Sanofi Gains Priority Review for Investigational Treatment for Pompe Disease
The FDA has granted a Priority Review to Sanofi's biologics license application (BLA) for an experimental enzyme-replacement therapy for patients suffering from Pompe disease, a rare illness that causes muscle deterioration and reduced lung capacity.
The agency's decision to accelerate its review of avalglucosidase alfa was supported by promising data from two clinical trials. In a phase 3 study, the investigational therapy was compared to the standard of care (alglucosidase alfa) in patients with late-onset Pompe disease. A separate phase 2 study assessed the therapy in patients with infantile-onset Pompe disease who were previously treated with alglucosidase alfa.
The FDA has set a target decision date of May 18, 2021 for the BLA. The drug has also received both Breakthrough Therapy and Fast-Track designations.