Summary
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.
History
Amicus Therapeutics was founded in 2002 to develop innovative therapies for rare, devastating diseases that lack effective treatments. Over the years, the company has invested heavily in research to successfully bring new treatments to market. The company has taken a rare disease focus and has become one of the largest biotech companies in the world.
Mission
Amicus Therapeutics is dedicated to discovering, developing and delivering novel, first-in-class treatments that improve and prolong the lives of individuals living with rare and devastating conditions.
Vision
Amicus Therapeutics’ vision is to be the leading biotechnology company in the world, developing therapies that significantly improve the lives of people living with rare and devastating diseases.
Key Team
Ms. Ellen S. Rosenberg (Chief Legal Officer & Corp. Sec.)
Mr. David M. Clark (Chief People Officer)
Andrew Faughnan (Sr. Director of Investor Relations)
Ms. Samantha Prout (VP, Global Controller & Principal Accounting Officer)
Dr. Jill Weimer Ph.D. (Chief Science Officer)
Mr. Patrik S. Florencio (Sr. VP, Global Chief Compliance & Risk Officer)
Ms. Diana Moore (Head of Global Corp. Communications)
Recognition and Awards
Amicus Therapeutics has received a wide range of awards for its scientific achievements and its dedication to blazing a path for a world of better treatments for rare diseases, including the Gold Corporate Achievement Award from the National Organization for Rare Disorders, the Biotech of the Year Award from MassBio, the Breakthrough Biotech Award from the Genetic Alliance, and the Rare Impact Award from Global Genes.
References
United States 
