806", "381973751", "404620993", "434136003", "438443417", "187072157", "316935715", "318025727", "319392015", "341852841", "160967829", "312228914", "348284024", "438594413", "160687027", "385782104", "386618840", "7980392", "50472840", "135652433", "319884268", "319885215", "366663284", "368501506" ], 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Street, #3F|New York|New York|10023|United States", "orphan_designation": { "original_text": "Treatment of megaloblastic anemia caused by folate deficiency", "umls": [ "C0002888", "C0016412" ], "parsed_text": [ "Megaloblastic anemia", "Folate deficiency" ] } } ] }, { "_id": "UMLS:C0016412", "fda_orphan_drug": [ { "pubchem_cid": 135564391, "pubchem_sid": [ "123101352", "135253403", "319358628", "319484944", "329980422", "355039778", "376024677", "382156364", "385751633", "386588285", "387061111", "434298427", "438460394", "440790822", "163409224", "313077165", "319340175", "348792444", "126659582", "318163731", "319408717", "319412205", "341744742", "375648924" ], "inchikey": "VWBBRFHSPXRJQD-QNTKWALQSA-L", "generic_name": "levomefolate calcium", "designated_date": "2015-02-10", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Cox Biosciences LLC|267 West 71st Street, #3F|New York|New York|10023|United States", 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Box 331329|Fort Worth|Texas|76163|United States", "orphan_designation": { "original_text": "Treatment of perioral dermatitis.", "umls": "C0263449", "parsed_text": "Perioral Dermatitis" } } ] }, { "_id": "UMLS:C1370932", "fda_orphan_drug": [ { "pubchem_cid": 5746, "pubchem_sid": [ "48414068", "49854359", "53787087", "134970853", "160963653", "319063729", "349990572", "349995294", "363898364", "386204510" ], "inchikey": "NWIBSHFKIJFRCO-WUDYKRTCSA-N", "generic_name": "mitomycin", "trade_name": "Jelmyto", "designated_date": "2014-09-08", "designation_status": "Designated/Approved", "approved_labeled_indication": "JELMYTO is indicated for the treatment of adult patients with low-grade Upper Tract Urothelial Cancer (LG-UTUC)", "marketing_approval_date": "2020-04-15", "exclusivity_end_date": "2027-04-15", "exclusivity_protected_indication": "Indicated for the treatment of adult patients with low-grade Upper Tract Urothelial Cancer (LG-UTUC).", "sponsor": "UroGen Pharma, Ltd.|9 Ha'Ta'asiya St., POB 2397|Ra'anana|Israel", "orphan_designation": { "original_text": "Treatment of upper tract urothelial cell cancers (transitional cell carcinoma of the renal pelvis and ureter)", "umls": [ "C1370932", "C4087468" ], "parsed_text": [ "carcinoma of the renal pelvis and ureter", "Transitional Cell Carcinoma of the Renal Pelvis" ] } } ] }, { "_id": "UMLS:C4087468", "fda_orphan_drug": [ { "pubchem_cid": 5746, "pubchem_sid": [ "48414068", "49854359", "53787087", "134970853", "160963653", "319063729", "349990572", "349995294", "363898364", "386204510" ], "inchikey": "NWIBSHFKIJFRCO-WUDYKRTCSA-N", "generic_name": "mitomycin", "trade_name": "Jelmyto", "designated_date": "2014-09-08", "designation_status": "Designated/Approved", "approved_labeled_indication": "JELMYTO is indicated for the treatment of adult patients with low-grade Upper Tract Urothelial Cancer (LG-UTUC)", "marketing_approval_date": "2020-04-15", "exclusivity_end_date": "2027-04-15", "exclusivity_protected_indication": "Indicated for the treatment of adult patients with low-grade Upper Tract Urothelial Cancer (LG-UTUC).", "sponsor": "UroGen Pharma, Ltd.|9 Ha'Ta'asiya St., POB 2397|Ra'anana|Israel", "orphan_designation": { "original_text": "Treatment of upper tract urothelial cell cancers (transitional cell carcinoma of the renal pelvis and ureter)", "umls": [ "C1370932", "C4087468" ], "parsed_text": [ "carcinoma of the renal pelvis and ureter", "Transitional Cell Carcinoma of the Renal Pelvis" ] } } ] }, { "_id": "MONDO:0019438", "fda_orphan_drug": [ { "pubchem_cid": 10038269, "pubchem_sid": [ "381125266", "426423761" ], "inchikey": "XIKSEDLKDYKPTO-UHFFFAOYSA-N", "generic_name": "N-(3,4-dihydroxyphenyl)-3,4-dihydroxybenzamide", "designated_date": "2014-10-15", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "ProtaMed, Inc.|14241 Woodinville Duvall Road, Suite # 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Box 8355|Wilmington|Delaware|19803|United States", "orphan_designation": { "original_text": "Treatment of Fallopian Tube Cancer", "umls": "C0238122", "parsed_text": "Fallopian Tube Cancer" } }, { "pubchem_cid": 23725625, "pubchem_sid": [ "85197660", "103605183", "126626885", "126644879", "126664211", "126666996", "131407195", "134222742", "134964396", "135261055", "135697767", "135708109", "136377786", "136895641", "152258100", "160646939", "160810829", "162058030", "162172208", "163088771", "187051821", "187072934", "223375126", "223736293", "251971206", "252159146", "252160170", "252215948", "254759126", "254761805", "255385074", "273884861", "275893966", "310278320", "312616324", "316580561", "316900182", "318163909", "319379284", "319436166", "319463087", "319530703", "319552093", "340506476", "340522971", "340548349", "341143310", "346435859", "347735953", "347827823", "349892423", "349988538", "350079491", "350080568", "350095305", "355040777", "371389352", "374073206", "375577677", "375581723", "375757262", "376033298", "376191636", "378130213", "381316845", "382371665", "384570915", "384572353", "385637718", "386244507", "386631899", "387020473", "387088344", "387250273", "402419171", "426257378", "433796082", "438507039", "440806628", "440999262", "441104665", "170559095", "402287500", "99218744", "164231742", "204362527", "221672931", "349485330", "364206739", "419498287" ], "inchikey": "FDLYAMZZIXQODN-UHFFFAOYSA-N", "generic_name": "olaparib", "trade_name": "Lynparza", "designated_date": "2018-05-15", "designation_status": "Designated/Approved", "approved_labeled_indication": "Lynparza is indicated for the maintenance treatment of adult patients with deleterious or suspected deleterious germline or somatic BRCA-mutated (gBRCAm or sBRCAm) advanced epithelial ovarian, fallopian tube or primary peritoneal cancer who are in complete or partial response to first-line platinum based chemotherapy. 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O. Box 61540|King of Prussia|Pennsylvania|19406|United States", "orphan_designation": { "original_text": "Treatment of new-onset type I diabetes mellitus", "umls": "C0011854", "parsed_text": "type i diabetes mellitus" } }, { "pubchem_sid": 252166668, "generic_name": "otelixizumab", "designated_date": "2006-02-06", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "GlaxoSmithKline|2301 Renaissance Blvd (RN0420)|P. O. Box 61540|King of Prussia|Pennsylvania|19406|United States", "orphan_designation": { "original_text": "Treatment of new-onset type I diabetes mellitus", "umls": "C0011854", "parsed_text": "type i diabetes mellitus" } }, { "pubchem_sid": 160666629, "generic_name": "otelixizumab", "designated_date": "2006-02-06", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "GlaxoSmithKline|2301 Renaissance Blvd (RN0420)|P. O. 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Box 61540|King of Prussia|Pennsylvania|19406|United States", "orphan_designation": { "original_text": "Treatment of new-onset type I diabetes mellitus", "umls": "C0011854", "parsed_text": "type i diabetes mellitus" } } ] }, { "_id": "UMLS:C0338427", "fda_orphan_drug": [ { "pubchem_cid": 3599, "pubchem_sid": [ "226520", "12014266", "26759174", "49966181", "53788176", "75270305", "103186893", "103942430", "126592939", "126645243", "126664051", "135030585", "136946455", "144075829", "144205019", "162173181", "162248636", "163136021", "163687676", "163835484", "170465622", "207116278", "210274930", "210280567", "223703158", "241100814", "250136791", "252352361", "252403054", "253660169", "254761337", "255385705", "255472386", "268736333", "275953906", "310264984", "310277248", "312636929", "312695133", "313081528", "315681576", "318153700", "318161670", "319062404", "319463380", "319554001", "336908992", "341142153", "346420637", "347758863", "349137307", "349974553", "350095980", 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amebic encephalitis", "PAM" ] } } ] }, { "_id": "MONDO:0018959", "fda_orphan_drug": [ { "pubchem_cid": 3599, "pubchem_sid": [ "226520", "12014266", "26759174", "49966181", "53788176", "75270305", "103186893", "103942430", "126592939", "126645243", "126664051", "135030585", "136946455", "144075829", "144205019", "162173181", "162248636", "163136021", "163687676", "163835484", "170465622", "207116278", "210274930", "210280567", "223703158", "241100814", "250136791", "252352361", "252403054", "253660169", "254761337", "255385705", "255472386", "268736333", "275953906", "310264984", "310277248", "312636929", "312695133", "313081528", "315681576", "318153700", "318161670", "319062404", "319463380", "319554001", "336908992", "341142153", "346420637", "347758863", "349137307", "349974553", "350095980", "355039540", "374048868", "374210771", "375154641", "375577638", "375586783", "375653581", "376146657", "376226254", "377355587", "382162760", "384416840", "385782522", "386619276", 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(USA)|Corporation Trust Center|1209 Orange Street|Wilmington|Delaware|19801|United States", "orphan_designation": { "original_text": "Treatment of leishmaniasis.", "umls": "C0023281", "parsed_text": "Leishmaniasis" } }, { "pubchem_cid": 6710777, "pubchem_sid": [ "103614911", "336269635", "349984264", "405475973", "434302079", "438484073", "440022632" ], "inchikey": "SLVOKEOPLJCHCQ-UHFFFAOYSA-O", "generic_name": "oleylphosphocholine", "designated_date": "2013-10-25", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Oblita Therapeutics bvba|Rogendijk 40|Zoersel|Belgium", "orphan_designation": { "original_text": "Treatment of leishmaniasis", "umls": "C0023281", "parsed_text": "Leishmaniasis" } }, { "pubchem_cid": 9932595, "pubchem_sid": [ "103614911", "336269635", "349984264", "405475973", "434302079", "438484073", "440022632" ], "inchikey": "SLVOKEOPLJCHCQ-SEYXRHQNSA-N", "generic_name": "oleylphosphocholine", "designated_date": 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"orphan_designation": { "original_text": "Treatment of diffuse non-Hodgkin's lymphoma, including AIDS-related diffuse non-Hodgkin's lymphoma.", "umls": [ "C0001175", "C0348394" ], "parsed_text": [ "AIDS", "Diffuse non-Hodgkin's lymphoma" ] } }, { "pubchem_cid": 5351154, "pubchem_sid": [ "611216", "12012645", "49974828", "77105216", "103504484", "103977023", "126591589", "126688706", "223442568", "241088157", "252559424", "254760962", "318041087", "318162965", "319298260", "347829107", "355181616", "404615922", "406167160", "406168308", "77105217", "249905171", "385639439", "386633650", "426078398", "434315414", "438493775", "77105218", "349067367", "375233855", "405486588", "440033246", "134973137", "384445465", "387126679", "543857", "549846", "77105219", "77105220", "77105221", "77105222", "127320128", "127320129", "127320130", "127320131", "127320132", "127320133", "127320134", "127320135", "127320136", "127320137", "127320138", "438637565", "439575205", "441071646" ], "inchikey": "MXWHMTNPTTVWDM-NXOFHUPFSA-N", "generic_name": "Mitoguazone", "designated_date": "1994-03-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "ILEX Oncology, Inc.|4545 Horizon Hill Blvd.|San Antonio|Texas|78229|United States", "orphan_designation": { "original_text": "Treatment of diffuse non-Hodgkin's lymphoma, including AIDS-related diffuse non-Hodgkin's lymphoma.", "umls": [ "C0001175", "C0348394" ], "parsed_text": [ "AIDS", "Diffuse non-Hodgkin's lymphoma" ] } } ] }, { "_id": "MONDO:0002801", "fda_orphan_drug": [ { "pubchem_cid": 4456, "pubchem_sid": [ "9467", "7980106", "46509161", "48416319", "49868416", "50765645", "53786926", "81044623", "93165415", "103933337", "111677888", "134972621", "160682474", "160964672", "176267024", "274087215", "310264771", "312228819", "315673937", "318161753", "319062879", "341106435", "341825746", "347944652", "348283908", "349982991", "355190418", "363603242", "363901029", "383819056", 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O. Box 9001|Shirley|New York|11967|United States", "orphan_designation": { "original_text": "Treatment of acute colonic pseudo-obstruction", "umls": "C0009377", "parsed_text": "Colonic Pseudo-Obstruction" } } ] }, { "_id": "MONDO:0002144", "fda_orphan_drug": [ { "pubchem_cid": 135398752, "pubchem_sid": [ "9801", "117503", "823708", "26697034", "50064614", "56394554", "57260128", "57264357", "78766632", "85088426", "85756464", "103224360", "134982401", "144211537", "162095823", "162209412", "164152652", "175426963", "176321029", "249979891", "254771797", "310278449", "312225893", "312694322", "313077606", "315677438", "319062986", "340517445", "341142063", "348274808", "349982454", "355079100", "355101896", "355116565", "355127636", "363604075", "375987859", "376073437", "376214668", "381317678", "384060588", "384257010", "385790437", "386627598", "387101915", "404769269", "433774764", "433775244", "433910282", "438471004", "405475189", "440021849", "441071805" ], "inchikey": "HXNFUBHNUDHIGC-UHFFFAOYSA-N", "generic_name": "Oxypurinol", "designated_date": "1998-11-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Cardiome Pharma Corp.|3650 Westbrook Mall|Vancouver|Canada", "orphan_designation": { "original_text": "Treatment of hyperuricemia in patients intolerant to allopurinol.", "umls": "C0740394", "parsed_text": "Hyperuricemia" } }, { "pubchem_sid": 347910417, "generic_name": "pegloticase", "trade_name": "Krystexxa", "designated_date": "2001-02-21", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of chronic gout in adult patients refractory to conventional treatment", "marketing_approval_date": "2010-09-14", "exclusivity_end_date": "2017-09-14", "sponsor": "Horizon Pharma Rheumatology, LLC|150 S. Saunders RoadDrive|Suite 400|Lake Forest|Illinois|60045|United States", "orphan_designation": { "original_text": "To control the clinical consequences of hyperuricemia in patients with severe gout in whom conventional therapy is contraindicated or has been ineffective.", "umls": [ "C0018099", "C0740394" ], "parsed_text": [ "Gout", "Hyperuricemia" ] } }, { "pubchem_sid": 210024028, "generic_name": "pegloticase", "trade_name": "Krystexxa", "designated_date": "2001-02-21", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of chronic gout in adult patients refractory to conventional treatment", "marketing_approval_date": "2010-09-14", "exclusivity_end_date": "2017-09-14", "sponsor": "Horizon Pharma Rheumatology, LLC|150 S. 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Box 5100|Wallingford|Connecticut|6492|United States", "orphan_designation": { "original_text": "Treatment of refractory childhood acute lymphocytic leukemia.", "umls": "C0023452", "parsed_text": "Childhood Acute Lymphocytic Leukemia" } }, { "pubchem_cid": 134694361, "pubchem_sid": [ "597750", "7980753", "46507536", "49965048", "51063681", "53788607", "103589230", "134996895", "136342519", "162174797", "163620769", "163686094", "178103449", "184811965", "187072639", "223384958", "241050196", "251916159", "251917507", "252220258", "252390155", "252401148", "252451125", "254760638", "274086943", "310280530", "312694428", "315492991", "318162390", "319224575", "319554060", "322094616", "341835404", "347247495", "348955702", "349975478", "355040008", "363900008", "374383352", "375201719", "375303160", "375683044", "377363221", "381327005", "381379685", "381832363", "382159607", "384428756", "384568841", "384572884", "385783570", "386620411", "387060799", "401961199", "404760992", "433780032", "434123408", "434131442", "438475183", "440064782", "440790457", "440814161", "441008584", "26732588", "51063679", "99214913", "126670671", "163123657", "250127411", "252659762", "312654760", "319063501", "332872472", "387130411", "387209890", "405478151", "440024810", "8140078", "51063680", "51063683", "92719254", "126620951", "126653280", "164347276", "196111601", "253662241", "312228145", "318242301", "319086257", "319215055", "342476268", "348283120", "419590778", "438619773", "48416597", "51063682", "144204974", "315674173", "319297085", "341139721", "387130410", "51063685", "268734996", "319331929", "376226553", "13335", "319379120", "329872996", "377310732", "434315799", "438494490", "416461", "164234053", "319399314", "319413544", "51063684", "126599440", "242083286", "313098677", "318153266", "318695273", "346441011", "369183227", "375580219", "441073933" ], "inchikey": "NRUKOCRGYNPUPR-DYPKOKSYSA-N", "generic_name": "Teniposide", "trade_name": "Vumon for injection", "designated_date": "1984-11-01", "designation_status": "Designated/Approved", "approved_labeled_indication": "Induction therapy in patients with refractory childhood acute lymphoblastic leukemia when used in combination with other approved anticancer agents.", "marketing_approval_date": "1992-07-14", "exclusivity_end_date": "1999-07-14", "sponsor": "Bristol-Myers Squibb Pharmaceutical Research Institute|5 Research Parkway|P.O. Box 5100|Wallingford|Connecticut|6492|United States", "orphan_designation": { "original_text": "Treatment of refractory childhood acute lymphocytic leukemia.", "umls": "C0023452", "parsed_text": "Childhood Acute Lymphocytic Leukemia" } }, { "pubchem_cid": 122172961, "pubchem_sid": [ "597750", "7980753", "46507536", "49965048", "51063681", "53788607", "103589230", "134996895", "136342519", "162174797", "163620769", "163686094", "178103449", "184811965", "187072639", "223384958", "241050196", "251916159", "251917507", "252220258", "252390155", "252401148", "252451125", "254760638", "274086943", "310280530", "312694428", "315492991", "318162390", "319224575", "319554060", "322094616", "341835404", "347247495", "348955702", "349975478", "355040008", "363900008", "374383352", "375201719", "375303160", "375683044", "377363221", "381327005", "381379685", "381832363", "382159607", "384428756", "384568841", "384572884", "385783570", "386620411", "387060799", "401961199", "404760992", "433780032", "434123408", "434131442", "438475183", "440064782", "440790457", "440814161", "441008584", "26732588", "51063679", "99214913", "126670671", "163123657", "250127411", "252659762", "312654760", "319063501", "332872472", "387130411", "387209890", "405478151", "440024810", "8140078", "51063680", "51063683", "92719254", "126620951", "126653280", "164347276", "196111601", "253662241", "312228145", "318242301", "319086257", "319215055", "342476268", "348283120", "419590778", "438619773", "48416597", "51063682", "144204974", "315674173", "319297085", "341139721", "387130410", "51063685", "268734996", "319331929", "376226553", "13335", "319379120", "329872996", "377310732", "434315799", "438494490", "416461", "164234053", "319399314", "319413544", "51063684", "126599440", "242083286", "313098677", "318153266", "318695273", "346441011", "369183227", "375580219", "441073933" ], "inchikey": "NRUKOCRGYNPUPR-VEUOYOFCSA-N", "generic_name": "Teniposide", "trade_name": "Vumon for injection", "designated_date": "1984-11-01", "designation_status": "Designated/Approved", "approved_labeled_indication": "Induction therapy in patients with refractory childhood acute lymphoblastic leukemia when used in combination with other approved anticancer agents.", "marketing_approval_date": "1992-07-14", "exclusivity_end_date": "1999-07-14", "sponsor": "Bristol-Myers Squibb Pharmaceutical Research Institute|5 Research Parkway|P.O. Box 5100|Wallingford|Connecticut|6492|United States", "orphan_designation": { "original_text": "Treatment of refractory childhood acute lymphocytic leukemia.", "umls": "C0023452", "parsed_text": "Childhood Acute Lymphocytic Leukemia" } }, { "pubchem_cid": 90659141, "pubchem_sid": [ "597750", "7980753", "46507536", "49965048", "51063681", "53788607", "103589230", "134996895", "136342519", "162174797", "163620769", "163686094", "178103449", "184811965", "187072639", "223384958", "241050196", "251916159", "251917507", "252220258", "252390155", "252401148", "252451125", "254760638", "274086943", "310280530", "312694428", "315492991", "318162390", "319224575", "319554060", "322094616", "341835404", "347247495", "348955702", "349975478", "355040008", "363900008", "374383352", "375201719", "375303160", "375683044", "377363221", "381327005", "381379685", "381832363", "382159607", "384428756", "384568841", "384572884", "385783570", "386620411", "387060799", "401961199", "404760992", "433780032", "434123408", "434131442", "438475183", "440064782", "440790457", "440814161", "441008584", "26732588", "51063679", "99214913", "126670671", "163123657", "250127411", "252659762", "312654760", "319063501", "332872472", "387130411", "387209890", "405478151", "440024810", "8140078", "51063680", "51063683", "92719254", "126620951", "126653280", "164347276", "196111601", "253662241", "312228145", "318242301", "319086257", "319215055", "342476268", "348283120", "419590778", "438619773", "48416597", "51063682", "144204974", "315674173", "319297085", "341139721", "387130410", "51063685", "268734996", "319331929", "376226553", "13335", "319379120", "329872996", "377310732", "434315799", "438494490", "416461", "164234053", "319399314", "319413544", "51063684", "126599440", "242083286", "313098677", "318153266", "318695273", "346441011", "369183227", "375580219", "441073933" ], "inchikey": "NRUKOCRGYNPUPR-OMLXZSNTSA-N", "generic_name": "Teniposide", "trade_name": "Vumon for injection", "designated_date": "1984-11-01", "designation_status": "Designated/Approved", "approved_labeled_indication": "Induction therapy in patients with refractory childhood acute lymphoblastic leukemia when used in combination with other approved anticancer agents.", "marketing_approval_date": "1992-07-14", "exclusivity_end_date": "1999-07-14", "sponsor": "Bristol-Myers Squibb Pharmaceutical Research Institute|5 Research Parkway|P.O. Box 5100|Wallingford|Connecticut|6492|United States", "orphan_designation": { "original_text": "Treatment of refractory childhood acute lymphocytic leukemia.", "umls": "C0023452", "parsed_text": "Childhood Acute Lymphocytic Leukemia" } }, { "pubchem_cid": 54610154, "pubchem_sid": [ "597750", "7980753", "46507536", "49965048", "51063681", "53788607", "103589230", "134996895", "136342519", "162174797", "163620769", "163686094", "178103449", "184811965", "187072639", "223384958", "241050196", "251916159", "251917507", "252220258", "252390155", "252401148", "252451125", "254760638", "274086943", "310280530", "312694428", "315492991", "318162390", "319224575", "319554060", "322094616", "341835404", "347247495", "348955702", "349975478", "355040008", "363900008", "374383352", "375201719", "375303160", "375683044", "377363221", "381327005", "381379685", "381832363", "382159607", "384428756", "384568841", "384572884", "385783570", "386620411", "387060799", "401961199", "404760992", "433780032", "434123408", "434131442", "438475183", "440064782", "440790457", "440814161", "441008584", "26732588", "51063679", "99214913", "126670671", "163123657", "250127411", "252659762", "312654760", "319063501", "332872472", "387130411", "387209890", "405478151", "440024810", "8140078", "51063680", "51063683", "92719254", "126620951", "126653280", "164347276", "196111601", "253662241", "312228145", "318242301", "319086257", "319215055", "342476268", "348283120", "419590778", "438619773", "48416597", "51063682", "144204974", "315674173", "319297085", "341139721", "387130410", "51063685", "268734996", "319331929", "376226553", "13335", "319379120", "329872996", "377310732", "434315799", "438494490", "416461", "164234053", "319399314", "319413544", "51063684", "126599440", "242083286", "313098677", "318153266", "318695273", "346441011", "369183227", "375580219", "441073933" ], "inchikey": "NRUKOCRGYNPUPR-ANTWWVKXSA-N", "generic_name": "Teniposide", "trade_name": "Vumon for injection", "designated_date": "1984-11-01", "designation_status": "Designated/Approved", "approved_labeled_indication": "Induction therapy in patients with refractory childhood acute lymphoblastic leukemia when used in combination with other approved anticancer agents.", "marketing_approval_date": "1992-07-14", "exclusivity_end_date": "1999-07-14", "sponsor": "Bristol-Myers Squibb Pharmaceutical Research Institute|5 Research Parkway|P.O. Box 5100|Wallingford|Connecticut|6492|United States", "orphan_designation": { "original_text": "Treatment of refractory childhood acute lymphocytic leukemia.", "umls": "C0023452", "parsed_text": "Childhood Acute Lymphocytic Leukemia" } }, { "pubchem_cid": 129009948, "pubchem_sid": [ "597750", "7980753", "46507536", "49965048", "51063681", "53788607", "103589230", "134996895", "136342519", "162174797", "163620769", "163686094", "178103449", "184811965", "187072639", "223384958", "241050196", "251916159", "251917507", "252220258", "252390155", "252401148", "252451125", "254760638", "274086943", "310280530", "312694428", "315492991", "318162390", "319224575", "319554060", "322094616", "341835404", "347247495", "348955702", "349975478", "355040008", "363900008", "374383352", "375201719", "375303160", "375683044", "377363221", "381327005", "381379685", "381832363", "382159607", "384428756", "384568841", "384572884", "385783570", "386620411", "387060799", "401961199", "404760992", "433780032", "434123408", "434131442", "438475183", "440064782", "440790457", "440814161", "441008584", "26732588", "51063679", "99214913", "126670671", "163123657", "250127411", "252659762", "312654760", "319063501", "332872472", "387130411", "387209890", "405478151", "440024810", "8140078", "51063680", "51063683", "92719254", "126620951", "126653280", "164347276", "196111601", "253662241", "312228145", "318242301", "319086257", "319215055", "342476268", "348283120", "419590778", "438619773", "48416597", "51063682", "144204974", "315674173", "319297085", "341139721", "387130410", "51063685", "268734996", "319331929", "376226553", "13335", "319379120", "329872996", "377310732", "434315799", "438494490", "416461", "164234053", "319399314", "319413544", "51063684", "126599440", "242083286", "313098677", "318153266", "318695273", "346441011", "369183227", "375580219", "441073933" ], "inchikey": "NRUKOCRGYNPUPR-DKQCQWADSA-N", "generic_name": "Teniposide", "trade_name": "Vumon