Sarepta Seeks Accelerated Approval for DMD Gene Therapy Candidate

Sarepta Therapeutics is seeking accelerated approval from the US Food and Drug Administration (FDA) for their experimental Duchenne muscular dystrophy (DMD) treatment SRP-9001.

SRP-9001 is a gene therapy candidate for the treatment of boys with DMD who can still walk. It aims to introduce the micro-dystrophin gene into the muscle tissue of patients using an adeno-associated virus (AAV).

“Duchenne robs children daily and hourly of their muscle, stealing them bit by bit from their families and loved ones,” said Doug Ingram, president and chief executive officer of Sarepta Therapeutics, in a press release. “Guided by rigorous science and productive regulatory discussions, our goal is to move with the urgency desperately needed by the patient community, and our upcoming [Biologics License Application] filing for SRP-9001 serves that goal.”

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DMD is caused by a mutation in the gene that encodes the dystrophin protein, which is essential for the health of the muscle tissue. In its absence, muscles get damaged at each contraction and degenerate over time, being replaced by scar tissue. This means patients gradually lose their mobility and ability to walk. In the later stages of the disease, they also require ventilation to be able to breathe as the diaphragm gets affected.

Read more about gene therapy for DMD.

Gene therapy for DMD is being researched extensively. The main issue is the large size of the DMD gene.

SRP-9001 contains a smaller gene that can still code for a functional protein. It is being tested in a number of clinical trials in patients with DMD. The FDA has already granted it Fast Track and Rare Pediatric Disease designations as well as Orphan Drug status.

A Biologics License Application is a request for permission to introduce a biologic product into interstate commerce. Upon approval, Sarepta intends to commercialize SRP-9001 in the US.