Pombiliti (cipaglucosidase alfa) + Opfolda (miglustat)

About Amicus Therapeutics

Amicus Therapeutics is a pioneer in the development of novel treatments for rare metabolic diseases, particularly lysosomal storage disorders. The company has successfully commercialized Galafold for Fabry disease and is advancing a next-generation pipeline that includes enzyme replacement therapies (ERTs) like Pombiliti + Opfolda for Pompe disease and gene therapies for conditions such as CLN6 Batten disease. Their strategy combines scientific innovation in protein engineering and gene therapy with a global commercial footprint to address significant unmet medical needs.

Therapeutic Areas