for injection", "designated_date": "1984-11-01", "designation_status": "Designated/Approved", "approved_labeled_indication": "Induction therapy in patients with refractory childhood acute lymphoblastic leukemia when used in combination with other approved anticancer agents.", "marketing_approval_date": "1992-07-14", "exclusivity_end_date": "1999-07-14", "sponsor": "Bristol-Myers Squibb Pharmaceutical Research Institute|5 Research Parkway|P.O. Box 5100|Wallingford|Connecticut|6492|United States", "orphan_designation": { "original_text": "Treatment of refractory childhood acute lymphocytic leukemia.", "umls": "C0023452", "parsed_text": "Childhood Acute Lymphocytic Leukemia" } }, { "pubchem_cid": 23724511, "pubchem_sid": [ "597750", "7980753", "46507536", "49965048", "51063681", "53788607", "103589230", "134996895", "136342519", "162174797", "163620769", "163686094", "178103449", "184811965", "187072639", "223384958", "241050196", "251916159", "251917507", "252220258", "252390155", "252401148", "252451125", "254760638", "274086943", "310280530", "312694428", "315492991", "318162390", "319224575", "319554060", "322094616", "341835404", "347247495", "348955702", "349975478", "355040008", "363900008", "374383352", "375201719", "375303160", "375683044", "377363221", "381327005", "381379685", "381832363", "382159607", "384428756", "384568841", "384572884", "385783570", "386620411", "387060799", "401961199", "404760992", "433780032", "434123408", "434131442", "438475183", "440064782", "440790457", "440814161", "441008584", "26732588", "51063679", "99214913", "126670671", "163123657", "250127411", "252659762", "312654760", "319063501", "332872472", "387130411", "387209890", "405478151", "440024810", "8140078", "51063680", "51063683", "92719254", "126620951", "126653280", "164347276", "196111601", "253662241", "312228145", "318242301", "319086257", "319215055", "342476268", "348283120", "419590778", "438619773", "48416597", "51063682", "144204974", "315674173", "319297085", "341139721", "387130410", "51063685", "268734996", "319331929", "376226553", "13335", "319379120", "329872996", "377310732", "434315799", "438494490", "416461", "164234053", "319399314", "319413544", "51063684", "126599440", "242083286", "313098677", "318153266", "318695273", "346441011", "369183227", "375580219", "441073933" ], "inchikey": "NRUKOCRGYNPUPR-ZVYBSHBNSA-N", "generic_name": "Teniposide", "trade_name": "Vumon for injection", "designated_date": "1984-11-01", "designation_status": "Designated/Approved", "approved_labeled_indication": "Induction therapy in patients with refractory childhood acute lymphoblastic leukemia when used in combination with other approved anticancer agents.", "marketing_approval_date": "1992-07-14", "exclusivity_end_date": "1999-07-14", "sponsor": "Bristol-Myers Squibb Pharmaceutical Research Institute|5 Research Parkway|P.O. Box 5100|Wallingford|Connecticut|6492|United States", "orphan_designation": { "original_text": "Treatment of refractory childhood acute lymphocytic leukemia.", "umls": "C0023452", "parsed_text": "Childhood Acute Lymphocytic Leukemia" } }, { "pubchem_cid": 6708778, "pubchem_sid": [ "597750", "7980753", "46507536", "49965048", "51063681", "53788607", "103589230", "134996895", "136342519", "162174797", "163620769", "163686094", "178103449", "184811965", "187072639", "223384958", "241050196", "251916159", "251917507", "252220258", "252390155", "252401148", "252451125", "254760638", "274086943", "310280530", "312694428", "315492991", "318162390", "319224575", "319554060", "322094616", "341835404", "347247495", "348955702", "349975478", "355040008", "363900008", "374383352", "375201719", "375303160", "375683044", "377363221", "381327005", "381379685", "381832363", "382159607", "384428756", "384568841", "384572884", "385783570", "386620411", "387060799", "401961199", "404760992", "433780032", "434123408", "434131442", "438475183", "440064782", "440790457", "440814161", "441008584", "26732588", "51063679", "99214913", "126670671", "163123657", "250127411", "252659762", "312654760", "319063501", "332872472", "387130411", "387209890", "405478151", "440024810", "8140078", "51063680", "51063683", "92719254", "126620951", "126653280", "164347276", "196111601", "253662241", "312228145", "318242301", "319086257", "319215055", "342476268", "348283120", "419590778", "438619773", "48416597", "51063682", "144204974", "315674173", "319297085", "341139721", "387130410", "51063685", "268734996", "319331929", "376226553", "13335", "319379120", "329872996", "377310732", "434315799", "438494490", "416461", "164234053", "319399314", "319413544", "51063684", "126599440", "242083286", "313098677", "318153266", "318695273", "346441011", "369183227", "375580219", "441073933" ], "inchikey": "NRUKOCRGYNPUPR-OQMCATNJSA-N", "generic_name": "Teniposide", "trade_name": "Vumon for injection", "designated_date": "1984-11-01", "designation_status": "Designated/Approved", "approved_labeled_indication": "Induction therapy in patients with refractory childhood acute lymphoblastic leukemia when used in combination with other approved anticancer agents.", "marketing_approval_date": "1992-07-14", "exclusivity_end_date": "1999-07-14", "sponsor": "Bristol-Myers Squibb Pharmaceutical Research Institute|5 Research Parkway|P.O. Box 5100|Wallingford|Connecticut|6492|United States", "orphan_designation": { "original_text": "Treatment of refractory childhood acute lymphocytic leukemia.", "umls": "C0023452", "parsed_text": "Childhood Acute Lymphocytic Leukemia" } }, { "pubchem_cid": 5284623, "pubchem_sid": [ "597750", "7980753", "46507536", "49965048", "51063681", "53788607", "103589230", "134996895", "136342519", "162174797", "163620769", "163686094", "178103449", "184811965", "187072639", "223384958", "241050196", "251916159", "251917507", "252220258", "252390155", "252401148", "252451125", "254760638", "274086943", "310280530", "312694428", "315492991", "318162390", "319224575", "319554060", "322094616", "341835404", "347247495", "348955702", "349975478", "355040008", "363900008", "374383352", "375201719", "375303160", "375683044", "377363221", "381327005", "381379685", "381832363", "382159607", "384428756", "384568841", "384572884", "385783570", "386620411", "387060799", "401961199", "404760992", "433780032", "434123408", "434131442", "438475183", "440064782", "440790457", "440814161", "441008584", "26732588", "51063679", "99214913", "126670671", "163123657", "250127411", "252659762", "312654760", "319063501", "332872472", "387130411", "387209890", "405478151", "440024810", "8140078", "51063680", "51063683", "92719254", "126620951", "126653280", "164347276", "196111601", "253662241", "312228145", "318242301", "319086257", "319215055", "342476268", "348283120", "419590778", "438619773", "48416597", "51063682", "144204974", "315674173", "319297085", "341139721", "387130410", "51063685", "268734996", "319331929", "376226553", "13335", "319379120", "329872996", "377310732", "434315799", "438494490", "416461", "164234053", "319399314", "319413544", "51063684", "126599440", "242083286", "313098677", "318153266", "318695273", "346441011", "369183227", "375580219", "441073933" ], "inchikey": "NRUKOCRGYNPUPR-NFIYYIGVSA-N", "generic_name": "Teniposide", "trade_name": "Vumon for injection", "designated_date": "1984-11-01", "designation_status": "Designated/Approved", "approved_labeled_indication": "Induction therapy in patients with refractory childhood acute lymphoblastic leukemia when used in combination with other approved anticancer agents.", "marketing_approval_date": "1992-07-14", "exclusivity_end_date": "1999-07-14", "sponsor": "Bristol-Myers Squibb Pharmaceutical Research Institute|5 Research Parkway|P.O. Box 5100|Wallingford|Connecticut|6492|United States", "orphan_designation": { "original_text": "Treatment of refractory childhood acute lymphocytic leukemia.", "umls": "C0023452", "parsed_text": "Childhood Acute Lymphocytic Leukemia" } }, { "pubchem_cid": 275784, "pubchem_sid": [ "597750", "7980753", "46507536", "49965048", "51063681", "53788607", "103589230", "134996895", "136342519", "162174797", "163620769", "163686094", "178103449", "184811965", "187072639", "223384958", "241050196", "251916159", "251917507", "252220258", "252390155", "252401148", "252451125", "254760638", "274086943", "310280530", "312694428", "315492991", "318162390", "319224575", "319554060", "322094616", "341835404", "347247495", "348955702", "349975478", "355040008", "363900008", "374383352", "375201719", "375303160", "375683044", "377363221", "381327005", "381379685", "381832363", "382159607", "384428756", "384568841", "384572884", "385783570", "386620411", "387060799", "401961199", "404760992", "433780032", "434123408", "434131442", "438475183", "440064782", "440790457", "440814161", "441008584", "26732588", "51063679", "99214913", "126670671", "163123657", "250127411", "252659762", "312654760", "319063501", "332872472", "387130411", "387209890", "405478151", "440024810", "8140078", "51063680", "51063683", "92719254", "126620951", "126653280", "164347276", "196111601", "253662241", "312228145", "318242301", "319086257", "319215055", "342476268", "348283120", "419590778", "438619773", "48416597", "51063682", "144204974", "315674173", "319297085", "341139721", "387130410", "51063685", "268734996", "319331929", "376226553", "13335", "319379120", "329872996", "377310732", "434315799", "438494490", "416461", "164234053", "319399314", "319413544", "51063684", "126599440", "242083286", "313098677", "318153266", "318695273", "346441011", "369183227", "375580219", "441073933" ], "inchikey": "NRUKOCRGYNPUPR-CRZAWEBOSA-N", "generic_name": "Teniposide", "trade_name": "Vumon for injection", "designated_date": "1984-11-01", "designation_status": "Designated/Approved", "approved_labeled_indication": "Induction therapy in patients with refractory childhood acute lymphoblastic leukemia when used in combination with other approved anticancer agents.", "marketing_approval_date": "1992-07-14", "exclusivity_end_date": "1999-07-14", "sponsor": "Bristol-Myers Squibb Pharmaceutical Research Institute|5 Research Parkway|P.O. Box 5100|Wallingford|Connecticut|6492|United States", "orphan_designation": { "original_text": "Treatment of refractory childhood acute lymphocytic leukemia.", "umls": "C0023452", "parsed_text": "Childhood Acute Lymphocytic Leukemia" } }, { "pubchem_cid": 34698, "pubchem_sid": [ "597750", "7980753", "46507536", "49965048", "51063681", "53788607", "103589230", "134996895", "136342519", "162174797", "163620769", "163686094", "178103449", "184811965", "187072639", "223384958", "241050196", "251916159", "251917507", "252220258", "252390155", "252401148", "252451125", "254760638", "274086943", "310280530", "312694428", "315492991", "318162390", "319224575", "319554060", "322094616", "341835404", "347247495", "348955702", "349975478", "355040008", "363900008", "374383352", "375201719", "375303160", "375683044", "377363221", "381327005", "381379685", "381832363", "382159607", "384428756", "384568841", "384572884", "385783570", "386620411", "387060799", "401961199", "404760992", "433780032", "434123408", "434131442", "438475183", "440064782", "440790457", "440814161", "441008584", "26732588", "51063679", "99214913", "126670671", "163123657", "250127411", "252659762", "312654760", "319063501", "332872472", "387130411", "387209890", "405478151", "440024810", "8140078", "51063680", "51063683", "92719254", "126620951", "126653280", "164347276", "196111601", "253662241", "312228145", "318242301", "319086257", "319215055", "342476268", "348283120", "419590778", "438619773", "48416597", "51063682", "144204974", "315674173", "319297085", "341139721", "387130410", "51063685", "268734996", "319331929", "376226553", "13335", "319379120", "329872996", "377310732", "434315799", "438494490", "416461", "164234053", "319399314", "319413544", "51063684", "126599440", "242083286", "313098677", "318153266", "318695273", "346441011", "369183227", "375580219", "441073933" ], "inchikey": "NRUKOCRGYNPUPR-PSZSYXFXSA-N", "generic_name": "Teniposide", "trade_name": "Vumon for injection", "designated_date": "1984-11-01", "designation_status": "Designated/Approved", "approved_labeled_indication": "Induction therapy in patients with refractory childhood acute lymphoblastic leukemia when used in combination with other approved anticancer agents.", "marketing_approval_date": "1992-07-14", "exclusivity_end_date": "1999-07-14", "sponsor": "Bristol-Myers Squibb Pharmaceutical Research Institute|5 Research Parkway|P.O. Box 5100|Wallingford|Connecticut|6492|United States", "orphan_designation": { "original_text": "Treatment of refractory childhood acute lymphocytic leukemia.", "umls": "C0023452", "parsed_text": "Childhood Acute Lymphocytic Leukemia" } }, { "pubchem_cid": 5396, "pubchem_sid": [ "597750", "7980753", "46507536", "49965048", "51063681", "53788607", "103589230", "134996895", "136342519", "162174797", "163620769", "163686094", "178103449", "184811965", "187072639", "223384958", "241050196", "251916159", "251917507", "252220258", "252390155", "252401148", "252451125", "254760638", "274086943", "310280530", "312694428", "315492991", "318162390", "319224575", "319554060", "322094616", "341835404", "347247495", "348955702", "349975478", "355040008", "363900008", "374383352", "375201719", "375303160", "375683044", "377363221", "381327005", "381379685", "381832363", "382159607", "384428756", "384568841", "384572884", "385783570", "386620411", "387060799", "401961199", "404760992", "433780032", "434123408", "434131442", "438475183", "440064782", "440790457", "440814161", "441008584", "26732588", "51063679", "99214913", "126670671", "163123657", "250127411", "252659762", "312654760", "319063501", "332872472", "387130411", "387209890", "405478151", "440024810", "8140078", "51063680", "51063683", "92719254", "126620951", "126653280", "164347276", "196111601", "253662241", "312228145", "318242301", "319086257", "319215055", "342476268", "348283120", "419590778", "438619773", "48416597", "51063682", "144204974", "315674173", "319297085", "341139721", "387130410", "51063685", "268734996", "319331929", "376226553", "13335", "319379120", "329872996", "377310732", "434315799", "438494490", "416461", "164234053", "319399314", "319413544", "51063684", "126599440", "242083286", "313098677", "318153266", "318695273", "346441011", "369183227", "375580219", "441073933" ], "inchikey": "NRUKOCRGYNPUPR-UHFFFAOYSA-N", "generic_name": "Teniposide", "trade_name": "Vumon for injection", "designated_date": "1984-11-01", "designation_status": "Designated/Approved", "approved_labeled_indication": "Induction therapy in patients with refractory childhood acute lymphoblastic leukemia when used in combination with other approved anticancer agents.", "marketing_approval_date": "1992-07-14", "exclusivity_end_date": "1999-07-14", "sponsor": "Bristol-Myers Squibb Pharmaceutical Research Institute|5 Research Parkway|P.O. Box 5100|Wallingford|Connecticut|6492|United States", "orphan_designation": { "original_text": "Treatment of refractory childhood acute lymphocytic leukemia.", "umls": "C0023452", "parsed_text": "Childhood Acute Lymphocytic Leukemia" } }, { "pubchem_cid": 452548, "pubchem_sid": [ "597750", "7980753", "46507536", "49965048", "51063681", "53788607", "103589230", "134996895", "136342519", "162174797", "163620769", "163686094", "178103449", "184811965", "187072639", "223384958", "241050196", "251916159", "251917507", "252220258", "252390155", "252401148", "252451125", "254760638", "274086943", "310280530", "312694428", "315492991", "318162390", "319224575", "319554060", "322094616", "341835404", "347247495", "348955702", "349975478", "355040008", "363900008", "374383352", "375201719", "375303160", "375683044", "377363221", "381327005", "381379685", "381832363", "382159607", "384428756", "384568841", "384572884", "385783570", "386620411", "387060799", "401961199", "404760992", "433780032", "434123408", "434131442", "438475183", "440064782", "440790457", "440814161", "441008584", "26732588", "51063679", "99214913", "126670671", "163123657", "250127411", "252659762", "312654760", "319063501", "332872472", "387130411", "387209890", "405478151", "440024810", "8140078", "51063680", "51063683", "92719254", "126620951", "126653280", "164347276", "196111601", "253662241", "312228145", "318242301", "319086257", "319215055", "342476268", "348283120", "419590778", "438619773", "48416597", "51063682", "144204974", "315674173", "319297085", "341139721", "387130410", "51063685", "268734996", "319331929", "376226553", "13335", "319379120", "329872996", "377310732", "434315799", "438494490", "416461", "164234053", "319399314", "319413544", "51063684", "126599440", "242083286", "313098677", "318153266", "318695273", "346441011", "369183227", "375580219", "441073933" ], "inchikey": "NRUKOCRGYNPUPR-QBPJDGROSA-N", "generic_name": "Teniposide", "trade_name": "Vumon for injection", "designated_date": "1984-11-01", "designation_status": "Designated/Approved", "approved_labeled_indication": "Induction therapy in patients with refractory childhood acute lymphoblastic leukemia when used in combination with other approved anticancer agents.", "marketing_approval_date": "1992-07-14", "exclusivity_end_date": "1999-07-14", "sponsor": "Bristol-Myers Squibb Pharmaceutical Research Institute|5 Research Parkway|P.O. Box 5100|Wallingford|Connecticut|6492|United States", "orphan_designation": { "original_text": "Treatment of refractory childhood acute lymphocytic leukemia.", "umls": "C0023452", "parsed_text": "Childhood Acute Lymphocytic Leukemia" } } ] }, { "_id": "UMLS:C0012236", "fda_orphan_drug": [ { "pubchem_cid": 16130571, "pubchem_sid": [ "50054734", "56459441", "85335659", "135234006", "160671058", "251971434", "313075632", "318162438", "319360135", "319557613", "340470686", "341108344", "349987930", "354732282", "355036819", "375686504", "376046473", "376229513", "384566835", "422600775", "354701772", "363743246", "375744190", "46504578", "318474065", "318478064", "341200324", "349253776" ], "inchikey": "NZVYCXVTEHPMHE-ZSUJOUNUSA-N", "generic_name": "Thymalfasin", "designated_date": "1998-01-08", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "SciClone Pharmaceuticals, Inc.|901 Mariner's Island Blvd.|San Mateo|California|94404|United States", "orphan_designation": { "original_text": "Treatment of DiGeorge anomaly with immune defects.", "umls": "C0012236", "parsed_text": "DiGeorge Anomaly" } }, { "pubchem_cid": 441350, "pubchem_sid": [ "10123", "46506079", "48416274", "103770845", "134977917", "135610156", "144204805", "160964108", "164230370", "170465112", "178103538", "224495450", "310277022", "312228378", "315673897", "318161653", "319222153", "348283399", "354278572", "355435716", "385511199", "387128500", "404847613", "406168158", "406850526", "434300821", "438462862", "438473251", "440790845", "178101787", "349984758", "355435708", "387195588", "387208314", "434300823", "438462863", "50065049" ], "inchikey": "NHTGHBARYWONDQ-JTQLQIEISA-N", "generic_name": "metyrosine", "designated_date": "2008-07-25", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Cerberus Princeton, LLC|c/o Cerberus Capital Management L.P.|875 Third Avenue|New York|New York|10022|United States", "orphan_designation": { "original_text": "Treatment of velocardiofacial syndrome associated psychosis.", "umls": [ "C0012236", "C0033975" ], "parsed_text": [ "Velocardiofacial Syndrome", "Psychosis" ] } } ] }, { "_id": "MONDO:0004993", "fda_orphan_drug": [ { "pubchem_cid": 32281, "pubchem_sid": [ "46505696", "50070190", "103771439", "135307205", "135651899", "135651953", "440790958", "440808714" ], "inchikey": "XNSAINXGIQZQOO-UHFFFAOYSA-N", "generic_name": "Thyrotropin alfa", "trade_name": "Thyrogen", "designated_date": "2001-08-03", "designation_status": "Designated/Approved", "approved_labeled_indication": "For use as an adjunctive treatment for radioiodine ablation of thyroid tissue remnants in patients who have undergone thyroidectomy for well-differentiated thyroid cancer and who do not have evidence of metastatic thyroid cancer", "marketing_approval_date": "2007-12-14", "exclusivity_end_date": "2014-12-14", "sponsor": "Genzyme Corporation|One Kendall Square|Cambridge|Massachusetts|2139|United States", "orphan_designation": { "original_text": "Treatment of well-differentiated papillary, follicular or combined papillary/follicular carcinomas of the thyroid", "umls": "C0007097", "parsed_text": "Carcinomas" } } ] }, { "_id": "UMLS:C0007758", "fda_orphan_drug": [ { "pubchem_cid": 7427, "pubchem_sid": [ "4320", "823151", "827622", "838550", "838702", "854522", "7890870", "10317885", "10317890", "11538469", "14719465", "24276928", "24276941", "24276942", "26736259", "26750680", "29204626", "57309725", "75748781", "81061296", "85164957", "99302925", "99302927", "99302960", "99302964", "99302966", "104252533", "111978637", "126596855", "131349515", "131481078", "134228443", "134971608", "136959136", "144241896", "160650245", "163831523", "164225518", "172659482", "172659484", "172659486", "196678970", "196678976", "204935552", "241176341", "254787951", "274087382", "275978253", "312225978", "313368946", "314975553", 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Box 50221|Austin|Texas|78763|United States", "orphan_designation": { "original_text": "Treatment of CLN Batten Disease", "umls": "C0751383", "parsed_text": "Batten Disease" } } ] }, { "_id": "UMLS:C4551720", "fda_orphan_drug": [ { "pubchem_cid": 6133, "pubchem_sid": [ "3375", "584151", "821167", "831552", "833275", "833716", "841750", "7891028", "8027183", "8027189", "8143371", "14720137", "17436071", "17436075", "24277146", "24277190", "26736753", "26737220", "46507277", "50061760", "56312062", "56312125", "56312759", "56312760", "85165099", "92765050", "92765052", "99455625", "103023975", "104234063", "104234205", "134971805", "135651592", "135724877", "136349857", "136380868", "136959286", "136959352", "144072367", "144072369", "144099830", "144221411", "144221415", "160654729", "162007880", "163565540", "163716015", "163826517", "163831462", "164161726", "164194272", "164194278", "165248689", "165248699", "172107229", "223483894", "224350837", "252082555", "252082560", "252255866", 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"retifanlimab", "designated_date": "2020-03-24", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Incyte Corporation|1801 Augustine Cut-Off|Wilmington|Delaware|19803|United States", "orphan_designation": { "original_text": "Treatment of anal cancer", "umls": "C0279637", "parsed_text": "Anal Cancer" } }, { "pubchem_sid": 404720524, "generic_name": "retifanlimab", "designated_date": "2020-03-24", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Incyte Corporation|1801 Augustine Cut-Off|Wilmington|Delaware|19803|United States", "orphan_designation": { "original_text": "Treatment of anal cancer", "umls": "C0279637", "parsed_text": "Anal Cancer" } }, { "pubchem_sid": 387061103, "generic_name": "nivolumab", "designated_date": "2017-08-07", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb 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O. Box 1000, UG2D-68|North Wales|Pennsylvania|19454|United States", "orphan_designation": { "original_text": "Prevention of human cytomegalovirus viremia and disease in at risk populations", "umls": [ "C0205725", "C0877635" ], "parsed_text": [ "Human cytomegalovirus", "Cytomegalovirus viremia" ] } } ] }, { "_id": "UMLS:C0854135", "fda_orphan_drug": [ { "pubchem_cid": 456591, "pubchem_sid": 603454, "inchikey": "LKCWBDHBTVXHDL-CAIQVSFASA-N", "generic_name": "liposomal amikacin", "designated_date": "2006-03-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Insmed, Inc.|Princeton Corporate Plaza|9 Deer Park Drive, Suite C|Monmouth Junction|New Jersey|8852|United States", "orphan_designation": { "original_text": "Treatment of bronchopulmonary Pseudomonas aeruginosa infections in cystic fibrosis patients", "umls": [ "C0010674", "C0854135" ], "parsed_text": [ "Cystic Fibrosis", "Pseudomonas aeruginosa Infections" ] } } ] }, { "_id": 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for Orphan Indication", "sponsor": "Ross Laboratories|625 Cleveland Avenue|Columbus|Ohio|43215|United States", "orphan_designation": { "original_text": "Treatment of full-term newborn infants with respiratory failure caused by meconium aspiration syndrome, persistent pulmonary hypertension of the newborn, or pneumonia and sepsis.", "umls": [ "C0025048", "C0031190", "C0032285", "C0243026", "C1145670" ], "parsed_text": [ "Meconium Aspiration Syndrome", "Persistent pulmonary hypertension of the newborn", "Pneumonia", "Sepsis", "Respiratory Failure" ] } }, { "pubchem_sid": 50070149, "generic_name": "Beractant", "designated_date": "1993-12-20", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Ross Laboratories|625 Cleveland Avenue|Columbus|Ohio|43215|United States", "orphan_designation": { "original_text": "Treatment of full-term newborn infants with respiratory failure caused by meconium aspiration syndrome, persistent pulmonary hypertension of the newborn, or pneumonia and sepsis.", "umls": [ "C0025048", "C0031190", "C0032285", "C0243026", "C1145670" ], "parsed_text": [ "Meconium Aspiration Syndrome", "Persistent pulmonary hypertension of the newborn", "Pneumonia", "Sepsis", "Respiratory Failure" ] } } ] }, { "_id": "UMLS:C3160897", "fda_orphan_drug": [ { "pubchem_cid": 5361918, "pubchem_sid": [ "50524128", "85335750", "123099065", "127331555", "127331556", "134224987", "135065087", "223365899", "318163726", "319299948", "348032505", "384437614", "385633548", "386619016", "387111525", "419501054", "439394514", "175427092", "249820229", "50065281", "50524129", "50524130", "164229822", "274136251", "387111526" ], "inchikey": "JVLBPIPGETUEET-GAAHOAFPSA-O", "generic_name": "Methylnaltrexone", "designated_date": "1996-06-17", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Progenics Pharmaceuticals, Inc.