Amicus Therapeutics, Inc., incorporated on February 4, 2002, is a biotechnology company. The Company is engaged in the discovery, development and commercialization of a set of treatments for patients living with devastating rare and orphan diseases. Its lead product, migalastat HCl is a small molecule that can be used as a monotherapy and in combination with enzyme replacement therapy (ERT) for Fabry disease. Its pipeline also includes SD-101, which is a product candidate in late-stage development, as a potential first-to-market therapy for the chronic, rare connective tissue disorder Epidermolysis Bullosa (EB). It is also leveraging its Chaperone-Advanced Replacement Therapy (CHART) platform technologies to develop ERT products for Pompe disease, Fabry disease, and potentially other lysosomal storage disorders (LSDs). It is also investigating preclinical and discovery programs in other rare and devastating diseases, including cyclin-dependent kinase-like 5 (CDKL5) deficiency.
The Company is in Phase III clinical development of a topical medicine, SD-101, for the treatment of the genetic connective tissue disorder EB. It also initiated a clinical study in patients with Pompe disease, another LSD, to investigate its treatment paradigm that consists of ATB200, an engineered recombinant human acid alpha-glucosidase (rhGAA) enzyme with an optimized carbohydrate structure to enhance uptake, co-administered with a pharmacological chaperone, AT2221, to improve activity and stability. Leveraging its biologics capabilities and platform technologies, it is also investigating preclinical and discovery programs in other rare diseases, including cyclin-dependent kinase-like 5 (CDKL5) deficiency. It has completed two Phase III registration studies of its lead product candidate, migalastat HCl, an orally administered small molecule pharmacological chaperone for the treatment of Fabry disease, an LSD.
The Company's technology platform, Pharmacological Chaperone Technology, comprises Pharmacological Chaperone Monotherapy, CHART Technology Platform and Enzyme Targeting Technology. The Company is leveraging its pharmacological chaperone technology to develop treatments for human genetic diseases by targeting mutated proteins that are unstable, unfolded or misfolded. The Company's personalized medicine approach consists of an oral small molecule pharmacological chaperone monotherapy that is designed to bind to and stabilize a patient's own endogenous target protein. In its CHART programs, each chaperone is designed to bind to and stabilize a specific therapeutic enzyme. The Company is also developing ERTs with higher amounts of mannose 6-phosphate (M6P) for lysosomal targeting.
The Company competes with Sanofi Aventis, Shire plc, Protalix Biotherapeutics, RegeneRx Biopharmaceuticals, Inc., Intercytex Ltd., Amryt, Castle Creek Pharma, Fibrocell, InMed Pharmaceuticals Ltd., Abeona Pharmaceuticals and Audentes.
1 Cedarbrook Dr
The company trades on NASDAQ exchange. The company headquarter is located in New Jersey, NJ. The company was established in the 2002.| Ticker | FOLD |
| Long Name | Amicus Therapeutics, Inc. |
| Short Name | Amicus Therapeutics |