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Full detail: Where this data comes from
Amicus Therapeutics is a global rare disease biotechnology company focused on discovering, developing, and commercializing medicines for people living with genetically defined conditions. Founded on February 4, 2002 and headquartered in Princeton, New Jersey, Amicus markets two approved therapies: Galafold (migalastat) for adults with Fabry disease who have an amenable GLA variant, and the two-component Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) regimen for adults with late-onset Pompe disease.
BioMarin Pharmaceutical agreed to acquire Amicus in December 2025 for $4.8 billion and completed the acquisition on April 27, 2026. Amicus now operates as a wholly owned subsidiary of BioMarin; the NASDAQ ticker FOLD has been delisted.
- Website: https://www.amicusrx.com
- Parent company: BioMarin Pharmaceutical
- Investor relations (BioMarin): https://investors.biomarin.com/
- Headquarters: Princeton, New Jersey
No public commercial developer APIs, SDKs, or GitHub organization were identified for Amicus Therapeutics. Like most pre-acquisition rare-disease biotechs, Amicus's digital surface is composed of corporate, product, patient-support, and clinical-trial websites. Investor and developer-adjacent assets have moved under the BioMarin umbrella post-acquisition.
| Brand | Generic | Class | Route | Indication | Site |
|---|---|---|---|---|---|
| Galafold | migalastat | Pharmacological Chaperone | Oral | Fabry disease (adults with amenable GLA variant) | galafold.com |
| Pombiliti | cipaglucosidase alfa-atga | Enzyme Replacement Therapy | Intravenous | Late-onset Pompe disease (with Opfolda) | pombilitiopfolda.com |
| Opfolda | miglustat | Enzyme Stabilizer | Oral | Late-onset Pompe disease (with Pombiliti) | pombilitiopfolda.com |
Galafold received European Commission approval in May 2016 as the first and only oral therapy for adults with Fabry disease who carry an amenable GLA variant.
| Study | Indication | Phase / Type | NCT |
|---|---|---|---|
| Pediatric Migalastat Study | Fabry disease (ages 2 to <12) | Interventional | NCT06904261 |
| AT1001-025 | Fabry disease (renal monotherapy) | Phase 3 | NCT04020055 |
| AT1001-037 | Fabry disease (women, observational) | Observational | NCT04252066 |
| ROSSELLA (ATB200-08) | Infantile-onset Pompe disease | Phase 3 | NCT04808505 |
| ZIP (ATB200-04) | Late-onset Pompe disease (pediatric) | Phase 3 | NCT03911505 |
| POM-005 Registry | Pompe disease (global registry) | Observational | NCT06121011 |
| ACTION3 (DMX-200) | Focal segmental glomerulosclerosis | Phase 3 | NCT05183646 |
| Schema | File |
|---|---|
| Amicus Medicine | json-schema/amicus-medicine-schema.json |
| Amicus Clinical Trial | json-schema/amicus-clinical-trial-schema.json |
| Structure | File |
|---|---|
| Amicus Medicine | json-structure/amicus-medicine-structure.json |
| Amicus Clinical Trial | json-structure/amicus-clinical-trial-structure.json |
| Context | File |
|---|---|
| Amicus Therapeutics | json-ld/amicus-therapeutics-context.jsonld |
| Example | File |
|---|---|
| Galafold Medicine | examples/amicus-galafold-medicine-example.json |
| Pombiliti Medicine | examples/amicus-pombiliti-medicine-example.json |
| ROSSELLA Trial | examples/amicus-rossella-trial-example.json |
| Vocabulary | File |
|---|---|
| Amicus Therapeutics | vocabulary/amicus-therapeutics-vocabulary.yml |