|765 Old Saw Mill River Rd.|Tarrytown|New 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"384570747", "387142912", "404721339", "419590681", "131458", "80132186", "80132187", "80132188", "80132190", "80132191", "80132192", "80132195", "80132196", "80132197", "80132198", "80132199", "80132200", "80132201", "80132202", "80132203", "80132204", "80132205", "80132206", "80132207", "80132208", "103962753", "144075687", "160964218", "162224759", "164233282", "164235807", "252160702", "375582801", "381118451", "438635167" ], "inchikey": "QFJCIRLUMZQUOT-HPLJOQBZSA-N", "generic_name": "rapamycin", "designated_date": "2017-12-21", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Aadi Bioscience, Inc.|1343 Luna Vista Drive|Los Angeles|California|90272|United States", "orphan_designation": { "original_text": "Treatment of perivascular epithelioid cell tumors (PEComa)", "umls": "C1300127", "parsed_text": "Perivascular Epithelioid Cell Tumors" } } ] }, { "_id": "UMLS:C0751436", "fda_orphan_drug": [ { "pubchem_cid": 135398579, "pubchem_sid": [ "2389", "4093", "50107031", "50107032", "56462995", "81065534", "92303171", "117589167", "123095528", "135023014", "162093831", "163565077", "250135347", "268738551", "274226010", "310279958", "312225678", "312345246", "319105937", "341154420", "348274342", "348831434", "355176293", "363669258", "376220337", "384494960", "385649460", "386256205", "386485896", "387165926", "425591816", "433774724", "433775148", "8145014", "50272053" ], "inchikey": "VPVOXUSPXFPWBN-VKHMYHEASA-N", "generic_name": "sepiapterin", "designated_date": "2020-02-19", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "PTC Therapeutics, Inc. (PTC)|65 William Street, Suite 200|Wellesley|Massachusetts|2481|United States", "orphan_designation": { "original_text": "Treatment of primary tetrahydrobiopterin deficiency", "umls": "C0751436", "parsed_text": "Tetrahydrobiopterin Deficiency" } }, { "pubchem_cid": 439280, "pubchem_sid": [ "8144010", "17388954", "46505826", "48423586", "48425235", "49854345", "85165245", "134985540", "135693923", "143491523", "144204624", "144208230", "152237788", "160966117", "162174832", "164195638", "170466308", "241085969", "249715138", "250133642", "250199697", "252372017", "252400300", "252453710", "253664312", "254776936", "312995306", "315675053", "316396466", "316903154", "318163392", "341151082", "341833807", "348274540", "348352157", "354266600", "355256470", "374016458", "375136640", "381148461", "384992071", "386393029", "387117837", "404639091", "434309841", "438488263", "85086878", "176255641", "250026973", "322070084", "340547403", "2047", "211534764", "342402241" ], "inchikey": "LDCYZAJDBXYCGN-VIFPVBQESA-N", "generic_name": "L-5-hydroxytryptophan", "designated_date": "1999-01-20", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Watson Laboratories, Inc.|311 Bonnie Circle|P.O. Box 1900|Corona|California|91718|United States", "orphan_designation": { "original_text": "Treatment of tetrahydrobiopterin deficiency.", "umls": "C0751436", "parsed_text": "Tetrahydrobiopterin Deficiency" } } ] }, { "_id": "UMLS:C0018674", "fda_orphan_drug": [ { "pubchem_cid": 517326, "pubchem_sid": [ "49861637", "56312195", "56312515", "78190501", "78190502", "99218248", "103270713", "125309078", "125309178", "126644322", "126692019", "134983791", "144207122", "144211711", "160791995", "162241433", "162278510", "162760662", "163808839", "176263557", "204360026", "251866151", "252156763", "252308341", "274954425", "315677688", "316404242", "332869019", "340124157", "341172422", "346549223", "354330009", "355045761", "355202580", "363905647", "374059876", "381385079", "382158424", "384257116", "385738493", "386575122", "387121130", "404571935", "404575260", "404619012", "434311840", "438490420", "438656970", "440974818", "441124748", "322095394", "341835343", "348903889", "53788498", "318160826", "91147426", "441082593" ], "inchikey": "LUPNKHXLFSSUGS-UHFFFAOYSA-M", "generic_name": "Sodium dichloroacetate", "designated_date": "1999-06-14", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Questcor Pharmaceuticals, Inc.|3260 Whipple Rd.|Union City|California|94587|United States", "orphan_designation": { "original_text": "Treatment of severe head injury.", "umls": "C0018674", "parsed_text": "HEAD INJURY" } }, { "pubchem_cid": 60767, "pubchem_sid": [ "49884801", "50726527", "135018707", "160708876", "319102513", "341389333", "355163947", "385729592", "386566211", "404618226", "434250850", "438455177", "349983209" ], "inchikey": "ZVVAINSYJGRDTR-TYLGTTGKSA-N", "generic_name": "Enadoline hydrochloride", "designated_date": "1997-01-28", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Warner-Lambert Company|Parke-Davis Pharmaceutical Research Division|2800 Plymouth Road|Ann Arbor|Michigan|48105|United States", "orphan_designation": { "original_text": "Treatment of severe head injury.", "umls": "C0018674", "parsed_text": "HEAD INJURY" } } ] }, { "_id": "MONDO:0006040", "fda_orphan_drug": [ { "pubchem_cid": 517326, "pubchem_sid": [ "49861637", "56312195", "56312515", "78190501", "78190502", "99218248", "103270713", "125309078", "125309178", "126644322", "126692019", "134983791", "144207122", "144211711", "160791995", "162241433", "162278510", "162760662", "163808839", "176263557", "204360026", "251866151", "252156763", "252308341", "274954425", "315677688", "316404242", "332869019", "340124157", "341172422", "346549223", "354330009", "355045761", "355202580", "363905647", "374059876", "381385079", "382158424", "384257116", "385738493", "386575122", "387121130", "404571935", "404575260", "404619012", "434311840", "438490420", "438656970", "440974818", "441124748", "322095394", "341835343", "348903889", "53788498", "318160826", "91147426", "441082593" ], "inchikey": "LUPNKHXLFSSUGS-UHFFFAOYSA-M", "generic_name": "Sodium dichloroacetate", "designated_date": "1994-11-10", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Stacpoole, Peter W. M.D., Ph.D.|University of Florida|P.O. Box 100277|Gainesville|Florida|32610|United States", "orphan_designation": { "original_text": "Treatment of lactic acidosis in patients with severe malaria.", "umls": [ "C0001125", "C2747816" ], "parsed_text": [ "LACTIC ACIDOSIS", "Severe Malaria" ] } }, { "pubchem_cid": 3686, "pubchem_sid": [ "12012810", "26719872", "46386682", "49681642", "49957336", "56313750", "78556875", "92710538", "103060386", "103556242", "126631778", "126658121", "126665893", "135003257", "135698082", "136990616", "143492810", "144075938", "144205417", "152242914", "160871272", "162038194", "162173185", "170465611", "172080219", "187072683", "196109651", "210024006", "210280241", "210282221", "223377118", "223441547", "223701090", "249866551", "250133395", "251960540", "252156937", "252218535", "252350841", "252448661", "253663492", "254761339", "255474572", "268736344", "274896695", "275956092", "277309071", "310275130", "312692157", "312695136", "313076962", "313081144", "315678276", "316962814", "318045949", "318154402", "318161318", "318166205", "318240733", "319062462", "319554474", "322079957", "336283453", "340570326", "341142188", "341833824", "346432224", "347913424", "349138141", "349990035", "350088986", "355038384", "363662844", "374035537", "375154621", "375297642", "375581763", "375670162", "376016590", "377009284", "377310507", "378134723", "381365179", "382162774", "383425983", "384568196", "384571997", "385787021", "386260897", "386624072", "401975479", "402288101", "404612844", "405492833", "406807229", "419553707", "433780482", "434132594", "434253456", "437412592", "438458683", "438471026", "440039491", "440062483", "440714083", "441005003", "206246355", "242077397", "438599173", "78556876", "250224518", "255370226", "310265009", "441075244" ], "inchikey": "JGPMMRGNQUBGND-UHFFFAOYSA-N", "generic_name": "idebenone", "designated_date": "2009-05-22", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Santhera Pharmaceuticals (Switzerland) Ltd.|25 Hammerstrasse|Liestal|Basel-Landschaft|Switzerland", "orphan_designation": { "original_text": "Treatment of mitochondrial myopathy, encephalopathy, lactic acidosis with stroke-like episodes syndrome (MELAS)", "umls": [ "C0001125", "C0039082", "C0085584", "C0162670", "C0162671", "C1857287" ], "parsed_text": [ "LACTIC ACIDOSIS", "Syndrome", "Encephalopathy", "MITOCHONDRIAL MYOPATHY", "MELAS", "Stroke-like episodes" ] } }, { "pubchem_cid": 439924, "pubchem_sid": [ "3006", "8145078", "26717794", "26717796", "53786905", "103538035", "135107223", "175429332", "210024391", "274180924", "312225926", "348274863", "348705031", "363910249", "381338237", "384647337", "386467212", "386474236", "404772378", "438797239", "439443081", "277425092", "312830467", "347753988", "355046315", "375590350", "377622181", "405449704", "439996368", "252212984", "441079100" ], "inchikey": "JLEBZPBDRKPWTD-TURQNECASA-O", "generic_name": "nicotinamide riboside", "designated_date": "2017-01-25", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Rejuvenation Therapeutics|774 Mays Boulevard|10-509 PMB|Incline Village|Nevada|89451|United States", "orphan_designation": { "original_text": "Treatment of mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes syndrome (MELAS)", "umls": [ "C0001125", "C0039082", "C0162671", "C1852373", "C1857287" ], "parsed_text": [ "LACTIC ACIDOSIS", "Syndrome", "MELAS", "Mitochondrial encephalopathy", "Stroke-like episodes" ] } } ] }, { "_id": "UMLS:C2747816", "fda_orphan_drug": [ { "pubchem_cid": 517326, "pubchem_sid": [ "49861637", "56312195", "56312515", "78190501", "78190502", "99218248", "103270713", "125309078", "125309178", "126644322", "126692019", "134983791", "144207122", "144211711", "160791995", "162241433", "162278510", "162760662", "163808839", "176263557", "204360026", "251866151", "252156763", "252308341", "274954425", "315677688", "316404242", "332869019", "340124157", "341172422", "346549223", "354330009", "355045761", "355202580", "363905647", "374059876", "381385079", "382158424", "384257116", "385738493", "386575122", "387121130", "404571935", "404575260", "404619012", "434311840", "438490420", "438656970", "440974818", "441124748", "322095394", "341835343", "348903889", "53788498", "318160826", "91147426", "441082593" ], "inchikey": "LUPNKHXLFSSUGS-UHFFFAOYSA-M", "generic_name": "Sodium dichloroacetate", "designated_date": "1994-11-10", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Stacpoole, Peter W. M.D., Ph.D.|University of Florida|P.O. Box 100277|Gainesville|Florida|32610|United States", "orphan_designation": { "original_text": "Treatment of lactic acidosis in patients with severe malaria.", "umls": [ "C0001125", "C2747816" ], "parsed_text": [ "LACTIC ACIDOSIS", "Severe Malaria" ] } } ] }, { "_id": "UMLS:C0017107", "fda_orphan_drug": [ { "pubchem_cid": 23668193, "pubchem_sid": [ "10501486", "11528239", "17388728", "17389803", "48414112", "48420075", "48424099", "48425624", "48425625", "53789712", "76196160", "103304140", "117375241", "117517318", "124391762", "125307130", "134987571", "144209353", "144210345", "160804277", "162094247", "162185694", "162654969", "163806552", "176258463", "204359940", "204940429", "223444117", "223737653", "252274886", "252414369", "274127748", "310280134", "315446203", "315671874", "316400516", "316439001", "316444147", "316962679", "316971729", "318467021", "319374991", "341140516", "347913935", "349998368", "349998369", "349998370", "349998371", "349998372", "355202722", "363896548", "363898509", "373420877", "374169369", "375088195", "377310151", "382164694", "383237389", "384257584", "385783149", "386263040", "386619951", "387120251", "387208915", "406176026", "438504990", "439427017", "439649462", "318154150", "349301497", "551009", "76196161", "349989978", "441065349" ], "inchikey": "LPXPTNMVRIOKMN-UHFFFAOYSA-M", "generic_name": "sodium nitrite", "designated_date": "2012-01-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hope Pharmaceuticals|16416 North 92nd St.|Suite 125|Scottsdale|Alaska|85260|United States", "orphan_designation": { "original_text": "Treatment of chlorine gas poisoning", "umls": "C0017107", "parsed_text": "Gas Poisoning" } } ] }, { "_id": "UMLS:C0043207", "fda_orphan_drug": [ { "pubchem_cid": 16760703, "pubchem_sid": [ "46505925", "49856166", "53788878", "93167016", "134971905", "310281647", "319062079", "348275431", "349979140", "349995411", "363603139", "404754746", "56459047", "124403703", "224090182", "336267894", "341826296", "385661363", "386497869", "419582696", "439383065", "117375016", "136946697", "348274902" ], "inchikey": "AEQFSUDEHCCHBT-UHFFFAOYSA-M", "generic_name": "sodium valproate", "designated_date": "2015-08-05", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "The University of Birmingham|Birmingham Research Park - Vincent Drive|Birmingham|United Kingdom", "orphan_designation": { "original_text": "Treatment of Wolfram syndrome", "umls": "C0043207", "parsed_text": "Wolfram Syndrome" } }, { "pubchem_cid": 136675526, "pubchem_sid": [ "8149931", "49977301", "50064615", "50086896", "50106202", "50224504", "96079580", "103241627", "104170142", "135022024", "135697908", "252359371", "252402840", "254759916", "313080600", "319331846", "319331847", "355037251", "376219638", "376228113", "381382564", "381394157", "383840781", "404622018", "404839553", "434131969", "440064426", "441056653", "50224503", "92303995", "126628766", "312344549", "316899266", "316966591", "318031413", "319353482", "350086829", "350095976", "374043658", "375134207", "49977300", "53789979", "144205566", "144212314", "160964552", "170464793", "315680529", "318160748", "319362120", "319362121", "322079894", "341838939", "349988026", "385663493", "386500005", "387115919", "404639670", "441114322", "85383553", "126659538", "134996352", "144075555", "315495698", "319334576", "319378575", "319378576", "438473989", "50104441", "387065024", "163564378", "255369980", "268735387", "268738067", "274885735", "319407141", "342560733", "348777476", "368494613", "375575894", "441078404" ], "inchikey": "KSRLIXGNPXAZHD-DFPPEFKWSA-N", "generic_name": "dantrolene sodium", "designated_date": "2016-02-17", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Washington University in St. Louis|660 S. Euclid Avenue|Campus Box 8127|St. Louis|Missouri|63110|United States", "orphan_designation": { "original_text": "Treatment of Wolfram Syndrome", "umls": "C0043207", "parsed_text": "Wolfram Syndrome" } }, { "pubchem_cid": 136675526, "pubchem_sid": [ "8149931", "49977301", "50064615", "50086896", "50106202", "50224504", "96079580", "103241627", "104170142", "135022024", "135697908", "252359371", "252402840", "254759916", "313080600", "319331846", "319331847", "355037251", "376219638", "376228113", "381382564", "381394157", "383840781", "404622018", "404839553", "434131969", "440064426", "441056653", "50224503", "92303995", "126628766", "312344549", "316899266", "316966591", "318031413", "319353482", "350086829", "350095976", "374043658", "375134207", "49977300", "53789979", "144205566", "144212314", "160964552", "170464793", "315680529", "318160748", "319362120", "319362121", "322079894", "341838939", "349988026", "385663493", "386500005", "387115919", "404639670", "441114322", "85383553", "126659538", "134996352", "144075555", "315495698", "319334576", "319378575", "319378576", "438473989", "50104441", "387065024", "163564378", "255369980", "268735387", "268738067", "274885735", "319407141", "342560733", "348777476", "368494613", "375575894", "441078404" ], "inchikey": "KSRLIXGNPXAZHD-DFPPEFKWSA-N", "generic_name": "dantrolene sodium", "designated_date": "2016-02-17", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Washington University in St. Louis|660 S. Euclid Avenue|Campus Box 8127|St. Louis|Missouri|63110|United States", "orphan_designation": { "original_text": "Treatment of Wolfram Syndrome", "umls": "C0043207", "parsed_text": "Wolfram Syndrome" } }, { "pubchem_cid": 71563341, "pubchem_sid": [ "8149931", "49977301", "50064615", "50086896", "50106202", "50224504", "96079580", "103241627", "104170142", "135022024", "135697908", "252359371", "252402840", "254759916", "313080600", "319331846", "319331847", "355037251", "376219638", "376228113", "381382564", "381394157", "383840781", "404622018", "404839553", "434131969", "440064426", "441056653", "50224503", "92303995", "126628766", "312344549", "316899266", "316966591", "318031413", "319353482", "350086829", "350095976", "374043658", "375134207", "49977300", "53789979", "144205566", "144212314", "160964552", "170464793", "315680529", "318160748", "319362120", "319362121", "322079894", "341838939", "349988026", "385663493", "386500005", "387115919", "404639670", "441114322", "85383553", "126659538", "134996352", "144075555", "315495698", "319334576", "319378575", "319378576", "438473989", "50104441", "387065024", "163564378", "255369980", "268735387", "268738067", "274885735", "319407141", "342560733", "348777476", "368494613", "375575894", "441078404" ], "inchikey": "KSRLIXGNPXAZHD-DFPPEFKWSA-M", "generic_name": "dantrolene sodium", "designated_date": "2016-02-17", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Washington University in St. Louis|660 S. Euclid Avenue|Campus Box 8127|St. Louis|Missouri|63110|United States", "orphan_designation": { "original_text": "Treatment of Wolfram Syndrome", "umls": "C0043207", "parsed_text": "Wolfram Syndrome" } }, { "pubchem_cid": 5353563, "pubchem_sid": [ "8149931", "49977301", "50064615", "50086896", "50106202", "50224504", "96079580", "103241627", "104170142", "135022024", "135697908", "252359371", "252402840", "254759916", "313080600", "319331846", "319331847", "355037251", "376219638", "376228113", "381382564", "381394157", "383840781", "404622018", "404839553", "434131969", "440064426", "441056653", "50224503", "92303995", "126628766", "312344549", "316899266", "316966591", "318031413", "319353482", "350086829", "350095976", "374043658", "375134207", "49977300", "53789979", "144205566", "144212314", "160964552", "170464793", "315680529", "318160748", "319362120", "319362121", "322079894", "341838939", "349988026", "385663493", "386500005", "387115919", "404639670", "441114322", "85383553", "126659538", "134996352", "144075555", "315495698", "319334576", "319378575", "319378576", "438473989", "50104441", "387065024", "163564378", "255369980", "268735387", "268738067", "274885735", "319407141", "342560733", "348777476", "368494613", "375575894", "441078404" ], "inchikey": "OZOMQRBLCMDCEG-CHHVJCJISA-M", "generic_name": "dantrolene sodium", "designated_date": "2016-02-17", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Washington University in St. Louis|660 S. Euclid Avenue|Campus Box 8127|St. Louis|Missouri|63110|United States", "orphan_designation": { "original_text": "Treatment of Wolfram Syndrome", "umls": "C0043207", "parsed_text": "Wolfram Syndrome" } }, { "pubchem_cid": 6604728, "pubchem_sid": [ "8149931", "49977301", "50064615", "50086896", "50106202", "50224504", "96079580", "103241627", "104170142", "135022024", "135697908", "252359371", "252402840", "254759916", "313080600", "319331846", "319331847", "355037251", "376219638", "376228113", "381382564", "381394157", "383840781", "404622018", "404839553", "434131969", "440064426", "441056653", "50224503", "92303995", "126628766", "312344549", "316899266", "316966591", "318031413", "319353482", "350086829", "350095976", "374043658", "375134207", "49977300", "53789979", "144205566", "144212314", "160964552", "170464793", "315680529", "318160748", "319362120", "319362121", "322079894", "341838939", "349988026", "385663493", "386500005", "387115919", "404639670", "441114322", "85383553", "126659538", "134996352", "144075555", "315495698", "319334576", "319378575", "319378576", "438473989", "50104441", "387065024", "163564378", "255369980", "268735387", "268738067", "274885735", "319407141", "342560733", "348777476", "368494613", "375575894", "441078404" ], "inchikey": "OZOMQRBLCMDCEG-VIZOYTHASA-M", "generic_name": "dantrolene sodium", "designated_date": "2016-02-17", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Washington University in St. Louis|660 S. Euclid Avenue|Campus Box 8127|St. Louis|Missouri|63110|United States", "orphan_designation": { "original_text": "Treatment of Wolfram Syndrome", "umls": "C0043207", "parsed_text": "Wolfram Syndrome" } }, { "pubchem_cid": 23707296, "pubchem_sid": [ "8149931", "49977301", "50064615", "50086896", "50106202", "50224504", "96079580", "103241627", "104170142", "135022024", "135697908", "252359371", "252402840", "254759916", "313080600", "319331846", "319331847", "355037251", "376219638", "376228113", "381382564", "381394157", "383840781", "404622018", "404839553", "434131969", "440064426", "441056653", "50224503", "92303995", "126628766", "312344549", "316899266", "316966591", "318031413", "319353482", "350086829", "350095976", "374043658", "375134207", "49977300", "53789979", "144205566", "144212314", "160964552", "170464793", "315680529", "318160748", "319362120", "319362121", "322079894", "341838939", "349988026", "385663493", "386500005", "387115919", "404639670", "441114322", "85383553", "126659538", "134996352", "144075555", "315495698", "319334576", "319378575", "319378576", "438473989", "50104441", "387065024", "163564378", "255369980", "268735387", "268738067", "274885735", "319407141", "342560733", "348777476", "368494613", "375575894", "441078404" ], "inchikey": "KSRLIXGNPXAZHD-HAZZGOGXSA-N", "generic_name": "dantrolene sodium", "designated_date": "2016-02-17", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Washington University in St. Louis|660 S. Euclid Avenue|Campus Box 8127|St. Louis|Missouri|63110|United States", "orphan_designation": { "original_text": "Treatment of Wolfram Syndrome", "umls": "C0043207", "parsed_text": "Wolfram Syndrome" } }, { "pubchem_cid": 16219293, "pubchem_sid": [ "8149931", "49977301", "50064615", "50086896", "50106202", "50224504", "96079580", "103241627", "104170142", "135022024", "135697908", "252359371", "252402840", "254759916", "313080600", "319331846", "319331847", "355037251", "376219638", "376228113", "381382564", "381394157", "383840781", "404622018", "404839553", "434131969", "440064426", "441056653", "50224503", "92303995", "126628766", "312344549", "316899266", "316966591", "318031413", "319353482", "350086829", "350095976", "374043658", "375134207", "49977300", "53789979", "144205566", "144212314", "160964552", "170464793", "315680529", "318160748", "319362120", "319362121", "322079894", "341838939", "349988026", "385663493", "386500005", "387115919", "404639670", "441114322", "85383553", "126659538", "134996352", "144075555", "315495698", "319334576", "319378575", "319378576", "438473989", "50104441", "387065024", "163564378", "255369980", "268735387", "268738067", "274885735", "319407141", "342560733", "348777476", "368494613", "375575894", "441078404" ], "inchikey": "BDZCLIOURDFSFQ-HAZZGOGXSA-N", "generic_name": "dantrolene sodium", "designated_date": "2016-02-17", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Washington University in St. Louis|660 S. Euclid Avenue|Campus Box 8127|St. Louis|Missouri|63110|United States", "orphan_designation": { "original_text": "Treatment of Wolfram Syndrome", "umls": "C0043207", "parsed_text": "Wolfram Syndrome" } }, { "pubchem_cid": 9568637, "pubchem_sid": [ "8149931", "49977301", "50064615", "50086896", "50106202", "50224504", "96079580", "103241627", "104170142", "135022024", "135697908", "252359371", "252402840", "254759916", "313080600", "319331846", "319331847", "355037251", "376219638", "376228113", "381382564", "381394157", "383840781", "404622018", "404839553", "434131969", "440064426", "441056653", "50224503", "92303995", "126628766", "312344549", "316899266", "316966591", "318031413", "319353482", "350086829", "350095976", "374043658", "375134207", "49977300", "53789979", "144205566", "144212314", "160964552", "170464793", "315680529", "318160748", "319362120", "319362121", "322079894", "341838939", "349988026", "385663493", "386500005", "387115919", "404639670", "441114322", "85383553", "126659538", "134996352", "144075555", "315495698", "319334576", "319378575", "319378576", "438473989", "50104441", "387065024", "163564378", "255369980", "268735387", "268738067", "274885735", "319407141", "342560733", "348777476", "368494613", "375575894", "441078404" ], "inchikey": "LTWQNYPDAUSXBC-CDJGKPBYSA-L", "generic_name": "dantrolene sodium", "designated_date": "2016-02-17", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Washington University in St. Louis|660 S. Euclid Avenue|Campus Box 8127|St. Louis|Missouri|63110|United States", "orphan_designation": { "original_text": "Treatment of Wolfram Syndrome", "umls": "C0043207", "parsed_text": "Wolfram Syndrome" } }, { "pubchem_cid": 32731, "pubchem_sid": [ "8149931", "49977301", "50064615", "50086896", "50106202", "50224504", "96079580", "103241627", "104170142", "135022024", "135697908", "252359371", "252402840", "254759916", "313080600", "319331846", "319331847", "355037251", "376219638", "376228113", "381382564", "381394157", "383840781", "404622018", "404839553", "434131969", "440064426", "441056653", "50224503", "92303995", "126628766", "312344549", "316899266", "316966591", "318031413", "319353482", "350086829", "350095976", "374043658", "375134207", "49977300", "53789979", "144205566", "144212314", "160964552", "170464793", "315680529", "318160748", "319362120", "319362121", "322079894", "341838939", "349988026", "385663493", "386500005", "387115919", "404639670", "441114322", "85383553", "126659538", "134996352", "144075555", "315495698", "319334576", "319378575", "319378576", "438473989", "50104441", "387065024", "163564378", "255369980", "268735387", "268738067", "274885735", "319407141", "342560733", "348777476", "368494613", "375575894", "441078404" ], "inchikey": "KSRLIXGNPXAZHD-UHFFFAOYSA-M", "generic_name": "dantrolene