Amicus Therapeutics Inc (Amicus Therapeutics) discovers, develops, and delivers novel oral therapeutics for the treatment of rare genetic diseases. The company specializes in research, development and marketing of small molecule drugs and orally administered monotherapy for the treatment of Fabry disease and other lysosomal storage diseases such as Pompe disease.

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Amicus R&D arm focuses on developing and delivering transformative medicines for rare diseases. The company focuses on research programs that target rare diseases, aiming to be first or best in class and provide meaningful benefits for patients. Amicus Therapeutics has developed treatments for Fabry and Pompe diseases and is expanding its pipeline to include an investigational oral medicine for Focal Segmental Glomerulosclerosis. The company actively involves patients in its research process and collaborates with healthcare providers and patient advocates in clinical trials. The R&D unit of Amicus is responsible for advancing the company's mission to develop transformative medicines for rare diseases. This unit manages the company's research programs, clinical trials, and pipeline development, ensuring that each program has the potential to be first or best in class and provides meaningful benefits for patients. The company own issued U.S. patents that cover the use of migalastat, the active pharmaceutical ingredient in Galafold , in the treatment of Fabry disease, which expire between 2027 and 2042 and are listed in the FDA Orange Book. In FY2025, the company incurred R&D expenses of US$135.8 million, which as a percentage of revenue stood at 20.7%.