{ "description": "Example Amicus medicine record for Galafold (migalastat), Amicus's first-and-only oral therapy for adults with Fabry disease who have an amenable GLA variant.", "medicine": { "medicineId": "amx-med-galafold", "brandName": "Galafold", "genericName": "migalastat", "drugClass": "Pharmacological Chaperone", "routeOfAdministration": "Oral", "indication": "Fabry disease", "patientPopulation": "Adults with Fabry disease who have a GLA gene variant that is responsive (amenable) to Galafold", "combinationWith": [], "mechanismOfAction": "Selective, reversible binding to amenable mutant forms of alpha-galactosidase A to stabilize the enzyme, facilitate lysosomal trafficking, and restore activity", "approvals": [ { "region": "EU", "agency": "European Commission", "approvalDate": "2016-05-26" } ], "productSite": "https://www.galafold.com" } }