EMA accepts Sanofi's submission for avalglucosidase alfa in Pompe disease

The European Medicines Agency (EMA) has accepted for review the Marketing Authorization Application (MAA) for avalglucosidase alfa, for long-term enzyme replacement therapy for the treatment of patients with Pompe disease.

Sanofi’s (Euronext: SAN) MAA is based on positive sets of data, one a Phase III study and the other a Phase II trial.

Avalglucosidase alfa is designed to improve the delivery of the lysosomal enzyme acid alpha-glucosidase into the lysosomes of muscle cells to breakdown glycogen and help address respiratory impairment, as well as decreased muscle strength and function, which are critical manifestations of Pompe disease. If approved, the drug would offer a potential new standard of care for patients with Pompe disease