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About AMICUS THERAPEUTICS, INC.
Amicus Therapeutics, Inc. operates as a biotechnology company. The company is headquartered in Princeton, New Jersey and currently employs 517 full-time employees. The company went IPO on 2007-05-31. Its two marketed therapies are Galafold, the first oral monotherapy for people living with Fabry disease who have amenable genetic variants, and Pombiliti + Opfolda, a novel treatment designed to improve uptake of active enzyme into key disease relevant tissues for adults living with late-onset Pompe disease. As an orally administered monotherapy, Galafold is designed to bind to and stabilize an endogenous alpha-galactosidase A (alpha-Gal A) enzyme in those patients with genetic variants identified as amenable in a Good Laboratory Practice (GLP) cell-based amenability assay. Pombiliti + Opfolda consists of a uniquely engineered rhGAA enzyme, cipaglucosidase alfa-atga, with an optimized carbohydrate structure to enhance lysosomal uptake, administered in combination with miglustat that functions as an enzyme stabilizer.
Industry: Pharmaceutical Preparations Peers: ACADIA PHARMACEUTICALS INC ARROWHEAD PHARMACEUTICALS, INC. Beam Therapeutics Inc. ChemoCentryx, Inc. Denali Therapeutics Inc. INSMED Inc PTC THERAPEUTICS, INC. Ultragenyx Pharmaceutical Inc. Vir Biotechnology, Inc.