Amicus Therapeutics
Amicus Therapeutics, headquartered in Princeton, New Jersey, is a commercial-stage rare-disease biotech focused on lysosomal storage disorders. It markets GALAFOLD (migalastat), an oral pharmacological chaperone that stabilizes a defective enzyme in Fabry disease, and POMBILITI + OPFOLDA, an enzyme replacement therapy combination for late-onset Pompe disease.
- Lead asset
- GALAFOLD · Approved · Fabry Disease
- small molecule · pharmacological chaperone of α-galactosidase A
- Pipeline
- 1 drug · 1 program
- 1 Rare Disease
- Modalities
- small molecule×1
Peers in this space
Pipeline & catalysts
- Preclin0
- Ph10
- Ph20
- Ph30
- Filed0
- Approved1
Past catalyst impact
Insider activity
Congressional trading
Top ETF holders
KOL network
Federal lobbying
- Patent Trial and Appeal Board reforms$60K
- CMS Quality Reporting Program$20K
- Inflation Reduction Act drug-price negotiation$14K
- Pharmacy Benefit Manager (PBM) reform$14K
- CMS reimbursement rates for ultra-rare conditions$11K