Profile

Amicus Therapeutics, Inc., a biotechnology company, focuses on discovering, developing, and delivering medicines for rare diseases. Its commercial product and product candidates include Galafold, an oral precision medicine for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene variant based on in vitro assay data. It also develops AT-GAA, a novel treatment paradigm for Pompe disease; enzyme replacement therapies for Pompe diseases; CLN3, which is in Phase 1/2 clinical study to evaluate the safety and efficacy of a single intrathecal administration of an AAV serotype AT-GTX-502 gene therapy in patients with CLN3; and CDKL5, a gene on the X-chromosome encoding the CDKL5 protein that regulates the expression of essential proteins for normal brain development. The company has collaboration and license agreements with Nationwide Children's Hospital; University of Pennsylvania; and GlaxoSmithKline. Amicus Therapeutics, Inc. was incorporated in 2002 and is headquartered in Philadelphia, Pennsylvania.