NeoGAA filed with FDA for Pompe disease.- Sanofi

The FDA has accepted for priority review the Biologics License Application (BLA) for neoGAA (avalglucosidase alfa), from Sanofi, for long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid alfa-glucosidase deficiency). The target action date for the FDA decision is May 18, 2021. The BLA is based on positive data from two trials: Pivotal Phase III, double-blind, global comparator-controlled trial (COMET), which evaluated the safety and efficacy of avalglucosidase alfa compared to alglucosidase alfa (standard of care) in patients with late-onset Pompe disease. Results from this trial were presented during a Sanofi-hosted virtual scientific session in June 2020 and in October 2020 at World Muscle Society and the American Association of Neuromuscular and Electrodiagnostic Medicine. The Phase II (mini-COMET) trial evaluated the safety and exploratory efficacy of avalglucosidase alfa in patients with infantile-onset Pompe disease previously treated with alglucosidase alfa. Results from this trial were presented at the WORLDSymposium, in February 2020.