Amicus receives positive CHMP opinion for Opfolda for LOPD

Amicus Therapeutics announced that the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion recommending marketing authorization of miglustat, the enzyme stabilizer component of AT-GAA. A decision for miglustat from the European Commission is expected in the third quarter of 2023, after which the two-component therapy will be fully approved, and the Company will begin the country-by-country reimbursement and launch process. Miglustat will be commercialized under the brand name Opfolda. The biologic component of the two-component therapy, Pombiliti, was approved by the European Commission in March 2023. Upon approval, Pombiliti + Opfolda will be the first two-component therapy available in the European Union for the treatment of adults living with late-onset Pompe disease. The indication for Opfolda is an enzyme stabilizer of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset Pompe disease.