Amicus Therapeutics Announces Approval and Launch of New Pompe Disease Therapy in the European Union

Category: News
June 28, 2023

PHILADELPHIA, June 27, 2023 (GLOBE NEWSWIRE) — Amicus Therapeutics, a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, today announced that the European Commission (EC) has granted approval for Opfolda® (miglustat) 65mg capsules, an enzyme stabilizer of cipaglucosidase alfa, a long-term enzyme replacement therapy for adults with late-onset Pompe disease. Pombiliti® (cipaglucosidase alfa) was previously approved by the EC in March 2023. Pompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid α-glucosidase (GAA). Amicus plans to immediately launch Pombiliti + Opfolda in Germany and is commencing reimbursement processes with healthcare authorities in other European countries.

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