Amicus Therapeutics, the Solution to Rare Diseases

Amicus Therapeutics is an American biopharmaceutical company based in New Jersey. It became a public company in 2007 and was funded by a variety venture capital companies including Enterprise Associates, Canaan Partners and Radius Ventures.


Main Focus of Amicus Therapeutics


The company mainly focuses on rare and orphan diseases. They offer medicine and unique technologies in the development of patients suffering from devastating and rare diseases such as the lysosomal storage disorder, Fabry disease, epidermolysis bullosa (EB); a genetic connective skin disorder, and Pompe diseases. The company’s lead upcoming product is Migalastat, a personalized drug that is in late-stage development to treat people with Fabry disease based on person’s genetic diagnosis.


Another candidate in the late stage development is SD-101, a possible first-to- market therapy for epidermolysis bullosa (EB). The company is also trying to advance its biologics and the Chaperone-Advanced Replacement Therapy, a platform advancement to invent Novel Enzyme Replacement Therapy products, for Pompe disease, Fabry diseases, and other various lysosomal storage disorders. The therapeutics frontline program is ATB200/AT2221, a uniquely Pompe disease enzyme replacement therapy, which is administered together with a pharmacological chaperone.


The Developmental Journey of Amicus Therapeutics


Amicus Therapeutics did not have a manufacturing capability and only relied on contract manufacturing alone. Luckily, the company expanded from New Jersey to another site in San Diego in 2008. Amicus then acquired competitor Callidus Biopharma, hence obtaining essential materials and advanced property for the Enzyme Replacement Therapy treatment of pompe disease. As of February 2014, Amicus Therapeutics still had no marketed products. Its most advanced potential product was migalastat (ReleaseFact). Between 2010 and 2013 the company collaborated with JCR Pharmaceutical and GlaxoSmithKline to research on formulation with recombinant alpha-galactosidase. Later in 2015 September, Amicus acquired Scioderm for around 947 million dollars in cash and stock.


Amicus Activities outside America


The company is committed to the Fabry community across the world and has recently seen approval of its migalastat by Health Canada for treatment of Fabry diseases in the country ( Amicus Therapeutics also attended and sponsored the Thirteenth International Congress of inborn metabolism errors, in Rio de Janeiro Brazil, to adopt the research of genetically passed metabolic disorders and related topics. The company’s CEO John Crowley was nominated in the 2017 PharmaVoicelist for his ground-breaking development of rare disease drugs.