Amicus Therapeutics, Inc. is a biotechnology company focused on the discovery, development and commercialization of therapies for rare and orphan diseases. Founded in 2002 and headquartered in Cranbury, New Jersey, the company applies its proprietary precision medicine platform to advance treatments that address the underlying causes of genetic disorders. Amicus aims to develop medicines that improve or restore the function of proteins affected by inherited metabolic or neurological conditions.
The company’s flagship product, Galafold (migalastat), is an oral pharmacological chaperone approved in multiple regions, including the United States and European Union, for the treatment of Fabry disease in patients with amenable genetic variants. Beyond Fabry disease, Amicus is advancing a pipeline of product candidates targeting other lysosomal storage disorders such as Pompe disease and Niemann-Pick type C, as well as therapies for amyotrophic lateral sclerosis (ALS). Key clinical programs include an investigational enzyme replacement therapy for Pompe disease (AT-GAA) and a small molecule program for ALS, with ongoing studies designed to evaluate safety and efficacy in affected patient populations.
Amicus serves a global patient community through collaborations with healthcare providers and patient advocacy organizations across North America, Europe, Asia and Latin America. The company’s leadership team is headed by John F. Crowley, Chairman and Chief Executive Officer, who has guided the firm’s strategy in rare disease drug development. Under his direction, Amicus continues to expand its research capabilities and foster partnerships aimed at delivering innovative treatments to patients with unmet medical needs.
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