Amicus Therapeutics Gets Positive CHMP Opinion For Pombiliti

(RTTNews) - Amicus Therapeutics (FOLD) Friday 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 or Pombiliti, a long-term enzyme replacement therapy used in combination with miglustat for adults with late-onset Pompe disease. The Company expects a CHMP opinion of miglustat, the enzyme stabilizer component of AT-GAA, in the second quarter of 2023.

A decision from the European Commission is expected in the first quarter of 2023. Cipaglucosidase alfa will be commercialized under the brand name POMBILITI.

Late-onset Pompe disease is a rare, debilitating, and life-threatening lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase.