Sarepta Therapeutics has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for the accelerated approval of SRP-9001, an investigational gene therapy developed in partnership with Roche to treat ambulant patients with Duchenne muscular dystrophy.

The application comes a year earlier than expected, as the company says drug reviewers are open to accelerated review based on data from early-stage trials.

“Every hour of every day, this ruthless disease, Duchenne, robs thousands of children in the United States of muscle as it steals their future from them. Sarepta’s BLA submission for an accelerated approval of SRP-9001 is a significant milestone in our quest to intervene with urgency on behalf of the children we serve.

If approved, SRP-9001 will be the first gene therapy available for Duchenne patients. We are enormously grateful to the courageous families who have participated in the SRP-9001 trials and to the participating clinical investigators and experts who have guided us and played a crucial part in reaching this milestone.”

Doug Ingram, President and Chief Executive Officer, Sarepta Therapeutics

SRP-9001 was granted Fast Track designation in July 2020, an FDA process designed to facilitate the development and expedited review of therapies that treat serious conditions and fill unmet medical needs. In addition to Fast Track, SRP-9001 has also been granted Rare Pediatric Disease (RPD) designation in the United States and Orphan Drug status in the United States, the European Union, Switzerland and Japan.

About SRP-9001 (delandistrogene moxeparvovec

SRP-9001 (delandistrogene moxeparvovec) is an investigational gene transfer therapy intended to deliver SRP-9001 to muscle tissue for the targeted production of functional components of dystrophin. Sarepta is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in the United States upon receiving FDA approval. In December 2019, Roche partnered with Sarepta to combine Roche’s global reach, commercial presence and regulatory expertise with Sarepta’s gene therapy candidate for Duchenne to accelerate access to SRP-9001 for patients outside the United States.