EU Approves Amicus Therapeutics Pombiliti for Late-Onset Pompe Disease

The European Commission has approved Amicus Therapeutics’ Pombiliti (cipaglucosidase alfa) as a long-term enzyme replacement therapy for adult patients with late-onset Pompe disease, a rare neuromuscular disorder that affects skeletal muscle and breathing.

The commission’s decision follows a positive opinion of Pombiliti in December 2022 from the EMA’s Committee for Medicinal Products for Human Use (CHMP), based on positive results from a late-stage study.

Pombiliti must be administered along with miglustat, an enzyme inhibitor. The CHMP is expected to issue a separate opinion on miglustat in the second quarter of this year.