Amicus Therapeutics

The company's lead product is Galafold, which is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene variant based on in vitro assay data.

In March 2023, Amicus announced that the EC has granted approval for Pombiliti (cipaglucosidase alfa), a long-term enzyme replacement therapy (ERT) used in combination with miglustat for adults with late-onset Pompe disease (LOPD). Miglustat is already approved as Galafold in Europe and the USA to treat Fabry disease, which like Pompe, is a glycogen storage disease.

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