Sanofi’s Avalglucosidase alfa Receives US FDA’s Priority Review as Enzyme Replacement Therapy for Pompe Disease

Shots:

• The US FDA accepted for priority review the BLA of avalglucosidase alfa for long-term enzyme replacement therapy for the treatment of patients with Pompe disease. The target action date for the FDA decision is May 18, 2021
• The BLA is based on positive data from two trials: P-III COMET study and P-II mini-COMET study and the results from both the studies were presented at World Muscle Society and the American Association of Neuromuscular & Electrodiagnostic Medicine and WORLDSymposium respectively
• Avalglucosidase alfa is an investigational enzyme replacement therapy designed to improve the delivery of GAA enzyme to muscle cells, and it would become a potential new SoC for patients with Pompe disease (if approved)

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