sodium", "designated_date": "2016-02-17", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Washington University in St. Louis|660 S. Euclid Avenue|Campus Box 8127|St. Louis|Missouri|63110|United States", "orphan_designation": { "original_text": "Treatment of Wolfram Syndrome", "umls": "C0043207", "parsed_text": "Wolfram Syndrome" } }, { "pubchem_cid": 13271066, "pubchem_sid": [ "8149931", "49977301", "50064615", "50086896", "50106202", "50224504", "96079580", "103241627", "104170142", "135022024", "135697908", "252359371", "252402840", "254759916", "313080600", "319331846", "319331847", "355037251", "376219638", "376228113", "381382564", "381394157", "383840781", "404622018", "404839553", "434131969", "440064426", "441056653", "50224503", "92303995", "126628766", "312344549", "316899266", "316966591", "318031413", "319353482", "350086829", "350095976", "374043658", "375134207", "49977300", "53789979", "144205566", "144212314", "160964552", "170464793", "315680529", "318160748", "319362120", "319362121", "322079894", "341838939", "349988026", "385663493", "386500005", "387115919", "404639670", "441114322", "85383553", "126659538", "134996352", "144075555", "315495698", "319334576", "319378575", "319378576", "438473989", "50104441", "387065024", "163564378", "255369980", "268735387", "268738067", "274885735", "319407141", "342560733", "348777476", "368494613", "375575894", "441078404" ], "inchikey": "KSRLIXGNPXAZHD-HAZZGOGXSA-M", "generic_name": "dantrolene sodium", "designated_date": "2016-02-17", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Washington University in St. Louis|660 S. Euclid Avenue|Campus Box 8127|St. Louis|Missouri|63110|United States", "orphan_designation": { "original_text": "Treatment of Wolfram Syndrome", "umls": "C0043207", "parsed_text": "Wolfram Syndrome" } }, { "pubchem_cid": 71563341, "pubchem_sid": [ "8149931", "49977301", "50064615", "50086896", "50106202", "50224504", "96079580", "103241627", "104170142", "135022024", "135697908", "252359371", "252402840", "254759916", "313080600", "319331846", "319331847", "355037251", "376219638", "376228113", "381382564", "381394157", "383840781", "404622018", "404839553", "434131969", "440064426", "441056653", "50224503", "92303995", "126628766", "312344549", "316899266", "316966591", "318031413", "319353482", "350086829", "350095976", "374043658", "375134207", "49977300", "53789979", "144205566", "144212314", "160964552", "170464793", "315680529", "318160748", "319362120", "319362121", "322079894", "341838939", "349988026", "385663493", "386500005", "387115919", "404639670", "441114322", "85383553", "126659538", "134996352", "144075555", "315495698", "319334576", "319378575", "319378576", "438473989", "50104441", "387065024", "163564378", "255369980", "268735387", "268738067", "274885735", "319407141", "342560733", "348777476", "368494613", "375575894", "441078404" ], "inchikey": "KSRLIXGNPXAZHD-DFPPEFKWSA-M", "generic_name": "dantrolene sodium", "designated_date": "2016-02-17", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Washington University in St. Louis|660 S. Euclid Avenue|Campus Box 8127|St. Louis|Missouri|63110|United States", "orphan_designation": { "original_text": "Treatment of Wolfram Syndrome", "umls": "C0043207", "parsed_text": "Wolfram Syndrome" } }, { "pubchem_cid": 13271066, "pubchem_sid": [ "8149931", "49977301", "50064615", "50086896", "50106202", "50224504", "96079580", "103241627", "104170142", "135022024", "135697908", "252359371", "252402840", "254759916", "313080600", "319331846", "319331847", "355037251", "376219638", "376228113", "381382564", "381394157", "383840781", "404622018", "404839553", "434131969", "440064426", "441056653", "50224503", "92303995", "126628766", "312344549", "316899266", "316966591", "318031413", "319353482", "350086829", "350095976", "374043658", "375134207", "49977300", "53789979", "144205566", "144212314", "160964552", "170464793", "315680529", "318160748", "319362120", "319362121", "322079894", "341838939", "349988026", "385663493", "386500005", "387115919", "404639670", "441114322", "85383553", "126659538", "134996352", "144075555", "315495698", "319334576", "319378575", "319378576", "438473989", "50104441", "387065024", "163564378", "255369980", "268735387", "268738067", "274885735", "319407141", "342560733", "348777476", "368494613", "375575894", "441078404" ], "inchikey": "KSRLIXGNPXAZHD-HAZZGOGXSA-M", "generic_name": "dantrolene sodium", "designated_date": "2016-02-17", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Washington University in St. Louis|660 S. Euclid Avenue|Campus Box 8127|St. Louis|Missouri|63110|United States", "orphan_designation": { "original_text": "Treatment of Wolfram Syndrome", "umls": "C0043207", "parsed_text": "Wolfram Syndrome" } } ] }, { "_id": "MONDO:0021681", "fda_orphan_drug": [ { "pubchem_cid": 213054, "pubchem_sid": [ "53013250", "135188011", "160681924", "341678923", "347242797", "347831669", "347832078", "349991547", "355036220", "385738106", "386574734", "439364272", "405476274", "440022933", "53013247", "53013248", "53013249", "275292501" ], "inchikey": "PWILYDZRJORZDR-MISYRCLQSA-N", "generic_name": "soraprazan", "designated_date": "2017-12-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Katairo GmbH|21 Lederstra\u07e5|Kusterdingen|Baden-Wrttemberg|Germany", "orphan_designation": { "original_text": "Treatment of Stargardt Disease (StD)", "umls": [ "C0036916", "C1412063" ], "parsed_text": [ "STD", "Stargardt disease" ] } } ] }, { "_id": "MONDO:0015614", "fda_orphan_drug": [ { "pubchem_cid": 5336, "pubchem_sid": [ "70988", "96525", "603199", "855656", "7980704", "8149538", "10321924", "10508192", "46506991", "48424128", "49855022", "51026952", "85089655", "85332653", "87575694", "99222767", "103194001", "103914454", "117528267", "124766378", "126632141", "126688114", "134974429", "144075230", "144203977", "160964231", "162177256", "170464707", "175442336", "176326584", "187072086", "187975081", "206252475", "210279353", "210281676", "223792827", "250063305", "252402081", "252620235", "268734713", "274190779", "277309201", "310280397", "312228478", "312693901", "313057908", "313082265", "313170622", "315446283", "315675358", "316410537", "316897851", "318154156", "318162331", "319063458", "319554856", "329980661", "340510677", "340516467", "340521111", "341140808", "346434787", "348283509", "348397539", "348770937", "349985635", "355038836", "363592688", "374087650", "374195286", "375296420", "375651944", "376126110", "376226511", "381340863", "382155934", "384571636", "385782072", "386618808", "387093324", "387196696", "387209852", "404617372", "404755157", "404839492", "405109048", "405455039", "406545210", "419494150", "433778981", "433779797", "433781188", "433788307", "433788308", "434131799", "434310911", "438469776", "438472763", "438489514", "440001701", "440064367", "440770305", "441070922" ], "inchikey": "GECHUMIMRBOMGK-UHFFFAOYSA-N", "generic_name": "Sulfapyridine", "designated_date": "1990-09-10", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Jacobus Pharmaceutical Company|37 Cleveland Lane|P.O. 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"C0343467", "parsed_text": "Erythema nodosum leprosum" } }, { "pubchem_cid": 2794, "pubchem_sid": [ "9132", "426821", "602744", "855829", "7978968", "10321645", "24714740", "24893114", "46386952", "46508174", "48415799", "49681815", "50004010", "50376246", "56314325", "93167165", "96079551", "103400985", "103914133", "104153270", "124766188", "126671524", "134223284", "134982031", "144075993", "144204067", "160857866", "172080532", "241038834", "249738327", "250130908", "252559282", "253661510", "254804139", "268735210", "275134025", "310270403", "312234670", "312323814", "312694194", "313077393", "315444469", "315661264", "315673496", "318154027", "318160670", "319061878", "319453250", "319556818", "329877105", "332868071", "335959191", "340516670", "340521318", "341140366", "341843591", "346441069", "347757410", "347914019", "348274027", "348283469", "348886799", "349992654", "350087295", "350096016", "354283943", "354326287", "363671221", "363740823", "364120288", "373774285", 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Ridgewood Avenue|Suite 415, South Tower|Paramus|New Jersey|7652|United States", "orphan_designation": { "original_text": "treatment for Niemann-Pick disease Type A and Type B", "umls": "C0268242", "parsed_text": "niemann-pick disease type a" } }, { "pubchem_sid": 381125841, "generic_name": "olipudase alfa", "designated_date": "2000-08-03", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genzyme Corporation|55 Corporate Drive|Bridgewater|New Jersey|8807|United States", "orphan_designation": { "original_text": "Treatment of acid sphingomyelinase deficiency (Niemann-Pick disease)", "umls": [ "C0028064", "C0268242" ], "parsed_text": [ "Sphingomyelinase deficiency", "Sphingomyelinase Deficiency" ] } }, { "pubchem_sid": 347911391, "generic_name": "olipudase alfa", "designated_date": "2000-08-03", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genzyme Corporation|55 Corporate Drive|Bridgewater|New Jersey|8807|United States", "orphan_designation": { "original_text": "Treatment of acid sphingomyelinase deficiency (Niemann-Pick disease)", "umls": [ "C0028064", "C0268242" ], "parsed_text": [ "Sphingomyelinase deficiency", "Sphingomyelinase Deficiency" ] } }, { "pubchem_sid": 318464595, "generic_name": "olipudase alfa", "designated_date": "2000-08-03", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genzyme Corporation|55 Corporate Drive|Bridgewater|New Jersey|8807|United States", "orphan_designation": { "original_text": "Treatment of acid sphingomyelinase deficiency (Niemann-Pick disease)", "umls": [ "C0028064", "C0268242" ], "parsed_text": [ "Sphingomyelinase deficiency", "Sphingomyelinase Deficiency" ] } } ] }, { "_id": "UMLS:C0677050", "fda_orphan_drug": [ { "pubchem_cid": 71433, "pubchem_sid": [ "12012854", "49966386", "76222015", "135030668", "315686180", "319111561", "332875451", 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"orphan_designation": { "original_text": "Treatment of iron overload in patients with hematologic disorders requiring chronic transfusion therapy", "umls": [ "C0018939", "C0282193" ], "parsed_text": [ "Hematologic Disorders", "Iron Overload" ] } } ] }, { "_id": "UMLS:C4551472", "fda_orphan_drug": [ { "pubchem_cid": 702, "pubchem_sid": [ "841053", "134971966", "318692494", "348274252", "349974374" ], "inchikey": "LFQSCWFLJHTTHZ-UHFFFAOYSA-N", "generic_name": "dehydrated alcohol", "trade_name": "Ablysinol", "designated_date": "2013-09-11", "designation_status": "Designated/Approved", "approved_labeled_indication": "ABLYSINOL is indicated to induce controlled cardiac septal infarction to improve exercise capacity in adults with symptomatic hypertrophic obstructive cardiomyopathy who are not candidates for surgical myectomy.", "marketing_approval_date": "2018-06-21", "exclusivity_end_date": "2025-06-21", "exclusivity_protected_indication": "ABLYSINOL is indicated to induce controlled 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O. Box 13398|Five Moore Drive|Research Triangle Park|North Carolina|27709|United States", "orphan_designation": { "original_text": "Treatment of systemic lupus erythematosus (SLE) and the reduction in the use of steroids in steroid-dependent SLE patients.", "umls": [ "C0014060", "C0024141" ], "parsed_text": [ "SLE", "SYSTEMIC LUPUS ERYTHEMATOSUS" ] } } ] }, { "_id": "UMLS:C0037578", "fda_orphan_drug": [ { "pubchem_cid": 679, "pubchem_sid": [ "3727071", "14709412", "49855700", "53788579", "127275119", "127275120", "127275121", "127275122", "127275123", "127275124", "127275125", "127275126", "127275127", "127275128", "127275129", "127275130", "127275131", "127275132", "127275133", "127275134", "127275135", "127275136", "127275137", "127275138", "127275139", "127275140", "127275141", "127275142", "127275143", "127275144", "127275145", "127275146", "127275147", "127275148", "127275149", "127275150", "127275151", "127275152", "127275153", "127275154", "127275155", "127275156", "127275157", 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Indication", "sponsor": "Cancer Technologies, Inc.|7301 East 22nd Street|Suite 10E|Tucson|Alaska|85710|United States", "orphan_designation": { "original_text": "Topical treatment for the prevention of soft tissue injury following extravastion of cytotoxic drugs.", "umls": "C0037578", "parsed_text": "SOFT TISSUE INJURY" } } ] }, { "_id": "UMLS:C3887709", "fda_orphan_drug": [ { "pubchem_cid": 11764719, "pubchem_sid": [ "85756524", "249732236", "318474068", "318478067", "319347251", "319557760", "336908571", "341714538", "347828303", "347911494", "385744720", "386581358", "404616408", "434250976", "438455073", "439591756", "354701291", "375744665", "355313147" ], "inchikey": "SFVLTCAESLKEHH-WKAQUBQDSA-N", "generic_name": "elamipretide", "designated_date": "2018-04-02", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Stealth BioTherapeutics Inc.|275 Grove Street 3-107|Newton|Massachusetts|2466|United States", "orphan_designation": { "original_text": "Treatment of Lebers Hereditary Optic Neuropathy", "umls": "C3887709", "parsed_text": "Optic Neuropathy" } } ] }, { "_id": "MONDO:0005385", "fda_orphan_drug": [ { "pubchem_cid": 21158562, "pubchem_sid": [ "46507362", "48415947", "53790208", "77700624", "93166672", "103326666", "135000098", "135651541", "224259470", "251916089", "251917437", "315673618", "318160982", "319296445", "349993883", "385782552", "386393050", "386619308", "434217383", "440791117", "4527", "77700623", "160964572", "312248208", "319295364", "348275210", "419492675", "439574700", "241064319", "252156930", "252359340", "375134176", "382163213", "50026794", "77700620", "77700622", "77700628", "318692103", "319143304", "341850141", "349173894", "405481564", "434251148", "438455388", "440028223", "77700621", "77700625", "77700626", "77700627", "103825406", "126652687", "136949627", "318156204", "322079921", "375566619", "387112546", "438555563", "441072744" ], "inchikey": 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O. Box 61540|King of Prussia|Pennsylvania|19406|United States", "orphan_designation": { "original_text": "Treatment of secondary pulmonary hypertension due to intrinsic precapillary pulmonary vascular disease.", "umls": [ "C0042373", "C0264936" ], "parsed_text": [ "Vascular Disease", "Secondary pulmonary hypertension" ] } }, { "pubchem_cid": 67548346, "pubchem_sid": [ "46507362", "48415947", "53790208", "77700624", "93166672", "103326666", "135000098", "135651541", "224259470", "251916089", "251917437", "315673618", "318160982", "319296445", "349993883", "385782552", "386393050", "386619308", "434217383", "440791117", "4527", "77700623", "160964572", "312248208", "319295364", "348275210", "419492675", "439574700", "241064319", "252156930", "252359340", "375134176", "382163213", "50026794", "77700620", "77700622", "77700628", "318692103", "319143304", "341850141", "349173894", "405481564", "434251148", "438455388", "440028223", "77700621", "77700625", "77700626", "77700627", "103825406", "126652687", "136949627", "318156204", "322079921", "375566619", "387112546", "438555563", "441072744" ], "inchikey": "WHVPOEPABJZBBY-XQYLJSSYSA-N", "generic_name": "epoprostenol", "trade_name": "Flolan", "designated_date": "1999-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "0", "marketing_approval_date": "2000-04-14", "exclusivity_end_date": "2007-04-14", "sponsor": "GlaxoSmithKline|2301 Renaissance Blvd|P. O. Box 61540|King of Prussia|Pennsylvania|19406|United States", "orphan_designation": { "original_text": "Treatment of secondary pulmonary hypertension due to intrinsic precapillary pulmonary vascular disease.", "umls": [ "C0042373", "C0264936" ], "parsed_text": [ "Vascular Disease", "Secondary pulmonary hypertension" ] } }, { "pubchem_cid": 6364626, "pubchem_sid": [ "46507362", "48415947", "53790208", "77700624", "93166672", "103326666", "135000098", "135651541", "224259470", "251916089", "251917437", "315673618", "318160982", "319296445", "349993883", "385782552", "386393050", "386619308", "434217383", "440791117", "4527", "77700623", "160964572", "312248208", "319295364", "348275210", "419492675", "439574700", "241064319", "252156930", "252359340", "375134176", "382163213", "50026794", "77700620", "77700622", "77700628", "318692103", "319143304", "341850141", "349173894", "405481564", "434251148", "438455388", "440028223", "77700621", "77700625", "77700626", "77700627", "103825406", "126652687", "136949627", "318156204", "322079921", "375566619", "387112546", "438555563", "441072744" ], "inchikey": "LMHIPJMTZHDKEW-XQYLJSSYSA-M", "generic_name": "epoprostenol", "trade_name": "Flolan", "designated_date": "1999-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "0", "marketing_approval_date": "2000-04-14", "exclusivity_end_date": "2007-04-14", "sponsor": "GlaxoSmithKline|2301 Renaissance Blvd|P. O. Box 61540|King of Prussia|Pennsylvania|19406|United States", "orphan_designation": { "original_text": "Treatment of secondary pulmonary hypertension due to intrinsic precapillary pulmonary vascular disease.", "umls": [ "C0042373", "C0264936" ], "parsed_text": [ "Vascular Disease", "Secondary pulmonary hypertension" ] } }, { "pubchem_cid": 5282411, "pubchem_sid": [ "46507362", "48415947", "53790208", "77700624", "93166672", "103326666", "135000098", "135651541", "224259470", "251916089", "251917437", "315673618", "318160982", "319296445", "349993883", "385782552", "386393050", "386619308", "434217383", "440791117", "4527", "77700623", "160964572", "312248208", "319295364", "348275210", "419492675", "439574700", "241064319", "252156930", "252359340", "375134176", "382163213", "50026794", "77700620", "77700622", "77700628", "318692103", "319143304", "341850141", "349173894", "405481564", "434251148", "438455388", "440028223", "77700621", "77700625", "77700626", "77700627", "103825406", "126652687", "136949627", "318156204", "322079921", "375566619", "387112546", "438555563", "441072744" ], "inchikey": "KAQKFAOMNZTLHT-OZUDYXHBSA-N", "generic_name": "epoprostenol", "trade_name": "Flolan", "designated_date": "1999-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "0", "marketing_approval_date": "2000-04-14", "exclusivity_end_date": "2007-04-14", "sponsor": "GlaxoSmithKline|2301 Renaissance Blvd|P. O. Box 61540|King of Prussia|Pennsylvania|19406|United States", "orphan_designation": { "original_text": "Treatment of secondary pulmonary hypertension due to intrinsic precapillary pulmonary vascular disease.", "umls": [ "C0042373", "C0264936" ], "parsed_text": [ "Vascular Disease", "Secondary pulmonary hypertension" ] } } ] }, { "_id": "UMLS:C0264936", "fda_orphan_drug": [ { "pubchem_cid": 21158562, "pubchem_sid": [ "46507362", "48415947", "53790208", "77700624", "93166672", "103326666", "135000098", "135651541", "224259470", "251916089", "251917437", "315673618", "318160982", "319296445", "349993883", "385782552", "386393050", "386619308", "434217383", "440791117", "4527", "77700623", "160964572", "312248208", "319295364", "348275210", "419492675", "439574700", "241064319", "252156930", "252359340", "375134176", "382163213", "50026794", "77700620", "77700622", "77700628", "318692103", "319143304", "341850141", "349173894", "405481564", "434251148", "438455388", "440028223", "77700621", "77700625", "77700626", "77700627", "103825406", "126652687", "136949627", "318156204", "322079921", "375566619", "387112546", "438555563", "441072744" ], "inchikey": "LMHIPJMTZHDKEW-XQYLJSSYSA-N", "generic_name": "epoprostenol", "trade_name": "Flolan", "designated_date": "1999-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "0", "marketing_approval_date": "2000-04-14", "exclusivity_end_date": "2007-04-14", "sponsor": "GlaxoSmithKline|2301 Renaissance Blvd|P. O. Box 61540|King of Prussia|Pennsylvania|19406|United States", "orphan_designation": { "original_text": "Treatment of secondary pulmonary hypertension due to intrinsic precapillary pulmonary vascular disease.", "umls": [ "C0042373", "C0264936" ], "parsed_text": [ "Vascular Disease", "Secondary pulmonary hypertension" ] } }, { "pubchem_cid": 67548346, "pubchem_sid": [ "46507362", "48415947", "53790208", "77700624", "93166672", "103326666", "135000098", "135651541", "224259470", "251916089", "251917437", "315673618", "318160982", "319296445", "349993883", "385782552", "386393050", "386619308", "434217383", "440791117", "4527", "77700623", "160964572", "312248208", "319295364", "348275210", "419492675", "439574700", "241064319", "252156930", "252359340", "375134176", "382163213", "50026794", "77700620", "77700622", "77700628", "318692103", "319143304", "341850141", "349173894", "405481564", "434251148", "438455388", "440028223", "77700621", "77700625", "77700626", "77700627", "103825406", "126652687", "136949627", "318156204", "322079921", "375566619", "387112546", "438555563", "441072744" ], "inchikey": "WHVPOEPABJZBBY-XQYLJSSYSA-N", "generic_name": "epoprostenol", "trade_name": "Flolan", "designated_date": "1999-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "0", "marketing_approval_date": "2000-04-14", "exclusivity_end_date": "2007-04-14", "sponsor": "GlaxoSmithKline|2301 Renaissance Blvd|P. 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Box 776|Spring House|Pennsylvania|19477|United States", "orphan_designation": { "original_text": "Treatment of pediatric Crohn's disease (0 through 16 years of age)", "umls": "C2931133", "parsed_text": "Pediatric Crohn's disease" } }, { "pubchem_sid": 349905851, "generic_name": "ustekinumab", "designated_date": "2016-05-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Janssen Research & Development, LLC|1400 McKean Road|P.O. Box 776|Spring House|Pennsylvania|19477|United States", "orphan_designation": { "original_text": "Treatment of pediatric Crohn's disease (0 through 16 years of age)", "umls": "C2931133", "parsed_text": "Pediatric Crohn's disease" } }, { "pubchem_sid": 347910190, "generic_name": "ustekinumab", "designated_date": "2016-05-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Janssen Research & Development, LLC|1400 McKean Road|P.O. Box 776|Spring House|Pennsylvania|19477|United States", "orphan_designation": { "original_text": "Treatment of pediatric Crohn's disease (0 through 16 years of age)", "umls": "C2931133", "parsed_text": "Pediatric Crohn's disease" } }, { "pubchem_sid": 178103467, "generic_name": "ustekinumab", "designated_date": "2016-05-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Janssen Research & Development, LLC|1400 McKean Road|P.O. Box 776|Spring House|Pennsylvania|19477|United States", "orphan_designation": { "original_text": "Treatment of pediatric Crohn's disease (0 through 16 years of age)", "umls": "C2931133", "parsed_text": "Pediatric Crohn's disease" } }, { "pubchem_sid": 135302166, "generic_name": "ustekinumab", "designated_date": "2016-05-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Janssen Research & Development, LLC|1400 McKean Road|P.O. Box 776|Spring House|Pennsylvania|19477|United States", "orphan_designation": { "original_text": "Treatment of pediatric Crohn's disease (0 through 16 years of age)", "umls": "C2931133", "parsed_text": "Pediatric Crohn's disease" } }, { "pubchem_sid": 103771533, "generic_name": "ustekinumab", "designated_date": "2016-05-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Janssen Research & Development, LLC|1400 McKean Road|P.O. Box 776|Spring House|Pennsylvania|19477|United States", "orphan_designation": { "original_text": "Treatment of pediatric Crohn's disease (0 through 16 years of age)", "umls": "C2931133", "parsed_text": "Pediatric Crohn's disease" } }, { "pubchem_sid": 387124020, "generic_name": "ustekinumab", "designated_date": "2016-05-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Janssen Research & Development, LLC|1400 McKean Road|P.O. Box 776|Spring House|Pennsylvania|19477|United States", "orphan_designation": { "original_text": "Treatment of pediatric Crohn's disease (0 through 16 years of age)", "umls": "C2931133", "parsed_text": "Pediatric Crohn's disease" } }, { "pubchem_sid": 377017835, "generic_name": "ustekinumab", "designated_date": "2016-05-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Janssen Research & Development, LLC|1400 McKean Road|P.O. Box 776|Spring House|Pennsylvania|19477|United States", "orphan_designation": { "original_text": "Treatment of pediatric Crohn's disease (0 through 16 years of age)", "umls": "C2931133", "parsed_text": "Pediatric Crohn's disease" } }, { "pubchem_sid": 355433663, "generic_name": "ustekinumab", "designated_date": "2016-05-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Janssen Research & Development, LLC|1400 McKean Road|P.O. Box 776|Spring House|Pennsylvania|19477|United States", "orphan_designation": { "original_text": "Treatment of pediatric Crohn's disease (0 through 16 years of age)", "umls": "C2931133", "parsed_text": "Pediatric Crohn's disease" } }, { "pubchem_sid": 349984255, "generic_name": "ustekinumab", "designated_date": "2016-05-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Janssen Research & Development, LLC|1400 McKean Road|P.O. Box 776|Spring House|Pennsylvania|19477|United States", "orphan_designation": { "original_text": "Treatment of pediatric Crohn's disease (0 through 16 years of age)", "umls": "C2931133", "parsed_text": "Pediatric Crohn's disease" } } ] }, { "_id": "UMLS:C5206582", "fda_orphan_drug": [ { "pubchem_cid": 3385, "pubchem_sid": [ "9851", "82653", "841046", "8149350", "26697058", "46508557", "49854375", "53787460", "76184298", "103507642", "117648200", "127324042", "127324043", "127324044", "127324045", "127324046", "127324047", "127324048", "127324049", "127324050", "127324051", "127324052", "127324053", "127324054", "127324055", "127324056", "127324057", "127324058", "127324059", "127324060", "127324061", "127324062", "127324063", "127324064", "134972265", "144075048", "160963889", "178101491", "242060174", "250007429", "252401391", "268734552", "312228218", "312616204", "316966891", "316966892", "318160069", "319062280", "340515774", "348283205", "349979663", "355039720", "355080119", 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M.D.