amicus-therapeutics

aid: amicus-therapeutics
accessModel:
  pricing: unknown
  onboarding: unknown
  trial: false
  try_now: false
  public: false
  label: Unknown
  confidence: low
  source: []
  generated: '2026-07-22'
  method: derived
image: https://kinlane-images.s3.amazonaws.com/shared/apis-json/icons/amicus-therapeutics.png
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