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)

 ­ Ref: Sanofi | Image: Sanofi