|National Jewish Center for Immunology and Respirat|1400 Jackson Street|Denver|Colorado|80206|United States", "orphan_designation": { "original_text": "Treatment of severe steroid-requiring asthma.", "umls": "C0004096", "parsed_text": "Asthma" } }, { "pubchem_cid": 202225, "pubchem_sid": [ "583142", "7980844", "53789840", "74380522", "80395118", "89449474", "103685161", "135269994", "160849360", "160964649", "252162373", "252358273", "274349017", "312210100", "318021316", "318162559", "341152220", "347829281", "348283887", "348401645", "375201952", "385643658", "386479964", "387120288", "419491869", "434216931", "434314931", "438492352", "440064238", "441030764", "10321565", "10584900", "49999285", "80395117", "124766284", "163138548", "252401998", "332871645", "349980566", "381744891", "48416680", "56310765", "80395119", "144204274", "162224730", "315674245", "387120287", "80395122", "144075488", "268734885", "376226825", "554550", "7890679", "80395120", "80395124", "381341460", "381991390", "80395123", "103914055", "405477041", "440023700", "828816", "80395121", "342551540", "366204393", "438541963" ], "inchikey": "LQCLVBQBTUVCEQ-QTFUVMRISA-N", "generic_name": "Troleandomycin", "designated_date": "1989-09-21", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Szefler, Stanley M. M.D.|National Jewish Center for Immunology and Respirat|1400 Jackson Street|Denver|Colorado|80206|United States", "orphan_designation": { "original_text": "Treatment of severe steroid-requiring asthma.", "umls": "C0004096", "parsed_text": "Asthma" } } ] }, { "_id": "UMLS:C0009404", "fda_orphan_drug": [ { "pubchem_cid": 51039094, "pubchem_sid": [ "223370214", "254803556", "312322617", "319393097", "347828023", "347915149", "355038095", "363894216", "374263426", "375567681", "375577608", "384571401", "385649381", "386485817", "405512365", "434315260", "438493266", "440059021" ], "inchikey": "SDEAXTCZPQIFQM-UHFFFAOYSA-N", "generic_name": "tucatinib", "designated_date": "2017-09-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Seagen Inc.|21823 30th Drive Southeast|Bothell|Washington|98021|United States", "orphan_designation": { "original_text": "Treatment of HER2+ colorectal cancer", "umls": "C0009404", "parsed_text": "Colorectal Cancer" } }, { "pubchem_sid": 46506712, "generic_name": "Interferon alfa-2a (recombinant)", "designated_date": "1990-05-14", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Hoffmann-La Roche, Inc.|340 Kingsland Street|Nutley|New Jersey|7110|United States", "orphan_designation": { "original_text": "For the concomitant administration with fluorouracil for the treatment of advanced colorectal cancer.", "umls": "C0009404", "parsed_text": "Colorectal Cancer" } } ] }, { "_id": "MONDO:0015938", "fda_orphan_drug": [ { "pubchem_cid": 10133, "pubchem_sid": [ "5536", "618559", "12012589", "49700781", "49857244", "53789450", "99443291", "103542516", "103914020", "134973140", "135651455", "144080909", "160967908", "249917408", "252402756", "318163583", "319068374", "347912912", "349978316", "355040110", "385633573", "386622563", "434131882", "440063969", "440790994", "8149789", "144075347", "268734514", "376227381", 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"Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Amgen, Inc.|One Amgen Center Drive|Bldg 17, 1A-4-4, Mail Stop 17-1-B|Thousand Oaks|California|91320|United States", "orphan_designation": { "original_text": "Treatment of secondary hyperparathyroidism (HPT) in pediatric patients with chronic kidney disease receiving dialysis", "umls": [ "C0020503", "C1384672", "C1561643" ], "parsed_text": [ "Secondary hyperparathyroidism", "HPT", "Chronic Kidney Disease" ] } } ] }, { "_id": "UMLS:C1384672", "fda_orphan_drug": [ { "pubchem_cid": 156419, "pubchem_sid": [ "46506315", "49658855", "49831866", "50963794", "123094726", "126616831", "126659507", "126666798", "135182927", "152258881", "160647730", "160964348", "162202444", "163365899", "164161045", "165237999", "178100343", "184816362", "224084531", "241053841", "249582800", "251916762", "251918001", "251971349", "252215149", "252448543", "254760424", "273253068", "312228564", "313076805", "315683471", 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hyperparathyroidism (HPT) in pediatric patients with chronic kidney disease receiving dialysis", "umls": [ "C0020503", "C1384672", "C1561643" ], "parsed_text": [ "Secondary hyperparathyroidism", "HPT", "Chronic Kidney Disease" ] } } ] }, { "_id": "UMLS:C1561643", "fda_orphan_drug": [ { "pubchem_cid": 156419, "pubchem_sid": [ "46506315", "49658855", "49831866", "50963794", "123094726", "126616831", "126659507", "126666798", "135182927", "152258881", "160647730", "160964348", "162202444", "163365899", "164161045", "165237999", "178100343", "184816362", "224084531", "241053841", "249582800", "251916762", "251918001", "251971349", "252215149", "252448543", "254760424", "273253068", "312228564", "313076805", "315683471", "316900746", "318160628", "319172388", "319552288", "332848485", "340099578", "341141340", "341856064", "346452543", "348283611", "348397184", "348913181", "349978256", "350087340", "355040147", "355045279", "375590417", "378132960", "381973610", "382158682", "385646193", 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fistula and arteriovenous graft failure in patients with chronic kidney disease (CKD) who are receiving hemodialysis or preparing for hemodialysis", "umls": [ "C0003855", "C1262018", "C1561643", "C4553188" ], "parsed_text": [ "Arteriovenous fistula", "Graft failure", "Chronic Kidney Disease", "Chronic kidney disease" ] } }, { "pubchem_sid": 249807668, "generic_name": "vonapanitase", "designated_date": "2009-04-03", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Proteon Therapeutics, Inc.|200 West Street|Waltham|Massachusetts|2451|United States", "orphan_designation": { "original_text": "Prevention of arteriovenous fistula and arteriovenous graft failure in patients with chronic kidney disease (CKD) who are receiving hemodialysis or preparing for hemodialysis", "umls": [ "C0003855", "C1262018", "C1561643", "C4553188" ], "parsed_text": [ "Arteriovenous fistula", "Graft failure", "Chronic Kidney Disease", "Chronic kidney disease" ] } } ] }, { "_id": "MONDO:0002447", "fda_orphan_drug": [ { "pubchem_cid": 38072, "pubchem_sid": [ "49864147", "74588985", "85335779", "117661201", "135000935", "144205901", "160666564", "170466071", "253661910", "315682045", "318163867", "319088178", "355156294", "375561911", "377642748", "384570844", "385327665", "385766505", "385835146", "386603189", "387193478", "404771205", "434249014", "438454850", "439432529", "440719671", "440771280" ], "inchikey": "UOMKBIIXHQIERR-UHFFFAOYSA-N", "generic_name": "cridanimod", "designated_date": "2011-01-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Xenetic Biosciences, Inc.|99 Hayden Avenue|Suite 230|Lexington|Massachusetts|2421|United States", "orphan_designation": { "original_text": "Treatment of progesterone receptor negative endometrial cancer in conjunction with progesterone therapy.", "umls": "C0476089", "parsed_text": "Endometrial Cancer" } } ] }, { "_id": "UMLS:C1845862", "fda_orphan_drug": [ { "pubchem_cid": 2896, "pubchem_sid": [ "529572", "3723557", "7980747", "8149819", "46386859", "50070276", "51834597", "103444751", "117611160", "135079636", "162079661", "173131998", "178102138", "206257071", "251895295", "252363436", "252402186", "254758593", "254774983", "268736099", "274238212", "315749259", "316466569", "319061920", "322083040", "340588713", "341195358", "349996098", "355156776", "374135304", "375147422", "375671780", "376226291", "383416228", "385650079", "386486518", "433780107", "433834656", "438810484", "439439230", "440065531", "438579210" ], "inchikey": "AMHZIUVRYRVYBA-UHFFFAOYSA-N", "generic_name": "cyclocreatine", "designated_date": "2012-06-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Lumos Pharma|4200 Marathon Blvd.|Suite 200|Austin|Texas|78756|United States", "orphan_designation": { "original_text": "Treatment of creatine transporter deficiency", "umls": "C1845862", "parsed_text": "Creatine transporter deficiency" } } ] }, { "_id": "UMLS:C1423715", "fda_orphan_drug": [ { "pubchem_cid": 9832404, "pubchem_sid": [ "135261304", "152186727", "160676377", "341146230", "347828827", "355159192", "385753713", "386590370", "404618169", "434145804", "438452890", "439573808" ], "inchikey": "DWLTUUXCVGVRAV-XWRHUKJGSA-N", "generic_name": "davunetide", "designated_date": "2018-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Coronis Neurosciences Ltd.|5 Golda Me'ir Street Lev-Hanevet Building|3rd Floor, Weizmann Science Park|Ness Ziona|Center District|Israel", "orphan_designation": { "original_text": "Treatment of Activity-Dependent Neuroprotective Protein (ADNP) Syndrome", "umls": [ "C0039082", "C1423715" ], "parsed_text": [ "Syndrome", "ADNP" ] } } ] }, { "_id": "UMLS:C5194180", "fda_orphan_drug": [ { "pubchem_cid": 2972, "pubchem_sid": [ "619486", "8139956", "12014095", 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"pubchem_sid": [ "583085", "856020", "7979081", "51530976", "56422387", "56423155", "99443292", "103771485", "134995746", "144206181", "160967909", "162184652", "165235879", "187051777", "210275696", "210281355", "223441703", "252359500", "254760468", "255345965", "273549008", "310271747", "312228968", "315682219", "317223313", "318154045", "318162788", "318166575", "319223367", "319555723", "340521785", "341138133", "341149072", "341847335", "347230961", "347756810", "347914085", "347945690", "348274137", "348284089", "348919264", "349976309", "354269196", "354326321", "374100636", "374369720", "385640598", "386634853", "387172645", "404617266", "419479648", "441051322", "50019430", "51530975", "126665827", "315444698", "316966665", "316966666", "126620393", "126651368", "196109790", "316899598", "318240849", "51530974", "51530978", "252160138", "313075573", "347674068", "368500859", "434145939", "438455965", "441107291", "340523091", "375757143", "51530977", "127347213", "319437733", 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Ltd.|Route Des Arsenaux 41|(P.O. Box 61)|Fribourg|Switzerland", "orphan_designation": { "original_text": "Treatment of endogenous and traumatic anterior uveitis and panuveitis.", "umls": [ "C0030343", "C4325554" ], "parsed_text": [ "Panuveitis", "Traumatic anterior uveitis" ] } } ] }, { "_id": "UMLS:C0494165", "fda_orphan_drug": [ { "pubchem_cid": 131842305, "pubchem_sid": [ "404719169", "404772850" ], "inchikey": "DNUXJWBKTMJNEP-JVSLBXKQSA-N", "generic_name": "Disaccharide tripeptide glycerol dipalmitoyl", "designated_date": "1990-03-01", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "ImmunoTherapeutics, Inc.|3505 Riverview Circle|Moorhead|Minnesota|56560|United States", "orphan_designation": { "original_text": "Treatment of pulmonary and hepatic metastases in patients with colorectal adenocarcinoma.", "umls": [ "C0494165", "C1319315" ], "parsed_text": [ "hepatic metastases", "Colorectal Adenocarcinoma" ] } } ] }, { "_id": "MONDO:0005008", "fda_orphan_drug": [ { "pubchem_cid": 131842305, "pubchem_sid": [ "404719169", "404772850" ], "inchikey": "DNUXJWBKTMJNEP-JVSLBXKQSA-N", "generic_name": "Disaccharide tripeptide glycerol dipalmitoyl", "designated_date": "1990-03-01", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "ImmunoTherapeutics, Inc.|3505 Riverview Circle|Moorhead|Minnesota|56560|United States", "orphan_designation": { "original_text": "Treatment of pulmonary and hepatic metastases in patients with colorectal adenocarcinoma.", "umls": [ "C0494165", "C1319315" ], "parsed_text": [ "hepatic metastases", "Colorectal Adenocarcinoma" ] } } ] }, { "_id": "UMLS:C0041218", "fda_orphan_drug": [ { "pubchem_cid": 57004, "pubchem_sid": [ "103675549", "162177030", "251915896", "251917244", "374035884", "384231567", "385109725", "126645773", "135015827", "319222156", "196106720", "318160939", "318239183", "349995060", "349258398" ], "inchikey": "VKDGNNYJFSHYKD-UHFFFAOYSA-N", "generic_name": "Eflornithine HCl", "trade_name": "Ornidyl", "designated_date": "1986-04-23", "designation_status": "Designated/Approved", "marketing_approval_date": "1990-11-28", "exclusivity_end_date": "1997-11-28", "sponsor": "Hoechst Marion Roussel|P.O. Box 9627|Kansas City|Missouri|64134|United States", "orphan_designation": { "original_text": "Treatment of Trypanosoma brucei gambiense infection (sleeping sickness).", "umls": [ "C0009450", "C0041218", "C0041228" ], "parsed_text": [ "Infection", "Trypanosoma brucei gambiense", "Sleeping sickness" ] } } ] }, { "_id": "UMLS:C0206677", "fda_orphan_drug": [ { "pubchem_cid": 5472495, "pubchem_sid": [ "535132", "26759583", "50064962", "50064964", "53789110", "135116651", "135610803", "160698611", "163687272", "316966829", "318162850", "319305207", "347238568", "349989967", "355427432", "378132942", "381364336", "385789411", "386626555", "404615855", "404770162", "419544911", "434251621", "434251624", "438456097", "438456098", "438656838", "405493632", "406810173", "440040290" ], "inchikey": "MVGSNCBCUWPVDA-MFOYZWKCSA-N", "generic_name": "Exisulind", "designated_date": "1994-02-14", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "OSI Pharmaceuticals, Inc.|41 Pinelawn Road|Melville|New York|11747|United States", "orphan_designation": { "original_text": "For the suppression and control of colonic adenomatous polyps in the inherited disease adenomatous polyposis coli.", "umls": [ "C0032580", "C0206677" ], "parsed_text": [ "Adenomatous Polyposis Coli", "Adenomatous Polyps" ] } } ] }, { "_id": "UMLS:C0013884", "fda_orphan_drug": [ { "pubchem_cid": 35802, "pubchem_sid": [ "455714", "631301", "12015477", "26719867", "46386742", "48416017", "49681603", "49832353", "49882646", "51397112", "53787036", "56313744", "126631650", "126664891", "134998533", "136923751", "160870943", "162174838", "163373417", "163814336", "165234677", "172080260", "210279438", "210281761", "223379747", "223442644", "223704402", "251885911", "252359381", "254760697", "274875444", "277309219", "310264937", "312694487", "312808571", "313077442", "315673678", "316897930", "316966867", "318154074", "318161122", "319086645", "319554971", "322070720", "340509210", "340515653", "340519219", "341142516", "341832998", "346441209", "348397675", "348968015", "350095921", "354266602", "355038772", "363592718", "363902430", "374016263", "374181616", "374379586", "375134216", "375577780", "375667699", "376019627", "376163421", "376163422", "376163423", "381378713", "384568513", "384572417", "385647262", "386289736", "386483662", "387080686", "404612910", "404758051", "405479219", "406548967", "406847481", "415721621", "434251872", "438456642", "438609279", "440025878", "440771257", "405483221", "440029880", "241065734", "405240638", "438558784", "441076578" ], "inchikey": "CPEUVMUXAHMANV-UHFFFAOYSA-N", "generic_name": "flubendazole", "designated_date": "2014-01-23", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Janssen Research & Development, LLC|920 Route 202 South|Raritan|New Jersey|8869|United States", "orphan_designation": { "original_text": "Treatment of lymphatic filariasis caused by nematodes of the family Filariodidea, in children and adults.", "umls": "C0013884", "parsed_text": "Lymphatic Filariasis" } } ] }, { "_id": "UMLS:C0013537", "fda_orphan_drug": [ { "pubchem_cid": 3637, "pubchem_sid": [ "9252", "418631", "7979543", "29203528", "46507533", "48416088", "50010164", "53790673", "56313050", "56313323", "56313748", "77248068", "99437371", "103171825", "126687469", "127325709", "127325710", "127325711", "127325712", "131342035", "134963935", "134971105", "135702859", "136890497", "160964601", "175442683", "176262174", "178103898", "223950657", "250041883", "274086691", "312228766", "313077327", "315673736", "318161291", "319062425", "341141149", "341834739", "348283842", "349382920", "349988730", "363903335", "375639606", "385786553", "386623560", "387149915", "405506403", "434296349", "438459903", "440053059", "440790544", "85086711", "438541601", "441074101" ], "inchikey": "RPTUSVTUFVMDQK-UHFFFAOYSA-N", "generic_name": "Hydralazine", "designated_date": "2004-04-09", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bioniche Pharma USA LLC|272 E. 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Deerpath Road|Suite 304|Lake Forest|Illinois|60045|United States", "orphan_designation": { "original_text": "Treatment of severe intrapartum hypertension (diastolic blood pressure greater than or equal to 110 or systolic blood pressure greater than or equal to 160) associated with severe preeclampsia/eclampsia of pregnancy", "umls": [ "C0013537", "C0341950", "C1963138" ], "parsed_text": [ "Eclampsia", "Severe Preeclampsia", "Hypertension" ] } } ] }, { "_id": "UMLS:C0013604", "fda_orphan_drug": [ { "pubchem_cid": 137705994, "pubchem_sid": [ "50251422", "53790943", "99443237", "135029602", "160689039", "163352395", "163355774", "210275601", "210281260", "252479129", "254759714", "310275118", "318163481", "319332812", "319554270", "348686512", "355039131", "375297494", "377431773", "384443586", "385641174", "386635440", "404616312", "440786226", "50064327", "50251424", "135652196", "160967852", "319111505", "341141138", "348284046", "349979215", "387145817", "160645886", "312244665", 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"designated_date": "1990-02-13", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "EMD Serono, Inc.|One Technology Place|Rockland|Massachusetts|2370|United States", "orphan_designation": { "original_text": "Adjunct to gonadotropin therapy in the induction of ovulation in women with anovulatory or oligo-ovulatory infertility who fail to ovulate in response to adequate treatment with clomiphene citrate alone and gonadotropin therapy alone.", "umls": [ "C0021359", "C0429468" ], "parsed_text": [ "Infertility", "Anovulatory" ] } }, { "pubchem_sid": 441076374, "generic_name": "Somatropin", "designated_date": "1987-09-01", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Novo Nordisk Pharmaceuticals|100 Overlook Center|Suite 200|Princeton|New Jersey|8540|United States", "orphan_designation": { "original_text": "Adjunct for the induction of ovulation in women with infertility due to hypogonadotropic hypogonadism or bilateral tubal occlusion or unexplained infertility, who are undergoing in vivo or in vitro fertilization procedures.", "umls": [ "C0021359", "C0022735", "C0404585" ], "parsed_text": [ "Infertility", "Hypogonadotropic Hypogonadism", "Unexplained infertility" ] } }, { "pubchem_sid": 178101641, "generic_name": "Somatropin", "designated_date": "1987-09-01", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Novo Nordisk Pharmaceuticals|100 Overlook Center|Suite 200|Princeton|New Jersey|8540|United States", "orphan_designation": { "original_text": "Adjunct for the induction of ovulation in women with infertility due to hypogonadotropic hypogonadism or bilateral tubal occlusion or unexplained infertility, who are undergoing in vivo or in vitro fertilization procedures.", "umls": [ "C0021359", "C0022735", "C0404585" ], "parsed_text": [ "Infertility", "Hypogonadotropic Hypogonadism", "Unexplained infertility" ] } }, { "pubchem_sid": 135274454, "generic_name": "Somatropin", "designated_date": "1987-09-01", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Novo Nordisk Pharmaceuticals|100 Overlook Center|Suite 200|Princeton|New Jersey|8540|United States", "orphan_designation": { "original_text": "Adjunct for the induction of ovulation in women with infertility due to hypogonadotropic hypogonadism or bilateral tubal occlusion or unexplained infertility, who are undergoing in vivo or in vitro fertilization procedures.", "umls": [ "C0021359", "C0022735", "C0404585" ], "parsed_text": [ "Infertility", "Hypogonadotropic Hypogonadism", "Unexplained infertility" ] } }, { "pubchem_sid": 103771423, "generic_name": "Somatropin", "designated_date": "1987-09-01", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Novo Nordisk Pharmaceuticals|100 Overlook Center|Suite 200|Princeton|New Jersey|8540|United States", "orphan_designation": { "original_text": "Adjunct for the induction of ovulation in women with infertility due to hypogonadotropic hypogonadism or bilateral tubal occlusion or unexplained infertility, who are undergoing in vivo or in vitro fertilization procedures.", "umls": [ "C0021359", "C0022735", "C0404585" ], "parsed_text": [ "Infertility", "Hypogonadotropic Hypogonadism", "Unexplained infertility" ] } }, { "pubchem_sid": 50046577, "generic_name": "Somatropin", "designated_date": "1987-09-01", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Novo Nordisk Pharmaceuticals|100 Overlook Center|Suite 200|Princeton|New Jersey|8540|United States", "orphan_designation": { "original_text": "Adjunct for the induction of ovulation in women with infertility due to hypogonadotropic hypogonadism or bilateral tubal occlusion or unexplained infertility, who are undergoing in vivo or in vitro fertilization procedures.", "umls": [ "C0021359", "C0022735", "C0404585" ], "parsed_text": [ "Infertility", "Hypogonadotropic Hypogonadism", "Unexplained infertility" ] } }, { "pubchem_sid": 46506010, "generic_name": "Somatropin", "designated_date": "1987-09-01", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Novo Nordisk Pharmaceuticals|100 Overlook Center|Suite 200|Princeton|New Jersey|8540|United States", "orphan_designation": { "original_text": "Adjunct for the induction of ovulation in women with infertility due to hypogonadotropic hypogonadism or bilateral tubal occlusion or unexplained infertility, who are undergoing in vivo or in vitro fertilization procedures.", "umls": [ "C0021359", "C0022735", "C0404585" ], "parsed_text": [ "Infertility", "Hypogonadotropic Hypogonadism", "Unexplained infertility" ] } } ] }, { "_id": "UMLS:C0429468", "fda_orphan_drug": [ { "pubchem_cid": 16132412, "pubchem_sid": [ "50070151", "126682713", "135236477", "277306965", "319360640", "340470689", "341312536", "341835253", "375640125", "385109733", "404617765", "434311125", "438489692", "439498162", "441060056", "277324764", "318162248", "319360641", "341105142", "349377778", "103771338", "178103577", "252218909", "319422362" ], "inchikey": "BVLCEKWPOSAKSZ-YQMCHIOTSA-N", "generic_name": "Sermorelin acetate", "designated_date": "1990-02-13", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "EMD Serono, Inc.|One Technology Place|Rockland|Massachusetts|2370|United States", "orphan_designation": { "original_text": "Adjunct to gonadotropin therapy in the induction of ovulation in women with anovulatory or oligo-ovulatory infertility who fail to ovulate in response to adequate treatment with clomiphene citrate alone and gonadotropin therapy alone.", "umls": [ "C0021359", "C0429468" ], "parsed_text": [ "Infertility", "Anovulatory" ] } } ] }, { "_id": "UMLS:C0206731", "fda_orphan_drug": [ { "pubchem_cid": 5284616, "pubchem_sid": [ "636884", "8145920", "11528741", "12014635", "46505675", "48416541", "56310631", "56310880", "56311583", "56311585", "56311714", "56311827", "56311949", "56311983", "56312137", "56312183", "56312184", "56312185", "56312186", "56312645", "56312887", "56313330", "56313354", "56313375", "56313498", "56313803", "56313974", "56314022", "56314023", "56314562", "57288669", "93167183", "103164548", "103959411", "118043176", "135022432", "144204421", "144206303", "162037398", "178102653", "223860387", "310279616", "312234699", "315674125", "318162259", "354702922", "375577133", "385640004", "386634234", "419506776", "433780360", "53787256", "80132194", "251912147", "251915220", "319297079", "349977797", "355040746", "376033024", "434132053", "438518917", "439399970", "440064774", "440790611", "250055867", "268734410", "376228088", "387065022", "80132189", "313075823", "387142913", "10111", "842012", "74380533", "80132193", "162227201", "348283496", "384570747", "387142912", "404721339", "419590681", "131458", "80132186", "80132187", "80132188", "80132190", "80132191", "80132192", "80132195", "80132196", "80132197", "80132198", "80132199", "80132200", "80132201", "80132202", "80132203", "80132204", "80132205", "80132206", "80132207", "80132208", "103962753", "144075687", "160964218", "162224759", "164233282", "164235807", "252160702", "375582801", "381118451", "438635167" ], "inchikey": "QFJCIRLUMZQUOT-HPLJOQBZSA-N", "generic_name": "sirolimus", "designated_date": "2017-05-17", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "NobelPharma Co., LTD|12-10 Nihonbashi-kobunacho|Chuo-ku|Tokyo-to|Japan", "orphan_designation": { "original_text": "Treatment of angiofibroma associated with tuberous sclerosis", "umls": [ "C0041341", "C0206731" ], "parsed_text": [ "Tuberous Sclerosis", "Angiofibroma" ] } } ] }, { "_id": "MONDO:0005609", "fda_orphan_drug": [ { "pubchem_cid": 5282192, "pubchem_sid": [ "626315", "7980638", "12012808", "50036052", "77279719", "103265675", "104084532", "135012287", "241137006", "254784126", "274619534", "318162272", "319296331", "329735800", "341152280", "347760065", "347828317", "349895380", "349978085", "355426556", "378134551", "385641237", "386635504", "404619163", "404983007", "405021636", "406804690", "434312337", "438490676", "440980976", "405502380", "440049036", "77279715", "77279716", "77279717", "77279718", "77279720", "342564912", "438548597" ], "inchikey": "GCQYYIHYQMVWLT-HQNLTJAPSA-N", "generic_name": "Sorivudine", "designated_date": "1995-11-09", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb|5 Research Parkway|P.O. Box 5100|Wallingford|Connecticut|6492|United States", "orphan_designation": { "original_text": "Treatment of herpes zoster (shingles) in immunocompromised patients", "umls": [ "C0019360", "C4553551" ], "parsed_text": [ "Zoster", "Shingles" ] } } ] }, { "_id": "UMLS:C4553551", "fda_orphan_drug": [ { "pubchem_cid": 5282192, "pubchem_sid": [ "626315", "7980638", "12012808", "50036052", "77279719", "103265675", "104084532", "135012287", "241137006", "254784126", "274619534", "318162272", "319296331", "329735800", "341152280", "347760065", "347828317", "349895380", "349978085", "355426556", "378134551", "385641237", "386635504", "404619163", "404983007", "405021636", "406804690", "434312337", "438490676", "440980976", "405502380", "440049036", "77279715", "77279716", "77279717", "77279718", "77279720", "342564912", "438548597" ], "inchikey": "GCQYYIHYQMVWLT-HQNLTJAPSA-N", "generic_name": "Sorivudine", "designated_date": "1995-11-09", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb|5 Research Parkway|P.O. Box 5100|Wallingford|Connecticut|6492|United States", "orphan_designation": { "original_text": "Treatment of herpes zoster (shingles) in immunocompromised patients", "umls": [ "C0019360", "C4553551" ], "parsed_text": [ "Zoster", "Shingles" ] } } ] }, { "_id": "UMLS:C0728899", "fda_orphan_drug": [ { "pubchem_cid": 2724354, "pubchem_sid": [ "9800", "46504680", "50048359", "76750784", "81065526", "103771056", "134973780", "135610136", "162221387", "241059553", "255370644", "312236789", "312694458", "315674141", "318162295", "319246991", "341143136", "349975619", "355178528", "355202866", "355203258", "387068439", "402382156", "404613890", "404639074", "434313106", "438489392", "438791036", "440790456", "598336", "48416560", "76750782", "103849901", "117392416", "126670172", "135056651", "160963911", "249958968", "319067694", "348283226", "387068438", "405478573", "407344867", "440025232", "76750783", "204359704", "441073219" ], "inchikey": "ACTRVOBWPAIOHC-XIXRPRMCSA-N", "generic_name": "Succimer", "designated_date": "1991-03-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Sanofi Winthrop, Inc.