Sarepta submits BLA for muscular dystrophy gene therapy

Sept. 29, 2022

Massachusetts-based biotech Sarepta Therapeutics announced this week that it had completed the submission of its Biologics License Application (BLA) to the FDA for the approval of its investigational gene therapy to treat Duchenne muscular dystrophy in ambulant patients.

The drug, called SRP-9001, was developed in partnership with Roche. It works by helping to catalyze the targeted production of functional components of dystrophin, a protein that aids in muscle strengthening and injury prevention. In patients with Duchenne, the mutations in the DMD alter the structure of the protein, inhibiting it from working properly and thus causing multi-system musculoskeletal symptoms. The disease is rare, but fatal, and affects approximately 1 in 3,500-5,000 newborn boys every year. 

In its BLA, Sarepta argues that the fact that the drug provides the expression of a functional version of the affected protein enables them to reasonably predict clinical success. Additionally, the company submitted efficacy and safety studies from three sets of trials (called ENDEAVOR), as well as an analysis across all studies comparing functional results. Lastly, Sarepta proposed that its ongoing EMBARK study would help with post-marketing confirmatory data to support the accelerated approval. 

According to Sarepta’s president and chief executive officer, if approved, the drug would be the first gene therapy available for patients with the condition. The FDA has also granted SRP-9001 Fast Track designation, as well as Rare Pediatric Disease designation and Orphan Drug status.