Amicus announces approval, launch of new Pompe disease therapy in EU

Amicus Therapeutics announced that the European Commission, EC, has granted approval for Opfolda 65mg capsules, an enzyme stabilizer of cipaglucosidase alfa, a long-term enzyme replacement therapy for adults with late-onset Pompe disease. Pombiliti was previously approved by the EC in March 2023. Amicus plans to immediately launch Pombiliti + Opfolda in Germany and is commencing reimbursement processes with healthcare authorities in other European countries. Pombiliti + Opfolda is a unique two-component therapy. Pombiliti, is a bis-M6P-enriched rhGAA enzyme, designed for increased uptake into muscle cells. The EC approval was based on clinical data from the Phase 3 pivotal study, the only trial in LOPD to study the real-world population of both ERT-naive and ERT-experienced participants in a controlled setting. In the U.K., the Medicines and Healthcare products Regulatory Agency regulatory approval is expected in the third quarter of 2023. The U.S. Food and Drug Administration’s review is ongoing, and the Company expects approval in the third quarter of 2023. The FDA previously granted Breakthrough Therapy designation for Pombiliti + Opfolda.