Based in Cranbury, NJ, Amicus Therapeutics, Inc. is a global patient-centric biotechnology company engaged in discovering, developing, and commercializing a diverse set of novel treatments for patients with rare metabolic diseases. The company has one marketed medicine in its portfolio, Galafold (migalastat), which is the first and the only approved oral precision medicine for patients living with Fabry disease, having amenable genetic variants.

Fabry disease is a genetic disorder caused by various mutations of the enzyme alpha-galactosidase A (alpha-Gal A). Migalastat is a potent, orally available inhibitor of alpha-Gal A. Galafold is currently approved in 40 countries including the United States, European Union (EU), U.K. and Japan. The drug was granted accelerated approval by the FDA in August 2018 and the company launched it in the United States for treating adult patients with a confirmed diagnosis of Fabry disease and an amenable genetic variant. In August 2021, after the label expansion, Galafold became the first and only oral therapy approved in the EU for treating adolescents with Fabry disease.

In September 2023, the FDA approved Pombiliti (cipaglucosidase alfa) + Opfolda (miglustat) 65mg capsules, a two-component therapy for treating late-onset Pompe disease (LOPD) in adults, who weigh over 40 kg and are not improving on their current enzyme replacement therapy.

The commercial launch of the drug is currently underway in various markets, including Europe and the United Kingdom.

In September 2021, Amicus announced that it has entered into a definitive combination agreement with ARYA Sciences Acquisition Corp IV, wherein it will spin off the gene-therapy business to focus on the rare disease business. However, on account of unfavorable marketing conditions and an increasingly challenging environment for standalone gene-therapy companies, both companies announced in February 2022 to terminate the agreement.

Amicus generated total revenues of $399.4 million in 2023, up 20% from 2022.