Amicus Therapeutics, Inc.
Amicus Therapeutics, Inc. is a biotechnology company that is focused on discovering, developing, and delivering medicines for rare diseases. The Company's portfolio is Galafold (migalastat), which is the approved oral precision medicine for people living with Fabry disease. Galafold is designed to bind to and stabilize an endogenous alpha-galactosidase A (alpha-Gal A) enzyme in patients with genetic variants. Migalastat is approved under the trade name Galafold in the United States, European Union, United Kingdom and Japan. Its biologics program in its pipeline is Amicus Therapeutics GAA (AT-GAA), which is a clinical-stage treatment paradigm for Pompe disease. Pompe disease is a lysosomal disorder (LD) from deficiency in an enzyme, GAA. AT-GAA consists of a uniquely engineered rhGAA enzyme, ATB200, or cipaglucosidase alfa, with an optimized carbohydrate structure to enhance lysosomal uptake, administered in combination with AT2221 (miglustat) that functions as an enzyme stabilizer.
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