PHILADELPHIA, PA — Amicus Therapeutics (Nasdaq: FOLD) recently announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending marketing authorization of miglustat, the enzyme stabilizer component of AT-GAA. A decision for miglustat from the European Commission (EC) 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 (cipaglucosidase alfa), 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 (EU) for the treatment of adults living with late-onset Pompe disease (LOPD). The indication for Opfolda is an enzyme stabilizer of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid α glucosidase [GAA] deficiency).
“With today’s positive CHMP opinion of Opfolda, we are now one step away from bringing this much-needed new treatment to adults living in Europe with late-onset Pompe disease. This was the realization of the hard work and efforts of so many individuals dedicated to the mission of improving the lives of people living with Pompe disease,” said John F. Crowley, Executive Chairman of Amicus Therapeutics, Inc.
“I am immensely proud of our team that has worked tirelessly over the past decade to develop this innovative therapy. We are grateful for the commitment and support from the Pompe community who have helped advance this therapy, especially the patients, families, and physicians who participated in our clinical studies. Based on the strength of the label and our launch readiness, once fully approved we believe there is significant opportunity to bring Pombiliti and Opfolda as the first two-component therapy for adult LOPD patients in Europe, and to establish this novel treatment combination as a potential new standard of care in Pompe disease,” said Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc.
“This significant milestone moves Pombiliti and Opfolda closer to the LOPD community, where there is a high medical need for novel treatment options,” said Prof. Benedikt Schoser, Professor of Neurology at Ludwig-Maximillians-University of Munich LMU Department of Neurology. “In clinical studies, Pombiliti and Opfolda has exhibited clinically meaningful and positive changes in the key manifestations of this challenging disease. The CHMP positive opinion and recommended indication reflect this robust body of evidence and gives me further hope for the potential of this innovative therapy for people living with LOPD.”
The CHMP based its positive opinion on clinical data from the Phase 3 PROPEL pivotal study, the only randomized, controlled trial in LOPD to include patients in the high unmet need ERT-experienced population, in addition to ERT-naïve patients.