Company Profile

We are passionate about what we do

Amicus Therapeutics, Inc. (Nasdaq:FOLD) is a global biotechnology company at the forefront of advanced therapies to treat a range of devastating rare and orphan diseases. We have a robust development pipeline of treatments for a range of human genetic diseases.

Our lead product candidate, migalastat, is a personalised medicine in late-stage development to treat individuals with Fabry disease on the basis of their genetic diagnosis.

SD-101, a product candidate in late-stage development, is a potential first-to-market therapy for the rare genetic connective tissue disorder Epidermolysis Bullosa (EB).

We are leveraging our biologics and Chaperone-Advanced Replacement Therapy (CHART) platform technologies to develop novel enzyme replacement therapy (ERT) products for Fabry disease, Pompe disease, and other Lysosomal Storage Disorders (LSDs).

Our lead biologics programme is ATB200/AT2221, a uniquely engineered Pompe disease ERT that will be administered in combination with a pharmacological chaperone.