FDA accepts new Pompe disease therapy for priority review

The US Food and Drug Administration has accepted for priority review the Biologics License Application (BLA) for avalglucosidase alfa for long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid α-glucosidase deficiency).

The target action date for the FDA decision is May 18, 2021, says the drug’s developer, Sanofi (Euronext: SAN), which is already a leader in the Pompe disease market with its Myozyme (alglucosidase alfa), which 918 million euros ($1.09 billion). Shares of the French firm were up 1.3% at 85.91 euros by early afternoon.

Avalglucosidase alfa is an investigational enzyme replacement therapy designed to improve the delivery of acid alpha-glucosidase (GAA) enzyme to muscle cells, and if approved, would offer a potential new standard of care for patients with Pompe disease.