Amicus Therapeutics
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Historical price data
Overview
Amicus Therapeutics is a fully integrated, global biotech with a mission to address the vast unmet need in rare diseases, where 95% of over 10,000 known conditions lack approved treatments. The company has achieved commercial success with two approved therapies for Fabry and Pompe disease, validating its dual-technology platform. Its strategy centers on deepening its impact in core lysosomal disorders, expanding into adjacent rare diseases like FSGS, and maintaining a patient-centric culture that drives innovation and operational execution.
Technology Platform
Proprietary pharmacological chaperone technology for oral small-molecule therapies and a next-generation enzyme replacement therapy (ERT) platform designed to enhance tissue targeting and cellular uptake of recombinant enzymes.
Pipeline
43| Drug | Indication | Stage | Watch |
|---|---|---|---|
| Cipaglucosidase Alfa + Miglustat | Pompe Disease (Late-onset) | Phase 3 | |
| migalastat hydrochloride + agalsidase | Fabry Disease | Phase 3 | |
| migalastat hydrochloride + Placebo | Fabry Disease | Phase 3 | |
| Migalastat HCl 20 mg | Fabry Disease | Phase 3 | |
| AT2221 + ATB200 | Pompe Disease (Late-onset) | Phase 3 |
Funding History
4FDA Approved Drugs
3Company Timeline
Founded in Philadelphia, United States
IPO — $60.0M
Series B: $60.0M
FDA Approval: GALAFOLD
Debt: $225.0M
FDA Approval: OPFOLDA