Amicus Therapeutics, Inc. (NASDAQ: FOLD) is a biotechnology company dedicated to developing and delivering therapies for rare and orphan diseases. Founded in 2002 and headquartered in Philadelphia, Pennsylvania, Amicus has established itself as a leader in precision medicine with a focus on enzyme enhancement, chaperone therapies and gene therapies. The company’s mission centers on addressing conditions that are often underserved by traditional pharmaceutical approaches, leveraging advanced science to improve patient outcomes across multiple genetic disorders.
The company’s flagship product, GALAFOLD® (migalastat), is an oral pharmacological chaperone approved for the treatment of Fabry disease in eligible patients in North America, Europe and other international markets. Beyond Fabry disease, Amicus maintains a robust pipeline that includes product candidates for Pompe disease, Tay-Sachs disease, Gaucher disease and Transthyretin Amyloidosis (ATTR). These programs utilize a range of modalities such as enzyme replacement therapies (ERT), next-generation ERTs, substrate reduction platforms and gene therapy approaches, reflecting the company’s commitment to innovation in rare disease treatment.
Amicus operates research and development facilities in the United States and Europe, enabling collaboration with academic institutions, patient advocacy groups and industry partners. The company’s strategic alliances include partnerships with established pharmaceutical firms and research organizations to accelerate clinical development and expand global reach. These collaborations have been critical in advancing both small molecule and gene therapy candidates through key regulatory milestones.
Leadership at Amicus is helmed by a team of seasoned executives with extensive experience in biotechnology, regulatory affairs and commercial strategy. John F. Crowley has served as Chairman and CEO since 2013, guiding the company through pivotal product launches and pipeline expansions. Under this leadership, Amicus has grown from a research-stage enterprise into a commercial biotech company, sustaining its commitment to scientific excellence and patients facing rare genetic disorders worldwide.
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