|90 Park Avenue|New York|New York|10016|United States", "orphan_designation": { "original_text": "Treatment of mercury intoxication.", "umls": "C0728899", "parsed_text": "Intoxication" } } ] }, { "_id": "UMLS:C0149921", "fda_orphan_drug": [ { "pubchem_cid": 2724354, "pubchem_sid": [ "9800", "46504680", "50048359", "76750784", "81065526", "103771056", "134973780", "135610136", "162221387", "241059553", "255370644", "312236789", "312694458", "315674141", "318162295", "319246991", "341143136", "349975619", "355178528", "355202866", "355203258", "387068439", "402382156", "404613890", "404639074", "434313106", "438489392", "438791036", "440790456", "598336", "48416560", "76750782", "103849901", "117392416", "126670172", "135056651", "160963911", "249958968", "319067694", "348283226", "387068438", "405478573", "407344867", "440025232", "76750783", "204359704", "441073219" ], "inchikey": "ACTRVOBWPAIOHC-XIXRPRMCSA-N", "generic_name": "Succimer", "trade_name": "Chemet capsules", "designated_date": "1984-05-09", "designation_status": "Designated/Approved", "marketing_approval_date": "1991-01-30", "exclusivity_end_date": "1998-01-30", "sponsor": "Bock Pharmacal Company|P.O. Box 419056|Saint Louis|Missouri|63141|United States", "orphan_designation": { "original_text": "Treatment of lead poisoning in children.", "umls": "C0149921", "parsed_text": "lead poisoning in children" } } ] }, { "_id": "MONDO:0007100", "fda_orphan_drug": [ { "pubchem_cid": 11001318, "pubchem_sid": [ "135262853", "160676378", "164233887", "210275145", "210280784", "223443864", "252166590", "273477332", "312725028", "315768485", "318163487", "319344436", "319553095", "341162673", "346418200", "347726370", "347828016", "348354930", "349147644", "355039404", "374096232", "375561461", "375978062", "381320519", "382162901", "382370993", "383521002", "384416402", "385787048", "386624099", "387157479", "404617125", "404640413", "405493447", "419497481", "433841931", "433909656", "434311236", "438490237", "440040105", "440794292", "251963992", "404425809" ], "inchikey": "TXEIIPDJKFWEEC-UHFFFAOYSA-N", "generic_name": "tafamidis", "designated_date": "2006-05-23", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Pfizer, Inc.|P. O. Box 8299|Philadelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "Treatment of familial amyloid polyneuropathy", "umls": "C0206245", "parsed_text": "Familial Amyloid Polyneuropathy" } } ] }, { "_id": "MONDO:0008847", "fda_orphan_drug": [ { "pubchem_cid": 108143, "pubchem_sid": [ "583253", "12013938", "46509039", "49888292", "53789511", "56352906", "75932479", "92723326", "99431954", "103194640", "103957180", "126617579", "126646241", "126667051", "135064835", "135649930", "135698181", "136375512", "137000976", "144206278", "152258587", "160647423", "160967833", "162172213", "163124733", "170465883", "204359625", "223367317", "223373630", "241167434", "251971239", "252157313", "252216372", "252393160", "254762167", "277309586", "310280472", "312228918", "312808516", "315682283", "316539749", "317223922", "318018906", "318152614", "318163141", "319139216", "319436754", "319552612", "329977057", "336300279", "341148750", "341852451", "342506111", "347914240", "348284028", "349493370", "349976604", "354267895", "354744484", "355040370", "374035541", "375092702", "375611224", "375648737", "376034295", "377310929", "381346129", "382166614", "384568390", "384572245", "385638378", "386632572", "387060575", "387126083", "419480508", "433841946", "434311354", "438475215", "438490360", "440773449", "364179423" ], "inchikey": "MUTNCGKQJGXKEM-UHFFFAOYSA-N", "generic_name": "Tamibarotene", "designated_date": "2007-10-11", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "CytRx Corporation|11726 San Vicente Blvd.|Los Angeles|California|90049|United States", "orphan_designation": { "original_text": "Treatment of acute promyelocytic leukemia (APL).", "umls": [ "C0023487", "C1859592" ], "parsed_text": [ "Acute Promyelocytic Leukemia", "APL" ] } } ] }, { "_id": "UMLS:C4521788", "fda_orphan_drug": [ { "pubchem_cid": 6918574, "pubchem_sid": [ "12015520", "51336053", "135253405", "160709011", "162224749", "319332946", "329978338", "341711750", "347828335", "349525486", "349986709", "355428936", "381377529", "385638436", "386632630", "404616028", "419494927", "439575133", "405480407", "434311982", "438491152", "440027066", "51336054", "51336055" ], "inchikey": "MODVSQKJJIBWPZ-VLLPJHQWSA-N", "generic_name": "tesetaxel", "designated_date": "2008-11-21", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Genta, Inc.|200 Connell Drive|Berkley Heights|New Jersey|7922|United States", "orphan_designation": { "original_text": "Treatment of stages IIB, IIC, III, and stage IV melanoma", "umls": [ "C0278883", "C4521788", "C4553416" ], "parsed_text": [ "stage iv melanoma", "IIB", "IIC" ] } } ] }, { "_id": "MONDO:0001014", "fda_orphan_drug": [ { "pubchem_cid": 444795, "pubchem_sid": [ "416403", "841067", "7980826", "8143163", "11528311", "12014646", "26759442", "46504843", "53789247", "57288862", "85165342", "85240509", "87575622", "93166641", "103165713", "127320318", "127320319", "127320320", "127320321", "127320322", "127320323", "127320324", "127320325", "127320326", "127320327", "127320328", "127320329", "127320330", "127320331", "127320332", "127320333", "127320334", "134973260", "135651435", "143489246", "144076109", "152240214", "163687411", "223992671", "241059530", "241181832", "250115657", "252220278", "252448590", "254760636", "273004237", "310279663", "312605880", "312694452", "316967920", "318162514", "318242815", "319223469", "340510889", "340516106", "340520587", "347753864", "348959340", "349976458", "355039982", "374036208", "375675181", "377309923", "384249520", "384250969", "384567982", "384571703", "385639998", "386634228", "387065073", "387153240", "402408939", "404615725", "434132253", "434315742", "438472661", "438494435", "438495619", "440776351", "440791060", "126663122", "160964098", "255398180", "275872202", "277299226", "313074810", "375784747", "387153238", "387196343", "348275782", "349986946", "350096150", "377019169", "50071448", "50071494", "57391542", "419554119" ], "inchikey": "SHGAZHPCJJPHSC-YCNIQYBTSA-N", "generic_name": "Tretinoin", "designated_date": "1993-01-14", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Antigenics, Inc.|3 Forbes Road|Lexington|Massachusetts|2421|United States", "orphan_designation": { "original_text": "Treatment of acute and chronic leukemia.", "umls": "C1279296", "parsed_text": "Chronic leukemia" } } ] }, { "_id": "UMLS:C0152021", "fda_orphan_drug": [ { "pubchem_cid": 135413547, "pubchem_sid": [ "12015432", "50430522", "85335670", "99443240", "134224591", "135271929", "160687015", "160967855", "210275732", "210281393", "223922924", "312228935", "318163444", "319332931", "319446394", "319553925", "332871412", "341137041", "341187652", "346452138", "348284049", "348397597", "348939363", "349975615", "355039038", "375590570", "378132945", 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Ltd.|3rd Floor C & H Building|Gangnam-gu 135-879|Seoul|South Korea", "orphan_designation": { "original_text": "Treatment of single ventricle congenital heart disease with Fontan physiology", "umls": [ "C0152021", "C0152424" ], "parsed_text": [ "Congenital heart disease", "Single ventricle" ] } } ] }, { "_id": "UMLS:C0152424", "fda_orphan_drug": [ { "pubchem_cid": 135413547, "pubchem_sid": [ "12015432", "50430522", "85335670", "99443240", "134224591", "135271929", "160687015", "160967855", "210275732", "210281393", "223922924", "312228935", "318163444", "319332931", "319446394", "319553925", "332871412", "341137041", "341187652", "346452138", "348284049", "348397597", "348939363", "349975615", "355039038", "375590570", "378132945", "381381313", "382159294", "383470099", "385003121", "385787242", "386270814", "386624304", "387107000", "404640386", "406867550", "419480192", "433843344", "433909915", "434312786", "438492595", "438580620", "440818435", "254760553", "310281554", "341856263", "375631004", "405476659", "440023318", "50066116", "316906344", "318049737", "319434287", "434123014", "441072653" ], "inchikey": "IYFNEFQTYQPVOC-UHFFFAOYSA-N", "generic_name": "udenafil", "designated_date": "2015-08-31", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Mezzion Pharma Co. 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Box 776|Spring House|Pennsylvania|19477|United States", "orphan_designation": { "original_text": "Treatment of juvenile idiopathic arthritis (JIA) in pediatric patients", "umls": [ "C3495559", "C3714757" ], "parsed_text": [ "juvenile idiopathic arthritis", "Juvenile idiopathic arthritis" ] } }, { "pubchem_sid": 249565670, "generic_name": "sirukumab", "designated_date": "2017-07-03", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Janssen Research & Development, LLC|1400 McKean Road|P.O. Box 776|Spring House|Pennsylvania|19477|United States", "orphan_designation": { "original_text": "Treatment of juvenile idiopathic arthritis (JIA) in pediatric patients", "umls": [ "C3495559", "C3714757" ], "parsed_text": [ "juvenile idiopathic arthritis", "Juvenile idiopathic arthritis" ] } }, { "pubchem_sid": 160698800, "generic_name": "sirukumab", "designated_date": "2017-07-03", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Janssen Research & Development, LLC|1400 McKean Road|P.O. Box 776|Spring House|Pennsylvania|19477|United States", "orphan_designation": { "original_text": "Treatment of juvenile idiopathic arthritis (JIA) in pediatric patients", "umls": [ "C3495559", "C3714757" ], "parsed_text": [ "juvenile idiopathic arthritis", "Juvenile idiopathic arthritis" ] } } ] }, { "_id": "UMLS:C0030290", "fda_orphan_drug": [ { "pubchem_cid": 133566183, "pubchem_sid": [ "46506923", "50472841", "135028044", "252157050", "316539782", "319332765", "319557371", "341148489", "354274927", "355036733", "363904445", "384442019", "434313605", "438492765", "50064081", "50472842", "136350435", "316967973", "319111466", "335987027", "363743247", "375611274", "375758806", "381973751", "404620993", "434136003", "438443417", "187072157", "316935715", "318025727", "319392015", "341852841", "160967829", "312228914", "348284024", "438594413", "160687027", "385782104", "386618840", "7980392", "50472840", "135652433", "319884268", "319885215", "366663284", "368501506" ], "inchikey": 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Madera|California|94925|United States", "orphan_designation": { "original_text": "Treatment of snakebite", "umls": "C0037379", "parsed_text": "Snakebite" } } ] }, { "_id": "MONDO:0005632", "fda_orphan_drug": [ { "pubchem_cid": 23674730, "pubchem_sid": [ "80339775", "135111525", "160709015", "315729804", "319375942", "341660415", "355190226", "375590021", "385751548", "386588199", "434313669", "438492770", "103394702", "103951877" ], "inchikey": "XZZUHXILQXLTGV-UHFFFAOYSA-M", "generic_name": "varespladib sodium", "designated_date": "2007-11-19", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Anthera Pharmaceuticals, Inc.|25801 Industrial Blvd.|Suite B|Hayward|California|94545|United States", "orphan_designation": { "original_text": "Prevention of acute chest syndrome in at-risk patients with sickle cell disease", "umls": [ "C0002895", "C0742343" ], "parsed_text": [ "Sickle Cell Disease", "Acute Chest Syndrome" ] } } ] }, { "_id": "UMLS:C1695984", "fda_orphan_drug": [ { "pubchem_cid": 16129679, "pubchem_sid": [ "135000393", "319393442", "50064740", "131342402" ], "inchikey": "VBUWHHLIZKOSMS-UHFFFAOYSA-N", "generic_name": "Vasoactive intestinal polypeptide", "designated_date": "1993-06-23", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Research Triangle Pharmaceuticals|200 Westpark Corporate Center|4364 South Alston Avenue|Durham|North Carolina|27713|United States", "orphan_designation": { "original_text": "Treatment of acute esophageal food impaction.", "umls": "C1695984", "parsed_text": "Esophageal food impaction" } } ] }, { "_id": "UMLS:C0266468", "fda_orphan_drug": [ { "pubchem_cid": 46184405, "pubchem_sid": [ "134451579", "162109047", "315713799", "319392161", "347828248", "348629496", "355164144", "375628649", "375986999", "375987846", "385743584", "386580220", "404616965", "405581008", "434313692", "438492069", "439574952" ], "inchikey": "LNOVHERIIMJMDG-XZXLULOTSA-N", "generic_name": "vatiquinone", "designated_date": "2017-10-18", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "PTC Therapeutics, Inc.|100 Corporate Court|South Plainfield|New Jersey|7080|United States", "orphan_designation": { "original_text": "Treatment of pontocerebellar hypoplasia", "umls": "C0266468", "parsed_text": "Pontocerebellar Hypoplasia" } } ] }, { "_id": "UMLS:C2919945", "fda_orphan_drug": [ { "pubchem_sid": 223844000, "generic_name": "4-Hydroxy-2,2,6,6-tetramethylpiperidine-N-oxyl", "designated_date": "2015-09-29", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Recursion Pharmaceuticals, LLC|383 S. Colorow Drive|Salt Lake City|Utah|84108|United States", "orphan_designation": { "original_text": "Treatment of cerebral cavernous malformation.", "umls": "C2919945", "parsed_text": "Cerebral Cavernous Malformation" } }, { "pubchem_sid": 117651832, "generic_name": "4-Hydroxy-2,2,6,6-tetramethylpiperidine-N-oxyl", "designated_date": "2015-09-29", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Recursion Pharmaceuticals, LLC|383 S. Colorow Drive|Salt Lake City|Utah|84108|United States", "orphan_designation": { "original_text": "Treatment of cerebral cavernous malformation.", "umls": "C2919945", "parsed_text": "Cerebral Cavernous Malformation" } }, { "pubchem_sid": 53789268, "generic_name": "4-Hydroxy-2,2,6,6-tetramethylpiperidine-N-oxyl", "designated_date": "2015-09-29", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Recursion Pharmaceuticals, LLC|383 S. Colorow Drive|Salt Lake City|Utah|84108|United States", "orphan_designation": { "original_text": "Treatment of cerebral cavernous malformation.", "umls": "C2919945", "parsed_text": "Cerebral Cavernous Malformation" } } ] }, { "_id": "UMLS:C0039483", "fda_orphan_drug": [ { "pubchem_sid": 441072535, "generic_name": "Abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 440790639, "generic_name": "Abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 386474197, "generic_name": "Abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 386467149, "generic_name": "Abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 178103473, "generic_name": "Abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 135355129, "generic_name": "Abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 103771521, "generic_name": "Abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 50070209, "generic_name": "Abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 46509198, "generic_name": "Abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 441072528, "generic_name": "infliximab", "designated_date": "2003-05-06", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Centocor, Inc.|200 Great Valley Parkway|Malvern|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 440790341, "generic_name": "infliximab", "designated_date": "2003-05-06", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Centocor, Inc.|200 Great Valley Parkway|Malvern|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 433791528, "generic_name": "infliximab", "designated_date": "2003-05-06", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Centocor, Inc.|200 Great Valley Parkway|Malvern|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 433790701, "generic_name": "infliximab", "designated_date": "2003-05-06", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Centocor, Inc.|200 Great Valley Parkway|Malvern|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 387060165, "generic_name": "infliximab", "designated_date": "2003-05-06", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Centocor, Inc.|200 Great Valley Parkway|Malvern|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 354355050, "generic_name": "infliximab", "designated_date": "2003-05-06", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Centocor, Inc.|200 Great Valley Parkway|Malvern|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 348825932, "generic_name": "infliximab", "designated_date": "2003-05-06", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Centocor, Inc.|200 Great Valley Parkway|Malvern|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 329976987, "generic_name": "infliximab", "designated_date": "2003-05-06", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Centocor, Inc.|200 Great Valley Parkway|Malvern|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 178101701, "generic_name": "infliximab", "designated_date": "2003-05-06", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Centocor, Inc.|200 Great Valley Parkway|Malvern|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 135305802, "generic_name": "infliximab", "designated_date": "2003-05-06", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Centocor, Inc.|200 Great Valley Parkway|Malvern|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 103771449, "generic_name": "infliximab", "designated_date": "2003-05-06", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Centocor, Inc.|200 Great Valley Parkway|Malvern|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 91145133, "generic_name": "infliximab", "designated_date": "2003-05-06", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Centocor, Inc.|200 Great Valley Parkway|Malvern|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 56313883, "generic_name": "infliximab", "designated_date": "2003-05-06", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Centocor, Inc.|200 Great Valley Parkway|Malvern|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 56313209, "generic_name": "infliximab", "designated_date": "2003-05-06", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Centocor, Inc.|200 Great Valley Parkway|Malvern|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 56311151, "generic_name": "infliximab", "designated_date": "2003-05-06", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Centocor, Inc.|200 Great Valley Parkway|Malvern|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 50067186, "generic_name": "infliximab", "designated_date": "2003-05-06", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Centocor, Inc.|200 Great Valley Parkway|Malvern|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 46505602, "generic_name": "infliximab", "designated_date": "2003-05-06", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Centocor, Inc.|200 Great Valley Parkway|Malvern|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of giant cell arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 347911343, "generic_name": "mavrilimumab", "designated_date": "2020-09-14", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Kiniksa Pharmaceuticals, Ltd.|c/o Kiniksa Pharmaceuticals Corp.|100 Hayden Avenue|Lexington|Massachusetts|2421|United States", "orphan_designation": { "original_text": "Treatment of Giant Cell Arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 223366117, "generic_name": "mavrilimumab", "designated_date": "2020-09-14", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Kiniksa Pharmaceuticals, Ltd.|c/o Kiniksa Pharmaceuticals Corp.|100 Hayden Avenue|Lexington|Massachusetts|2421|United States", "orphan_designation": { "original_text": "Treatment of Giant Cell Arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 160693417, "generic_name": "mavrilimumab", "designated_date": "2020-09-14", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Kiniksa Pharmaceuticals, Ltd.|c/o Kiniksa Pharmaceuticals Corp.|100 Hayden Avenue|Lexington|Massachusetts|2421|United States", "orphan_designation": { "original_text": "Treatment of Giant Cell Arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } }, { "pubchem_sid": 135326482, "generic_name": "mavrilimumab", "designated_date": "2020-09-14", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Kiniksa Pharmaceuticals, Ltd.|c/o Kiniksa Pharmaceuticals Corp.|100 Hayden Avenue|Lexington|Massachusetts|2421|United States", "orphan_designation": { "original_text": "Treatment of Giant Cell Arteritis", "umls": "C0039483", "parsed_text": "Giant Cell Arteritis" } } ] }, { "_id": "MONDO:0020122", "fda_orphan_drug": [ { "pubchem_sid": 441072535, "generic_name": "abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of idiopathic inflammatory myopathy (IIM)", "umls": [ "C0751356", "C3888318" ], "parsed_text": [ "idiopathic inflammatory myopathy", "IIM" ] } }, { "pubchem_sid": 440790639, "generic_name": "abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of idiopathic inflammatory myopathy (IIM)", "umls": [ "C0751356", "C3888318" ], "parsed_text": [ "idiopathic inflammatory myopathy", "IIM" ] } }, { "pubchem_sid": 386474197, "generic_name": "abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of idiopathic inflammatory myopathy (IIM)", "umls": [ "C0751356", "C3888318" ], "parsed_text": [ "idiopathic inflammatory myopathy", "IIM" ] } }, { "pubchem_sid": 386467149, "generic_name": "abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of idiopathic inflammatory myopathy (IIM)", "umls": [ "C0751356", "C3888318" ], "parsed_text": [ "idiopathic inflammatory myopathy", "IIM" ] } }, { "pubchem_sid": 178103473, "generic_name": "abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of idiopathic inflammatory myopathy (IIM)", "umls": [ "C0751356", "C3888318" ], "parsed_text": [ "idiopathic inflammatory myopathy", "IIM" ] } }, { "pubchem_sid": 135355129, "generic_name": "abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of idiopathic inflammatory myopathy (IIM)", "umls": [ "C0751356", "C3888318" ], "parsed_text": [ "idiopathic inflammatory myopathy", "IIM" ] } }, { "pubchem_sid": 103771521, "generic_name": "abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of idiopathic inflammatory myopathy (IIM)", "umls": [ "C0751356", "C3888318" ], "parsed_text": [ "idiopathic inflammatory myopathy", "IIM" ] } }, { "pubchem_sid": 50070209, "generic_name": "abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of idiopathic inflammatory myopathy (IIM)", "umls": [ "C0751356", "C3888318" ], "parsed_text": [ "idiopathic inflammatory myopathy", "IIM" ] } }, { "pubchem_sid": 46509198, "generic_name": "abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of idiopathic inflammatory myopathy (IIM)", "umls": [ "C0751356", "C3888318" ], "parsed_text": [ "idiopathic inflammatory myopathy", "IIM" ] } } ] }, { "_id": "UMLS:C3888318", "fda_orphan_drug": [ { "pubchem_sid": 441072535, "generic_name": "abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of idiopathic inflammatory myopathy (IIM)", "umls": [ "C0751356", "C3888318" ], "parsed_text": [ "idiopathic inflammatory myopathy", "IIM" ] } }, { "pubchem_sid": 440790639, "generic_name": "abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of idiopathic inflammatory myopathy (IIM)", "umls": [ "C0751356", "C3888318" ], "parsed_text": [ "idiopathic inflammatory myopathy", "IIM" ] } }, { "pubchem_sid": 386474197, "generic_name": "abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of idiopathic inflammatory myopathy (IIM)", "umls": [ "C0751356", "C3888318" ], "parsed_text": [ "idiopathic inflammatory myopathy", "IIM" ] } }, { "pubchem_sid": 386467149, "generic_name": "abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of idiopathic inflammatory myopathy (IIM)", "umls": [ "C0751356", "C3888318" ], "parsed_text": [ "idiopathic inflammatory myopathy", "IIM" ] } }, { "pubchem_sid": 178103473, "generic_name": "abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of idiopathic inflammatory myopathy (IIM)", "umls": [ "C0751356", "C3888318" ], "parsed_text": [ "idiopathic inflammatory myopathy", "IIM" ] } }, { "pubchem_sid": 135355129, "generic_name": "abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of idiopathic inflammatory myopathy (IIM)", "umls": [ "C0751356", "C3888318" ], "parsed_text": [ "idiopathic inflammatory myopathy", "IIM" ] } }, { "pubchem_sid": 103771521, "generic_name": "abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of idiopathic inflammatory myopathy (IIM)", "umls": [ "C0751356", "C3888318" ], "parsed_text": [ "idiopathic inflammatory myopathy", "IIM" ] } }, { "pubchem_sid": 50070209, "generic_name": "abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of idiopathic inflammatory myopathy (IIM)", "umls": [ "C0751356", "C3888318" ], "parsed_text": [ "idiopathic inflammatory myopathy", "IIM" ] } }, { "pubchem_sid": 46509198, "generic_name": "abatacept", "designated_date": "2017-02-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Bristol-Myers Squibb Research & Development|Marketed Products Development - Regulatory Strategy|P.O. Box 5326|Princeton|New Jersey|8543|United States", "orphan_designation": { "original_text": "Treatment of idiopathic inflammatory myopathy (IIM)", "umls": [ "C0751356", "C3888318" ], "parsed_text": [ "idiopathic inflammatory myopathy", "IIM" ] } } ] }, { "_id": "MONDO:0006559", "fda_orphan_drug": [ { "pubchem_sid": 441072525, "generic_name": "adalimumab", "trade_name": "HUMIRA", "designated_date": "2015-05-13", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of moderate to severe hidradenitis suppurativa", "marketing_approval_date": "2015-09-09", "exclusivity_end_date": "2022-09-09", "exclusivity_protected_indication": "Treatment of moderate to severe hidradenitis suppurativa", "sponsor": "AbbVie, Inc.