Amicus Therapeutics

Amicus Therapeutics, Inc. is a biotechnology company, which engages in discovering, developing, and delivering medicines for people living with metabolic diseases. The company is headquartered in Princeton, New Jersey and currently employs 511 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 two-component treatment for adults living with late-onset Pompe disease. As an orally miglustat monotherapy, Galafold is designed to bind to and stabilize an endogenous alpha-galactosidase A enzyme in those patients with genetic variants identified as amenable in a Good Laboratory Practice cell-based amenability assay. Pombiliti + Opfolda consists of an engineered rhGAA enzyme, cipaglucosidase alfa-atga, with an optimized carbohydrate structure to enhance cellular uptake, administered intravenously in combination with orally administered miglustat. In addition, clinical studies are ongoing in pediatric patients for both the LOPD and IOPD populations.