Amicus receives positive CHMP opinion for Pombiliti 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 cipaglucosidase alfa, a long-term enzyme replacement therapy used in combination with miglustat for adults with late-onset Pompe disease. A decision from the European Commission on cipaglucosidase alfa, the enzyme replacement therapy component of AT-GAA, is expected in the first quarter of 2023. Cipaglucosidase alfa will be commercialized under the brand name POMBILITI. The Company expects a CHMP opinion of miglustat, the enzyme stabilizer component of AT-GAA, in the second quarter of 2023. The CHMP based its positive opinion on clinical data from the Phase 3 pivotal study, the only randomized, controlled trial in LOPD to include patients in the high unmet need ERT-experienced population, in addition to ERT-naive patients. As anticipated and consistent with a recent opinion of another ERT in this disease space, the CHMP also determined cipaglucosidase alfa does not qualify as a New Active Substance.