Novel Pompe disease combination approved in Europe

27 June 2023

A new Pompe disease therapy has been approved by the European Medicines Agency, with Amicus Therapeutics (Nasdaq: FOLD) picking up authorization for the enzyme stabilizer Opfolda (miglustat).

The marketing nod allows Amicus to offer the medicine, together with the previously-approved Pombiliti (cipaglucosidase alfa), for adults with late-onset Pompe disease.

Pompe disease is an inherited lysosomal disorder caused by deficiency of a certain enzyme acid. The late-onset form affects skeletal muscle and respiratory function.

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