Sarepta Plans on Submitting BLA for Duchenne Gene Therapy

Sarepta Therapeutics said that it plans to submit a Biologics License Application (BLA) for accelerated approval of its investigational gene therapy candidate SRP-9001 (delandistrogene moxeparvovec) for treatment of Duchenne muscular dystrophy.

The gene therapy candidate for the rare genetic neuromuscular disease is being developed as part of a collaboration and licensing deal inked with Roche in December 2019.

The agency has granted the investigational therapy Fast Track designation in July 2020, which facilitates the development and expedites the review of drugs to treat serious conditions and fill an unmet medical need.

SRP-9001 has also received the FDA’s Rare Pediatric Disease and Orphan Drug designations. The review is expected to begin sometime this year and continue into the first half of 2023.