aid: amicus-therapeutics
url: https://raw.githubusercontent.com/api-evangelist/amicus-therapeutics/refs/heads/main/apis.yml
modified: '2026-05-23'
description: >-
  Amicus Therapeutics is a global rare disease biotechnology company focused on
  discovering, developing, and commercializing medicines for people living with
  genetically defined conditions. Founded in 2002 and headquartered in
  Princeton, New Jersey, Amicus markets two approved therapies: Galafold
  (migalastat) — the first and only oral therapy for adults with Fabry disease
  who have an amenable GLA variant, approved by the European Commission in May
  2016 — and the two-component Pombiliti (cipaglucosidase alfa-atga) + Opfolda
  (miglustat) enzyme replacement / stabilizer combination for adults with
  late-onset Pompe disease. Amicus operates wholly owned subsidiaries in
  Australia, Canada, France, Germany, Italy, Japan, the Netherlands, Spain,
  Switzerland, and the United Kingdom. BioMarin Pharmaceutical Inc. agreed to
  acquire Amicus in December 2025 for $4.8 billion; the acquisition completed
  on April 27, 2026 and Amicus now operates as a wholly owned subsidiary of
  BioMarin, having previously traded on NASDAQ under the ticker FOLD. The
  company maintains no publicly documented commercial developer APIs, SDKs, or
  GitHub organization; its digital surface is composed of corporate, product,
  patient-support, and clinical-trial websites.
apis: []
common:
  - type: DomainSecurity
    url: security/amicus-therapeutics-domain-security.yml
  - type: Website
    url: https://www.amicusrx.com
  - type: About
    url: https://www.amicusrx.com/about-amicus/
  - type: Products
    url: https://www.amicusrx.com/our-medicines/
  - type: Pipeline
    url: https://www.amicusrx.com/our-research-and-pipeline/
  - type: ClinicalTrials
    url: https://www.amicusrx.com/our-research-and-pipeline/clinical-trials/
  - type: ExpandedAccess
    url: https://www.amicusrx.com/our-research-and-pipeline/expanded-access-program/
  - type: PatientAdvocacy
    url: https://www.amicusrx.com/for-patients/patient-advocacy/
  - type: ProductSite
    url: https://www.galafold.com
  - type: ProductSite
    url: https://www.pombilitiopfolda.com
  - type: ParentCompany
    url: https://www.biomarin.com
  - type: Investors
    url: https://investors.biomarin.com/
  - type: Careers
    url: https://www.biomarin.com/careers/
  - type: LinkedIn
    url: https://www.linkedin.com/company/amicus-therapeutics
  - type: Vocabulary
    url: https://raw.githubusercontent.com/api-evangelist/amicus-therapeutics/refs/heads/main/vocabulary/amicus-therapeutics-vocabulary.yml
  - type: JSONLD
    url: https://raw.githubusercontent.com/api-evangelist/amicus-therapeutics/refs/heads/main/json-ld/amicus-therapeutics-context.jsonld
products:
  - name: Galafold
    genericName: migalastat
    indication: Fabry disease (adults with amenable GLA variant)
    mechanism: Oral pharmacological chaperone
    approvalEC: '2016-05'
    productSite: https://www.galafold.com
  - name: Pombiliti
    genericName: cipaglucosidase alfa-atga
    indication: Late-onset Pompe disease (adults, in combination with Opfolda)
    mechanism: Bis-M6P-enriched enzyme replacement therapy
    productSite: https://www.pombilitiopfolda.com
  - name: Opfolda
    genericName: miglustat
    indication: Late-onset Pompe disease (adults, in combination with Pombiliti)
    mechanism: Oral enzyme stabilizer
    productSite: https://www.pombilitiopfolda.com
pipeline:
  - name: Pediatric Migalastat Study
    indication: Fabry disease (ages 2 to <12)
    nct: NCT06904261
  - name: AT1001-025
    indication: Fabry disease — renal monotherapy
    phase: Phase 3
    nct: NCT04020055
  - name: AT1001-037
    indication: Fabry disease — prospective observational study of women
    nct: NCT04252066
  - name: ROSSELLA (ATB200-08)
    indication: Infantile-onset Pompe disease (ages 0 to <18)
    phase: Phase 3
    nct: NCT04808505
  - name: ZIP (ATB200-04)
    indication: Late-onset Pompe disease (ages 0 to <18)
    phase: Phase 3
    nct: NCT03911505
  - name: POM-005 Registry
    indication: Pompe disease — global prospective observational registry
    nct: NCT06121011
  - name: ACTION3 (DMX-200)
    indication: Focal segmental glomerulosclerosis
    phase: Phase 3
    nct: NCT05183646
tags:
  - Biotechnology
  - Pharmaceuticals
  - Rare Diseases
  - Genetic Diseases
  - Fabry Disease
  - Pompe Disease
  - Lysosomal Storage Disorders
  - Enzyme Replacement Therapy
  - Pharmacological Chaperones
  - Clinical Research
  - Life Sciences
  - Patient Advocacy
maintainers:
  - FN: API Evangelist
    url: https://apievangelist.com