Amicus Therapeutics Inc

Amicus Therapeutics, Inc. is a global biotechnology company focused on discovering, developing and delivering medicines for people living with rare diseases. 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.