Amicus Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of small molecule drugs known as pharmacological chaperones. It develops pharmacological chaperones as next-generation medicines for a range of rare and orphan diseases with a focus on improved therapies for lysosomal storage disorders. The company's development programs include small molecules as monotherapy treatments for Fabry and other lysosomal storage diseases, and enzyme replacement therapy (ERT). Its chaperone-advanced replacement therapy programs comprise chaperones co-administered with currently marketed ERTs, as well as proprietary therapeutic enzymes co-formulated with pharmacological chaperones as next-generation ERTs. The company's primary product includes migalastat HCl, a product candidate for Fabry disease that is in Phase III global registration studies for patients with genetic mutations. Its products also comprise AT2220, which has completed Phase II safety used for the treatment of Pompe disease; and AT3375 and afegostat tartrate that are in preclinical studies used for treating Gaucher disease. The company has strategic collaboration with GlaxoSmithKline PLC to develop and commercialize migalastat HCl. Amicus Therapeutics, Inc. was founded in 2002 and is based in Cranbury, New Jersey.
Medicine Biopharmaceutical Rare Diseases Inborn Errors Of Metabolism Lipid Storage Disorders Enzyme Replacement Therapy Orphan Drugs Fabry Disease Gaucher Disease Lysosomal Storage Disorders Pompe Disease Protein Folding Rare And Orphan Diseases