|1 North Waukegan Road|North Chicago|Illinois|60064|United States", "orphan_designation": { "original_text": "Treatment of moderate to severe hidradenitis suppurativa (Hurley stage 2 and Hurley stage 3 disease)", "umls": "C0162836", "parsed_text": "Hidradenitis Suppurativa" } }, { "pubchem_sid": 440790468, "generic_name": "adalimumab", "trade_name": "HUMIRA", "designated_date": "2015-05-13", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of moderate to severe hidradenitis suppurativa", "marketing_approval_date": "2015-09-09", "exclusivity_end_date": "2022-09-09", "exclusivity_protected_indication": "Treatment of moderate to severe hidradenitis suppurativa", "sponsor": "AbbVie, Inc.|1 North Waukegan Road|North Chicago|Illinois|60064|United States", "orphan_designation": { "original_text": "Treatment of moderate to severe hidradenitis suppurativa (Hurley stage 2 and Hurley stage 3 disease)", "umls": "C0162836", "parsed_text": "Hidradenitis Suppurativa" } }, { "pubchem_sid": 387061214, "generic_name": "adalimumab", "trade_name": "HUMIRA", "designated_date": "2015-05-13", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of moderate to severe hidradenitis 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Haven|Connecticut|6516|United States", "orphan_designation": { "original_text": "For replacement therapy in the alpha-1-proteinase inhibitor congenital deficiency state.", "umls": [ "C0162429", "C0333006" ], "parsed_text": [ "Deficiency state", "congenital deficiency" ] } }, { "pubchem_sid": 46506364, "generic_name": "Alpha1-proteinase inhibitor (human)", "trade_name": "Prolastin", "designated_date": "1984-12-07", "designation_status": "Designated/Approved", "approved_labeled_indication": "For chronic replacement therapy of individuals having congenital deficiency of alpha1- proteinase inhibitor with clinically demonstrable panacinar emphysema.", "marketing_approval_date": "1987-12-02", "exclusivity_end_date": "1994-12-02", "sponsor": "Bayer Corporation|Pharmaceutical Division, Biological Products|400 Morgan Lane|New Haven|Connecticut|6516|United States", "orphan_designation": { "original_text": "For replacement therapy in the alpha-1-proteinase inhibitor congenital deficiency state.", 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Arbor|Michigan|48109|United States", "orphan_designation": { "original_text": "Treatment of plastic bronchitis", "umls": "C0264342", "parsed_text": "Plastic bronchitis" } }, { "pubchem_sid": 7978677, "generic_name": "alteplase", "designated_date": "2014-10-20", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Kathleen A Stringer, PharmD, FCCP - Professor|University of %i|428 Church St.|Ann Arbor|Michigan|48109|United States", "orphan_designation": { "original_text": "Treatment of plastic bronchitis", "umls": "C0264342", "parsed_text": "Plastic bronchitis" } } ] }, { "_id": "UMLS:C2919015", "fda_orphan_drug": [ { "pubchem_sid": 349940505, "generic_name": "Ancrod", "designated_date": "1989-10-20", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Knoll Pharmaceutical Company|3000 Continental Dr., North|Mt. 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Olive|New Jersey|7828|United States", "orphan_designation": { "original_text": "To establish and maintain anticoagulation in heparin-intolerant patients undergoing cardiopulmonary bypass.", "umls": "C2919015", "parsed_text": "ANTICOAGULATION" } }, { "pubchem_sid": 50026835, "generic_name": "Ancrod", "designated_date": "1989-10-20", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Knoll Pharmaceutical Company|3000 Continental Dr., North|Mt. 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O. Box 8299|Philadelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "For the control and prevention of hemorrhagic episodes and for surgical prophylaxis in patients with hemophilia A (congenital factor VIII deficiency or classic hemophilia).", "umls": [ "C0019069", "C2674164" ], "parsed_text": [ "Hemophilia A", "Hemorrhagic episodes" ] } }, { "pubchem_sid": 46506209, "generic_name": "Antihemophilic factor (recombinant)", "trade_name": "ReFacto, Xyntha", "designated_date": "1996-02-08", "designation_status": "Designated/Approved", "marketing_approval_date": "2000-03-06", "sponsor": "Wyeth Pharmaceuticals, Inc.|P. O. 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States", "orphan_designation": { "original_text": "Prevention of bleeding episodes in patients with hemophilia A or B, with or without inhibitors", "umls": "C0019069", "parsed_text": "Hemophilia A" } }, { "pubchem_sid": 135284356, "generic_name": "Coagulation factor VIIa (recombinant)", "trade_name": "NovoSeven", "designated_date": "2004-06-18", "designation_status": "Designated/Approved", "approved_labeled_indication": "Prevention of bleeding in surgical interventions or invasive procedures in hemophilia A or B patients with inhibitors to Factor VIII or Factor IX", "marketing_approval_date": "2005-08-12", "exclusivity_end_date": "2012-08-12", "sponsor": "Novo Nordisk, Inc.|100 Overlook Center|Suite 200|Princeton|New Jersey|8540|United States", "orphan_designation": { "original_text": "Prevention of bleeding episodes in patients with hemophilia A or B, with or without inhibitors", "umls": "C0019069", "parsed_text": "Hemophilia A" } }, { "pubchem_sid": 135275129, "generic_name": 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FDA Approved for Orphan Indication", "sponsor": "Novo Nordisk, Inc.|P.O Box 846|Plainsboro|New Jersey|8536|United States", "orphan_designation": { "original_text": "Treatment of hemophilia A", "umls": "C0019069", "parsed_text": "Hemophilia A" } }, { "pubchem_sid": 404719421, "generic_name": "concizumab", "designated_date": "2018-10-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Novo Nordisk, Inc.|P.O Box 846|Plainsboro|New Jersey|8536|United States", "orphan_designation": { "original_text": "Treatment of hemophilia A", "umls": "C0019069", "parsed_text": "Hemophilia A" } }, { "pubchem_sid": 381126946, "generic_name": "concizumab", "designated_date": "2018-10-22", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Novo Nordisk, Inc.|P.O Box 846|Plainsboro|New Jersey|8536|United States", "orphan_designation": { "original_text": "Treatment of hemophilia A", "umls": 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O. Box 8299|Philadelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "For the control and prevention of hemorrhagic episodes and for surgical prophylaxis in patients with hemophilia A (congenital factor VIII deficiency or classic hemophilia).", "umls": [ "C0019069", "C2674164" ], "parsed_text": [ "Hemophilia A", "Hemorrhagic episodes" ] } }, { "pubchem_sid": 46506209, "generic_name": "Antihemophilic factor (recombinant)", "trade_name": "ReFacto, Xyntha", "designated_date": "1996-02-08", "designation_status": "Designated/Approved", "marketing_approval_date": "2000-03-06", "sponsor": "Wyeth Pharmaceuticals, Inc.|P. O. Box 8299|Philadelphia|Pennsylvania|19101|United States", "orphan_designation": { "original_text": "For the control and prevention of hemorrhagic episodes and for surgical prophylaxis in patients with hemophilia A (congenital factor VIII deficiency or classic hemophilia).", "umls": [ "C0019069", "C2674164" ], "parsed_text": [ "Hemophilia A", "Hemorrhagic episodes" ] } }, { "pubchem_sid": 381129584, "generic_name": "Coagulation factor IX (recombinant), Fc fusion protein", "trade_name": "ALPROLIX", "designated_date": "2008-10-30", "designation_status": "Designated/Approved", "approved_labeled_indication": "Adults and children with Hemophilia B for control and prevention of bleeding episodes, perioperative management, and routine prophylaxis to prevent or reduce the frequency of bleeding episodes.", "marketing_approval_date": "2014-03-28", "exclusivity_end_date": "2021-03-28", "exclusivity_protected_indication": "Adults and children with Hemophilia B for control and prevention of bleeding episodes, perioperative management, and routine prophylaxis to prevent or reduce the frequency of bleeding episodes.", "sponsor": "Bioverativ Therapeutics, Inc.|225 2nd Avenue|Waltham|Massachusetts|2451|United States", "orphan_designation": { "original_text": "Control and prevention of hemorrhagic episodes in patients with hemophilia B (congenital factor IX deficiency or Christmas disease)", "umls": [ "C0008533", "C1366464", "C2674164" ], "parsed_text": [ "Hemophilia B", "hemophilia B", "Hemorrhagic episodes" ] } } ] }, { "_id": "UMLS:C2586031", "fda_orphan_drug": [ { "pubchem_sid": 347911209, "generic_name": "Antithrombin III (human)", "trade_name": "ATnativ", "designated_date": "1985-02-08", "designation_status": "Designated/Approved", "marketing_approval_date": "1989-12-13", "exclusivity_end_date": "1996-12-13", "sponsor": "Pharmacia & Upjohn AB|Lindhagensgatan 133|Stockholm|Sweden", "orphan_designation": { "original_text": "For the treatment of patients with hereditary antithrombin 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or arresting episodes of thrombosis in patients with congenital AT-III deficiency and/or to prevent the occurrence of thrombosis in patients with AT-III deficiency who have undergone trauma or who are about to undergo surgery or parturition.", "umls": [ "C0302148", "C3714660" ], "parsed_text": [ "Thrombosis", "Trauma" ] } }, { "pubchem_sid": 135346995, "generic_name": "Antithrombin III human", "designated_date": "1986-01-02", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "American National Red Cross|9312 Old Georgetown Road|Bethesda|Maryland|20814|United States", "orphan_designation": { "original_text": "Preventing or arresting episodes of thrombosis in patients with congenital AT-III deficiency and/or to prevent the occurrence of thrombosis in patients with AT-III deficiency who have undergone trauma or who are about to undergo surgery or parturition.", "umls": [ "C0302148", "C3714660" ], "parsed_text": [ "Thrombosis", "Trauma" ] } } ] }, { "_id": "UMLS:C0040533", "fda_orphan_drug": [ { "pubchem_sid": 404771575, "generic_name": "Antivenin crotaline (pit-viper) equine immune F(ab)2)", "trade_name": "Anavip", "designated_date": "2004-01-29", "designation_status": "Designated/Approved", "approved_labeled_indication": "Management of adult and pediatric patients with North American rattlesnake envenomation", "marketing_approval_date": "2015-05-06", "exclusivity_end_date": "2022-05-06", "exclusivity_protected_indication": "Management of adult and pediatric patients with North American rattlesnake envenomation", "sponsor": "Rare Disease Therapeutics, Inc.|12975 Brookprinter Place|Suite 170|Poway|California|92064|United States", "orphan_designation": { "original_text": "Treatment of envenomation by Crotaline snakes", "umls": "C0040533", "parsed_text": "Envenomation" } } ] }, { "_id": "MONDO:0018570", "fda_orphan_drug": [ { "pubchem_sid": 381126438, "generic_name": "Asfotase alfa", "trade_name": "Strensiq", "designated_date": "2008-09-12", "designation_status": "Designated/Approved", "approved_labeled_indication": "For the treatment of patients with perinatal/infantile-and juvenile-onset hypophosphatasia (HPP)", "marketing_approval_date": "2015-10-23", "exclusivity_end_date": "2022-10-23", "exclusivity_protected_indication": "For the treatment of patients with perinatal/infantile-and juvenile-onset hypophosphatasia (HPP)", "sponsor": "Alexion Pharmaceuticals, Inc.|100 College St.|New Haven|Connecticut|6510|United States", "orphan_designation": { "original_text": "Treatment of hypophosphatasia", "umls": "C0020630", "parsed_text": "Hypophosphatasia" } }, { "pubchem_sid": 347910406, "generic_name": "Asfotase alfa", "trade_name": "Strensiq", "designated_date": "2008-09-12", "designation_status": "Designated/Approved", "approved_labeled_indication": "For the treatment of patients with perinatal/infantile-and juvenile-onset hypophosphatasia (HPP)", "marketing_approval_date": "2015-10-23", "exclusivity_end_date": "2022-10-23", "exclusivity_protected_indication": "For the treatment of patients with perinatal/infantile-and juvenile-onset hypophosphatasia (HPP)", "sponsor": "Alexion Pharmaceuticals, Inc.|100 College St.|New Haven|Connecticut|6510|United States", "orphan_designation": { "original_text": "Treatment of hypophosphatasia", "umls": "C0020630", "parsed_text": "Hypophosphatasia" } }, { "pubchem_sid": 160682278, "generic_name": "Asfotase alfa", "trade_name": "Strensiq", "designated_date": "2008-09-12", "designation_status": "Designated/Approved", "approved_labeled_indication": "For the treatment of patients with perinatal/infantile-and juvenile-onset hypophosphatasia (HPP)", "marketing_approval_date": "2015-10-23", "exclusivity_end_date": "2022-10-23", "exclusivity_protected_indication": "For the treatment of patients with perinatal/infantile-and juvenile-onset hypophosphatasia (HPP)", "sponsor": "Alexion Pharmaceuticals, Inc.|100 College St.|New Haven|Connecticut|6510|United States", "orphan_designation": { "original_text": "Treatment of hypophosphatasia", "umls": "C0020630", "parsed_text": "Hypophosphatasia" } } ] }, { "_id": "UMLS:C0270611", "fda_orphan_drug": [ { "pubchem_sid": 440234488, "generic_name": "autologous umbilical cord blood", "designated_date": "2012-07-24", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Duke University|2301 Erwin Road|Durham|North Carolina|27710|United States", "orphan_designation": { "original_text": "Treatment of pediatric (0-16 yrs old inclusive) cerebral palsy due to acquired brain injury", "umls": [ "C0007789", "C0270611" ], "parsed_text": [ "Cerebral Palsy", "Brain Injury" ] } } ] }, { "_id": "UMLS:C0014358", "fda_orphan_drug": [ { "pubchem_sid": 441073676, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 440701517, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 440064057, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 439508002, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 438644584, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 438536569, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 405235684, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 404614577, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 387113800, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 385840929, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 385834696, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 384385661, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 383548014, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 381395584, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 377311345, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 376051446, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 348746094, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 348397129, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 347913992, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 347636808, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 342544612, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 340507965, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 318153988, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 313075802, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 176331282, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 162224794, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 50071585, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 50071584, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 50071583, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 49873563, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 605350, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 4814, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 387079919, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 377014793, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 404425800, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 387079921, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 387079920, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 316962251, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 315444088, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 252402155, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 144206731, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 144204057, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 144074916, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 126676473, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 546764, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 440790689, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 438474345, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 406582657, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 374155019, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 346444061, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 335958847, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 318238404, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 316898684, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 254759847, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 204380020, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 196105735, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 163814587, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 126655275, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 126629464, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 440681435, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 387166216, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 376227004, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 341139910, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 268734448, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 162228041, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 124766072, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 103914703, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 52518470, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 8149211, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 434131544, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 373567635, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 310267241, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 162180419, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 52518469, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 7978755, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 438451119, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 434142455, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 433925319, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 350086297, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 349989661, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 348283279, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 347673985, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 312693845, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 312228282, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 224054832, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 134981168, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 103770541, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 99381480, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } }, { "pubchem_sid": 46504727, "generic_name": "Bacitracin", "designated_date": "1984-03-13", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "A. L. Laboratories, Inc.|One Executive Drive|P.O. Box 1399|Fort Lee|New Jersey|7024|United States", "orphan_designation": { "original_text": "Treatment of antibiotic-associated pseudomembranous enterocolitis caused by toxins A and B elaborated by Clostridium difficile.", "umls": "C0014358", "parsed_text": "Pseudomembranous enterocolitis" } } ] }, { "_id": "MONDO:0005249", "fda_orphan_drug": [ { "pubchem_sid": 347910369, "generic_name": "Beractant", "designated_date": "1993-12-20", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Ross Laboratories|625 Cleveland Avenue|Columbus|Ohio|43215|United States", "orphan_designation": { "original_text": "Treatment of full-term newborn infants with respiratory failure caused by meconium aspiration syndrome, persistent pulmonary hypertension of the newborn, or pneumonia and sepsis.", "umls": [ "C0025048", "C0031190", "C0032285", "C0243026", "C1145670" ], "parsed_text": [ "Meconium Aspiration Syndrome", "Persistent pulmonary hypertension of the newborn", "Pneumonia", "Sepsis", "Respiratory Failure" ] } }, { "pubchem_sid": 135335308, "generic_name": "Beractant", "designated_date": "1993-12-20", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Ross Laboratories|625 Cleveland Avenue|Columbus|Ohio|43215|United States", "orphan_designation": { "original_text": "Treatment of full-term newborn infants with respiratory failure caused by meconium aspiration syndrome, persistent pulmonary hypertension of the newborn, or pneumonia and sepsis.", "umls": [ "C0025048", "C0031190", "C0032285", "C0243026", "C1145670" ], "parsed_text": [ "Meconium Aspiration Syndrome", "Persistent pulmonary hypertension of the newborn", "Pneumonia", "Sepsis", "Respiratory Failure" ] } }, { "pubchem_sid": 103771378, "generic_name": "Beractant", "designated_date": "1993-12-20", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Ross 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"fda_orphan_drug": [ { "pubchem_sid": 347910369, "generic_name": "Beractant", "designated_date": "1993-12-20", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Ross Laboratories|625 Cleveland Avenue|Columbus|Ohio|43215|United States", "orphan_designation": { "original_text": "Treatment of full-term newborn infants with respiratory failure caused by meconium aspiration syndrome, persistent pulmonary hypertension of the newborn, or pneumonia and sepsis.", "umls": [ "C0025048", "C0031190", "C0032285", "C0243026", "C1145670" ], "parsed_text": [ "Meconium Aspiration Syndrome", "Persistent pulmonary hypertension of the newborn", "Pneumonia", "Sepsis", "Respiratory Failure" ] } }, { "pubchem_sid": 135335308, "generic_name": "Beractant", "designated_date": "1993-12-20", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Ross Laboratories|625 Cleveland Avenue|Columbus|Ohio|43215|United States", "orphan_designation": { "original_text": "Treatment of full-term newborn infants with respiratory failure caused by meconium aspiration syndrome, persistent pulmonary hypertension of the newborn, or pneumonia and sepsis.", "umls": [ "C0025048", "C0031190", "C0032285", "C0243026", "C1145670" ], "parsed_text": [ "Meconium Aspiration Syndrome", "Persistent pulmonary hypertension of the newborn", "Pneumonia", "Sepsis", "Respiratory Failure" ] } }, { "pubchem_sid": 103771378, "generic_name": "Beractant", "designated_date": "1993-12-20", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Ross Laboratories|625 Cleveland Avenue|Columbus|Ohio|43215|United States", "orphan_designation": { "original_text": "Treatment of full-term newborn infants with respiratory failure caused by meconium aspiration syndrome, persistent pulmonary hypertension of the newborn, or pneumonia and sepsis.", "umls": [ 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"designated_date": "1992-01-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of patients with cervical dystonia to reduce the severity of abnormal head position and neck pain associated with cervical dystonia.", "marketing_approval_date": "2000-12-08", "exclusivity_end_date": "2007-12-08", "sponsor": "Soltice Neurosciences, LLC|4010 Dupont Circle|Suite L-07|Louisville|Kentucky|40207|United States", "orphan_designation": { "original_text": "Treatment of cervical dystonia.", "umls": "C0152116", "parsed_text": "Cervical Dystonia" } }, { "pubchem_sid": 46504750, "generic_name": "Botulinum toxin type B", "trade_name": "Myobloc", "designated_date": "1992-01-16", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of patients with cervical dystonia to reduce the severity of abnormal head position and neck pain associated with cervical dystonia.", "marketing_approval_date": "2000-12-08", "exclusivity_end_date": "2007-12-08", "sponsor": "Soltice Neurosciences, LLC|4010 