CHMP recommends cipaglucosidase alfa + miglustat to treat late stage Pompe disease.- Amicus
Amicus Therapeutics 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 cipaglucosidase alfa, a long-term enzyme replacement therapy (ERT) used in combination with miglustat,for adults with late-onset Pompe disease (LOPD).
A decision from the European Commission (EC) 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. Late-onset Pompe disease is a rare, debilitating, and life-threatening lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA lead to the accumulation of the substrate glycogen in the lysosomes of muscles and other tissues. Disease severity ranges on a spectrum, but predominant manifestations are skeletal muscle weakness and progressive respiratory involvement.
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