Amicus Therapeutics (FOLD)

Amicus Therapeutics, Inc. is a global patient-centric biotechnology company engaged in the discovery, development and commercialization of a diverse set of novel treatments for patients living with rare metabolic diseases.'The company has one marketed medicine in its portfolio named Galafold which is the first and the only approved oral precision medicine for patients living with Fabry disease, having amenable genetic variants. Migalastat is a potent orally available inhibitor of alpha-Gal A.'The drug was granted an accelerated approval by the FDA for treating adult patients with a confirmed diagnosis of Fabry disease and an amenable genetic variant. Galafold is the first and only oral therapy approved in the EU for the long-term treatment of adolescents with Fabry disease.'The lead pipeline candidate in the company's portfolio is AT-GAA, a differentiated biologic for Pompe disease. The company has one gene-therapy program in its pipeline CLN3, a type of Batten disease, being evaluated in a phase I/II study.