Amicus Therapeutics, Building the future of Therapies for Rare and Orphan Diseases

Founded in 2002, Amicus Therapeutics has dedicated itself to the treatment of rare and orphan diseases, largely specializing in the development of Chaperone-Advanced Replacement therapies(CHART) and Enzyme Replacement Therapies (ERT). This is the process of replacing enzymes that are deficient or absent from the body or using molecules to serve as cell ‘scaffolding’ for proteins to route correctly in the cell. In 2014, Amicus Therapeutics was recognized for having the “Broadest portfolio of small molecule pharmacological chaperones” in the pharmacological industry.

For three years, from 2010 to 2013, Amicus Therapeutics collaborated with GlaxoSmithKline and JCR Pharmaceutical to further research into coformulation and recombinant alpha-galactosidase, which helped lead to advanced therapy treatment for late-stage Fabry disease patients. Migalastat (Galafold), a leading product candidate with Amicus Therapeutics, was approved by the Committee for Medicinal Products for Human Use(CHMP) in 2006 as well as by the European Commission in 2016 ( This drug, for late-stage development, is one of the leading products for Fabry disease.


Along with their avid work into the research of Fabry disease, Amicus Therapeutics also leads ERT therapy for Pompe disease as well as other Lysosomal diseases. By November of 2013, Amicus Therapeutics obtained ownership of materials and intellectual property of ERT treatment for Pompe disease when they acquired Callidus Biopharma, a company known for best-in-class Enzyme Replacement Therapies for lysosomal storage diseases. This acquirement has led Amicus Therapeutics’ leading research for lysosomal storage disease, to flourish.


Originally located in New Jersey, Amicus Therapeutics, while financially on the rise, opened a new research facility in San Diego in 2008 (YahooFinance). By 2010 the company received multiple grants to expand research for studies in collaboration with David Geffen school of medicine at UCLA and into pre-clinical work in collaboration with Icahn School of Medicine at Mount Sinai, through the Alzheimer’s Disease Research Center.