Dupont Circle|Suite L-07|Louisville|Kentucky|40207|United States", "orphan_designation": { "original_text": "Treatment of cervical dystonia.", "umls": "C0152116", "parsed_text": "Cervical Dystonia" } }, { "pubchem_sid": 440790701, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1986-08-20", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of cervical dystonia in adults to decrease the severity of abnormal head position and neck pain associated with cervical dystonia.", "marketing_approval_date": "2000-12-21", "exclusivity_end_date": "2007-12-21", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92623|United States", "orphan_designation": { "original_text": "Treatment of cervical dystonia.", "umls": "C0152116", "parsed_text": "Cervical Dystonia" } }, { "pubchem_sid": 404720407, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1986-08-20", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of cervical dystonia in adults to decrease the severity of abnormal head position and neck pain associated with cervical dystonia.", "marketing_approval_date": "2000-12-21", "exclusivity_end_date": "2007-12-21", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92623|United States", "orphan_designation": { "original_text": "Treatment of cervical dystonia.", "umls": "C0152116", "parsed_text": "Cervical Dystonia" } }, { "pubchem_sid": 135351517, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1986-08-20", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of cervical dystonia in adults to decrease the severity of abnormal head position and neck pain associated with cervical dystonia.", "marketing_approval_date": "2000-12-21", "exclusivity_end_date": "2007-12-21", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92623|United States", "orphan_designation": { "original_text": "Treatment of cervical dystonia.", "umls": "C0152116", "parsed_text": "Cervical Dystonia" } }, { "pubchem_sid": 81065599, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1986-08-20", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of cervical dystonia in adults to decrease the severity of abnormal head position and neck pain associated with cervical dystonia.", "marketing_approval_date": "2000-12-21", "exclusivity_end_date": "2007-12-21", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92623|United States", "orphan_designation": { "original_text": "Treatment of cervical dystonia.", "umls": "C0152116", "parsed_text": "Cervical Dystonia" } }, { "pubchem_sid": 49895113, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1986-08-20", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of cervical dystonia in adults to decrease the severity of abnormal head position and neck pain associated with cervical dystonia.", "marketing_approval_date": "2000-12-21", "exclusivity_end_date": "2007-12-21", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92623|United States", "orphan_designation": { "original_text": "Treatment of cervical dystonia.", "umls": "C0152116", "parsed_text": "Cervical Dystonia" } }, { "pubchem_sid": 46505347, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1986-08-20", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of cervical dystonia in adults to decrease the severity of abnormal head position and neck pain associated with cervical dystonia.", "marketing_approval_date": "2000-12-21", "exclusivity_end_date": "2007-12-21", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92623|United States", "orphan_designation": { "original_text": "Treatment of cervical dystonia.", "umls": "C0152116", "parsed_text": "Cervical Dystonia" } }, { "pubchem_sid": 10147, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1986-08-20", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of cervical dystonia in adults to decrease the severity of abnormal head position and neck pain associated with cervical dystonia.", "marketing_approval_date": "2000-12-21", "exclusivity_end_date": "2007-12-21", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92623|United States", "orphan_designation": { "original_text": "Treatment of cervical dystonia.", "umls": "C0152116", "parsed_text": "Cervical Dystonia" } }, { "pubchem_sid": 440790701, "generic_name": "Botulinum toxin type A", "designated_date": "1998-08-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Ipsen Biopharm Limited|1 Bath Road|Maidenhead|Berkshire, SL6 4UH|United Kingdom", "orphan_designation": { "original_text": "Treatment of spasmodic torticollis (cervical dystonia).", "umls": "C0152116", "parsed_text": "Spasmodic torticollis" } }, { "pubchem_sid": 404720407, "generic_name": "Botulinum toxin type A", "designated_date": "1998-08-12", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Ipsen Biopharm Limited|1 Bath Road|Maidenhead|Berkshire, SL6 4UH|United Kingdom", "orphan_designation": { "original_text": "Treatment of 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"exclusivity_end_date": "2012-08-30", "sponsor": "Ipsen Biopharmaceuticals, Inc.|106 Allen Road|Basking Ridge|New Jersey|7920|United States", "orphan_designation": { "original_text": "Treatment of growth hormone insensitivity syndrome.", "umls": "C4318479", "parsed_text": "Growth Hormone Insensitivity Syndrome" } } ] }, { "_id": "UMLS:C0014644", "fda_orphan_drug": [ { "pubchem_sid": 374364310, "generic_name": "tabelecleucel", "designated_date": "2016-02-04", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "ATARA Biotherapeutics, Inc.|4360 Park Terrace Drive|Suite 100|Westlake Village|California|91361|United States", "orphan_designation": { "original_text": "Treatment of Epstein-Barr virus-positive post-transplant lymphoproliferative disorders", "umls": [ "C0014644", "C0024314" ], "parsed_text": [ "Epstein-Barr virus", "Lymphoproliferative Disorders" ] } } ] }, { "_id": "UMLS:C0024314", "fda_orphan_drug": [ { "pubchem_sid": 374364310, "generic_name": "tabelecleucel", "designated_date": "2016-02-04", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "ATARA Biotherapeutics, Inc.|4360 Park Terrace Drive|Suite 100|Westlake Village|California|91361|United States", "orphan_designation": { "original_text": "Treatment of Epstein-Barr virus-positive post-transplant lymphoproliferative disorders", "umls": [ "C0014644", "C0024314" ], "parsed_text": [ "Epstein-Barr virus", "Lymphoproliferative Disorders" ] } }, { "pubchem_sid": 347910376, "generic_name": "Brentuximab vedotin", "trade_name": "Adcetris", "designated_date": "2016-09-07", "designation_status": "Designated/Approved", "approved_labeled_indication": "For adult patients with primary cutaneous anaplastic large cell lymphoma who have received prior systemic therapy", "marketing_approval_date": "2017-11-09", "exclusivity_end_date": "2024-11-09", "exclusivity_protected_indication": "For adult patients with primary cutaneous anaplastic large cell lymphoma who have received prior systemic therapy", "sponsor": "Seagen Inc.|21823 30th Drive Southeast|Bothell|Washington|98021|United States", "orphan_designation": { "original_text": "Treatment of primary cutaneous CD30-positive T-cell lymphoproliferative disorders", "umls": "C0024314", "parsed_text": "Lymphoproliferative Disorders" } }, { "pubchem_sid": 329976977, "generic_name": "Brentuximab vedotin", "trade_name": "Adcetris", "designated_date": "2016-09-07", "designation_status": "Designated/Approved", "approved_labeled_indication": "For adult patients with primary cutaneous anaplastic large cell lymphoma who have received prior systemic therapy", "marketing_approval_date": "2017-11-09", "exclusivity_end_date": "2024-11-09", "exclusivity_protected_indication": "For adult patients with primary cutaneous anaplastic large cell lymphoma who have received prior systemic therapy", "sponsor": "Seagen Inc.|21823 30th Drive Southeast|Bothell|Washington|98021|United States", "orphan_designation": { "original_text": "Treatment of primary cutaneous CD30-positive T-cell lymphoproliferative disorders", "umls": "C0024314", "parsed_text": "Lymphoproliferative Disorders" } }, { "pubchem_sid": 178103378, "generic_name": "Brentuximab vedotin", "trade_name": "Adcetris", "designated_date": "2016-09-07", "designation_status": "Designated/Approved", "approved_labeled_indication": "For adult patients with primary cutaneous anaplastic large cell lymphoma who have received prior systemic therapy", "marketing_approval_date": "2017-11-09", "exclusivity_end_date": "2024-11-09", "exclusivity_protected_indication": "For adult patients with primary cutaneous anaplastic large cell lymphoma who have received prior systemic therapy", "sponsor": "Seagen Inc.|21823 30th Drive Southeast|Bothell|Washington|98021|United States", "orphan_designation": { "original_text": "Treatment of primary cutaneous CD30-positive T-cell lymphoproliferative disorders", "umls": "C0024314", "parsed_text": "Lymphoproliferative Disorders" } }, { "pubchem_sid": 135300399, "generic_name": "Brentuximab vedotin", "trade_name": "Adcetris", "designated_date": "2016-09-07", "designation_status": "Designated/Approved", "approved_labeled_indication": "For adult patients with primary cutaneous anaplastic large cell lymphoma who have received prior systemic therapy", "marketing_approval_date": "2017-11-09", "exclusivity_end_date": "2024-11-09", "exclusivity_protected_indication": "For adult patients with primary cutaneous anaplastic large cell lymphoma who have received prior systemic therapy", "sponsor": "Seagen Inc.|21823 30th Drive Southeast|Bothell|Washington|98021|United States", "orphan_designation": { "original_text": "Treatment of primary cutaneous CD30-positive T-cell lymphoproliferative disorders", "umls": "C0024314", "parsed_text": "Lymphoproliferative Disorders" } }, { "pubchem_sid": 131277422, "generic_name": "Brentuximab vedotin", "trade_name": "Adcetris", "designated_date": "2016-09-07", "designation_status": "Designated/Approved", "approved_labeled_indication": "For adult patients with primary cutaneous anaplastic large cell lymphoma who have received prior systemic therapy", "marketing_approval_date": "2017-11-09", "exclusivity_end_date": "2024-11-09", "exclusivity_protected_indication": "For adult patients with primary cutaneous anaplastic large cell lymphoma who have received prior systemic therapy", "sponsor": "Seagen Inc.|21823 30th Drive Southeast|Bothell|Washington|98021|United States", "orphan_designation": { "original_text": "Treatment of primary cutaneous CD30-positive T-cell lymphoproliferative disorders", "umls": "C0024314", "parsed_text": "Lymphoproliferative Disorders" } } ] }, { "_id": "MONDO:0011728", "fda_orphan_drug": [ { "pubchem_sid": 440790701, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1984-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of blepharospasm associated with dystonia in adults (patients 12 years of age and above)", "marketing_approval_date": "1989-12-30", "exclusivity_end_date": "1996-12-30", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92713|United States", "orphan_designation": { "original_text": "Treatment of strabismus and blepharospasms", "umls": [ "C0005747", "C0038379" ], "parsed_text": [ "Blepharospasms", "Strabismus" ] } }, { "pubchem_sid": 404720407, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1984-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of blepharospasm associated with dystonia in adults (patients 12 years of age and above)", "marketing_approval_date": "1989-12-30", "exclusivity_end_date": "1996-12-30", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92713|United States", "orphan_designation": { "original_text": "Treatment of strabismus and blepharospasms", "umls": [ "C0005747", "C0038379" ], "parsed_text": [ "Blepharospasms", "Strabismus" ] } }, { "pubchem_sid": 135351517, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1984-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of blepharospasm associated with dystonia in adults (patients 12 years of age and above)", "marketing_approval_date": "1989-12-30", "exclusivity_end_date": "1996-12-30", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92713|United States", "orphan_designation": { "original_text": "Treatment of strabismus and blepharospasms", "umls": [ "C0005747", "C0038379" ], "parsed_text": [ "Blepharospasms", "Strabismus" ] } }, { "pubchem_sid": 81065599, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1984-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of blepharospasm associated with dystonia in adults (patients 12 years of age and above)", "marketing_approval_date": "1989-12-30", "exclusivity_end_date": "1996-12-30", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92713|United States", "orphan_designation": { "original_text": "Treatment of strabismus and blepharospasms", "umls": [ "C0005747", "C0038379" ], "parsed_text": [ "Blepharospasms", "Strabismus" ] } }, { "pubchem_sid": 49895113, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1984-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of blepharospasm associated with dystonia in adults (patients 12 years of age and above)", "marketing_approval_date": "1989-12-30", "exclusivity_end_date": "1996-12-30", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92713|United States", "orphan_designation": { "original_text": "Treatment of strabismus and blepharospasms", "umls": [ "C0005747", "C0038379" ], "parsed_text": [ "Blepharospasms", "Strabismus" ] } }, { "pubchem_sid": 46505347, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1984-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of blepharospasm associated with dystonia in adults (patients 12 years of age and above)", "marketing_approval_date": "1989-12-30", "exclusivity_end_date": "1996-12-30", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92713|United States", "orphan_designation": { "original_text": "Treatment of strabismus and blepharospasms", "umls": [ "C0005747", "C0038379" ], "parsed_text": [ "Blepharospasms", "Strabismus" ] } }, { "pubchem_sid": 10147, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1984-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of blepharospasm associated with dystonia in adults (patients 12 years of age and above)", "marketing_approval_date": "1989-12-30", "exclusivity_end_date": "1996-12-30", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92713|United States", "orphan_designation": { "original_text": "Treatment of strabismus and blepharospasms", "umls": [ "C0005747", "C0038379" ], "parsed_text": [ "Blepharospasms", "Strabismus" ] } }, { "pubchem_sid": 440790701, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1984-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of strabismus associated with dystonia in adults (patients 12 years of age and above)", "marketing_approval_date": "1989-12-29", "exclusivity_end_date": "1996-12-29", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92713|United States", "orphan_designation": { "original_text": "Treatment of strabismus and blepharospasms", "umls": [ "C0005747", "C0038379" ], "parsed_text": [ "Blepharospasms", "Strabismus" ] } }, { "pubchem_sid": 404720407, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1984-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of strabismus associated with dystonia in adults (patients 12 years of age and above)", "marketing_approval_date": "1989-12-29", "exclusivity_end_date": "1996-12-29", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92713|United States", "orphan_designation": { "original_text": "Treatment of strabismus and blepharospasms", "umls": [ "C0005747", "C0038379" ], "parsed_text": [ "Blepharospasms", "Strabismus" ] } }, { "pubchem_sid": 135351517, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1984-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of strabismus associated with dystonia in adults (patients 12 years of age and above)", "marketing_approval_date": "1989-12-29", "exclusivity_end_date": "1996-12-29", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92713|United States", "orphan_designation": { "original_text": "Treatment of strabismus and blepharospasms", "umls": [ "C0005747", "C0038379" ], "parsed_text": [ "Blepharospasms", "Strabismus" ] } }, { "pubchem_sid": 81065599, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1984-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of strabismus associated with dystonia in adults (patients 12 years of age and above)", "marketing_approval_date": "1989-12-29", "exclusivity_end_date": "1996-12-29", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92713|United States", "orphan_designation": { "original_text": "Treatment of strabismus and blepharospasms", "umls": [ "C0005747", "C0038379" ], "parsed_text": [ "Blepharospasms", "Strabismus" ] } }, { "pubchem_sid": 49895113, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1984-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of strabismus associated with dystonia in adults (patients 12 years of age and above)", "marketing_approval_date": "1989-12-29", "exclusivity_end_date": "1996-12-29", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92713|United States", "orphan_designation": { "original_text": "Treatment of strabismus and blepharospasms", "umls": [ "C0005747", "C0038379" ], "parsed_text": [ "Blepharospasms", "Strabismus" ] } }, { "pubchem_sid": 46505347, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1984-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of strabismus associated with dystonia in adults (patients 12 years of age and above)", "marketing_approval_date": "1989-12-29", "exclusivity_end_date": "1996-12-29", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92713|United States", "orphan_designation": { "original_text": "Treatment of strabismus and blepharospasms", "umls": [ "C0005747", "C0038379" ], "parsed_text": [ "Blepharospasms", "Strabismus" ] } }, { "pubchem_sid": 10147, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1984-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of strabismus associated with dystonia in adults (patients 12 years of age and above)", "marketing_approval_date": "1989-12-29", "exclusivity_end_date": "1996-12-29", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92713|United States", "orphan_designation": { "original_text": "Treatment of strabismus and blepharospasms", "umls": [ "C0005747", "C0038379" ], "parsed_text": [ "Blepharospasms", "Strabismus" ] } } ] }, { "_id": "MONDO:0003432", "fda_orphan_drug": [ { "pubchem_sid": 440790701, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1984-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of blepharospasm associated with dystonia in adults (patients 12 years of age and above)", "marketing_approval_date": "1989-12-30", "exclusivity_end_date": "1996-12-30", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. 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Box 19534|Irvine|California|92713|United States", "orphan_designation": { "original_text": "Treatment of strabismus and blepharospasms", "umls": [ "C0005747", "C0038379" ], "parsed_text": [ "Blepharospasms", "Strabismus" ] } }, { "pubchem_sid": 46505347, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1984-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of blepharospasm associated with dystonia in adults (patients 12 years of age and above)", "marketing_approval_date": "1989-12-30", "exclusivity_end_date": "1996-12-30", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92713|United States", "orphan_designation": { "original_text": "Treatment of strabismus and blepharospasms", "umls": [ "C0005747", "C0038379" ], "parsed_text": [ "Blepharospasms", "Strabismus" ] } }, { "pubchem_sid": 10147, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1984-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of blepharospasm associated with dystonia in adults (patients 12 years of age and above)", "marketing_approval_date": "1989-12-30", "exclusivity_end_date": "1996-12-30", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92713|United States", "orphan_designation": { "original_text": "Treatment of strabismus and blepharospasms", "umls": [ "C0005747", "C0038379" ], "parsed_text": [ "Blepharospasms", "Strabismus" ] } }, { "pubchem_sid": 440790701, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1984-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of strabismus associated with dystonia in adults (patients 12 years of age and above)", "marketing_approval_date": "1989-12-29", "exclusivity_end_date": "1996-12-29", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92713|United States", "orphan_designation": { "original_text": "Treatment of strabismus and blepharospasms", "umls": [ "C0005747", "C0038379" ], "parsed_text": [ "Blepharospasms", "Strabismus" ] } }, { "pubchem_sid": 404720407, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1984-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of strabismus associated with dystonia in adults (patients 12 years of age and above)", "marketing_approval_date": "1989-12-29", "exclusivity_end_date": "1996-12-29", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92713|United States", "orphan_designation": { "original_text": "Treatment of strabismus and blepharospasms", "umls": [ "C0005747", "C0038379" ], "parsed_text": [ "Blepharospasms", "Strabismus" ] } }, { "pubchem_sid": 135351517, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1984-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of strabismus associated with dystonia in adults (patients 12 years of age and above)", "marketing_approval_date": "1989-12-29", "exclusivity_end_date": "1996-12-29", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92713|United States", "orphan_designation": { "original_text": "Treatment of strabismus and blepharospasms", "umls": [ "C0005747", "C0038379" ], "parsed_text": [ "Blepharospasms", "Strabismus" ] } }, { "pubchem_sid": 81065599, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1984-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of strabismus associated with dystonia in adults (patients 12 years of age and above)", "marketing_approval_date": "1989-12-29", "exclusivity_end_date": "1996-12-29", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92713|United States", "orphan_designation": { "original_text": "Treatment of strabismus and blepharospasms", "umls": [ "C0005747", "C0038379" ], "parsed_text": [ "Blepharospasms", "Strabismus" ] } }, { "pubchem_sid": 49895113, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1984-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of strabismus associated with dystonia in adults (patients 12 years of age and above)", "marketing_approval_date": "1989-12-29", "exclusivity_end_date": "1996-12-29", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92713|United States", "orphan_designation": { "original_text": "Treatment of strabismus and blepharospasms", "umls": [ "C0005747", "C0038379" ], "parsed_text": [ "Blepharospasms", "Strabismus" ] } }, { "pubchem_sid": 46505347, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1984-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of strabismus associated with dystonia in adults (patients 12 years of age and above)", "marketing_approval_date": "1989-12-29", "exclusivity_end_date": "1996-12-29", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92713|United States", "orphan_designation": { "original_text": "Treatment of strabismus and blepharospasms", "umls": [ "C0005747", "C0038379" ], "parsed_text": [ "Blepharospasms", "Strabismus" ] } }, { "pubchem_sid": 10147, "generic_name": "Botulinum toxin type A", "trade_name": "Botox", "designated_date": "1984-03-22", "designation_status": "Designated/Approved", "approved_labeled_indication": "Treatment of strabismus associated with dystonia in adults (patients 12 years of age and above)", "marketing_approval_date": "1989-12-29", "exclusivity_end_date": "1996-12-29", "sponsor": "Allergan, Inc.|2525 Dupont Drive|P.O. Box 19534|Irvine|California|92713|United States", "orphan_designation": { "original_text": "Treatment of strabismus and blepharospasms", "umls": [ "C0005747", "C0038379" ], "parsed_text": [ "Blepharospasms", "Strabismus" ] } } ] }, { "_id": "UMLS:C1258104", "fda_orphan_drug": [ { "pubchem_sid": 347910234, "generic_name": "Recombinant human relaxin", "designated_date": "1995-11-03", "designation_status": "Designated/Withdrawn", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "Connetics Corporation|3400 West Bayshore Road|Palo Alto|California|94303|United States", "orphan_designation": { "original_text": "Treatment of progressive systemic sclerosis.", "umls": "C1258104", "parsed_text": "Progressive systemic sclerosis" } } ] }, { "_id": "MONDO:0009662", "fda_orphan_drug": [ { "pubchem_sid": 381127826, "generic_name": "vestronidase alfa-vjbk", "trade_name": "Mepsevii", "designated_date": "2012-02-16", "designation_status": "Designated/Approved", 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{ "pubchem_sid": 381127311, "generic_name": "Vasomera", "designated_date": "2015-11-19", "designation_status": "Designated", "approval_status": "Not FDA Approved for Orphan Indication", "sponsor": "PhaseBio Pharmaceuticals, Inc.|1 Great Valley Parkway|Suite 30|Malvern|Pennsylvania|19355|United States", "orphan_designation": { "original_text": "Treatment of cardiomyopathy associated with dystrophinopathies; Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy BMD), and X-linked dilated cardiomyopathy (XL-dCMP).", "umls": [ "C0013264", "C0339510", "C0878544", "C0917713", "C1414083", "C1512016", "C3668940" ], "parsed_text": [ "DUCHENNE MUSCULAR DYSTROPHY", "BMD", "Cardiomyopathy", "Becker Muscular Dystrophy", "DMD", "DMD", "X-linked dilated cardiomyopathy" ] } } ] }, { "_id": "MONDO:0005700", "fda_orphan_drug": [ { "pubchem_sid": 381127735, "generic_name": "Varicella Zoster Immune Globulin (Human)", "trade_name": "VARIZIG", "designated_date": "2006-11-07", "designation_status": 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