Located in New Jersey, Amicus Therapeutics is a biopharmaceutical company that focuses on rare diseases relating to lysosomal storage disorders. Functioning without manufacturing facilities, the company relies on manufacturing contracts to produce their products. Amicus Therapeutics has developed working relationships with the David Geffen School of Medicine located at UCLA and the Icahn School of Medicine at Mount Sinai which resulted in financial backing from national nonprofit organizations like the Alzheimer’s Drug Discovery Foundation. In the last 10 years, Amicus has grown as a company by acquiring competitors Callidus Biopharma and Scioderm on top of establishing another research facility based in San Diego, California.
Leading Amicus Therapeutics is John F. Crowley who served as a chief executive officer since 2005 and has also held the position of chairman of the board since 2010. Having two children who were diagnosed with Pompe disease which led him to leave his job and become an entrepreneur who would later establish Novazyne Pharmaceuticals. Inspired by his success and his efforts, the Wall Street Journal featured him on their front page and his family’s story was adapted into a feature film starring Brendan Fraser along with Harrison Ford. His experience and pharmaceutical success eventually led John Crowley to Amicus Therapeutics which has experienced tremendous success through his leadership.
The company’s leading product is a late-stage treatment of Fabry disease. Other products like SD-101 work to treat Epidermolysis Bullosa which is a rare connective tissue disorder (GoogleFinance). Amicus Therapeutics works diligently throughout the year to bolster their Chaperone-Advanced Replacement Therapy, commonly referred to as CHART, which aims to utilize enzyme replacement therapy for numerous rare diseases like Fabry disease. Being a publicly traded NASDAQ stock demands that the company continue to innovate and improve the quality of their pharmaceutical products.