A new Biologics License Application (BLA) has been submitted to the US Food and Drug Administration (FDA) for the accelerated approval of delandistrogene moxeparvovec (SRP-9001), a novel gene therapy for the treatment of Duchenne muscular dystrophy (DMD).

If approved, the investigational gene therapy, being developed by Sarepta Therapeutics in partnership with Roche, would be used to treat ambulant patients with DMD.

“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,” said Doug Ingram, president and chief executive officer of Sarepta Therapeutics. “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.”

Continue Reading

Read more about experimental therapies for DMD

The BLA was submitted with positive preclinical, biomarker, and clinical trial results. Safety and efficacy data from 3 clinical trials—SRP-9001-101, SRP-9001-102, and SRP-9001-103—and an integrated analysis across all 3 trials compared functional results to a propensity-score-matched external control.

Sarepta also proposed their fully enrolled global, randomized, double-blind, placebo-controlled clinical trial, SRP-9001-301, as a postmarketing confirmatory study.

SRP-9001 is a potential gene therapy designed to produce an internally shortened and functional version of dystrophin, the SRP-9001 dystrophin protein, in targeted muscle tissue. It was granted Fast Track designation in July 2020 by the FDA as well as Rare Pediatric Disease designation by the FDA and Orphan Drug status in the US, the European Union, Switzerland, and Japan.

Sarepta has partnered with Roche due to its global reach, commercial presence, and regulatory expertise to help accelerate the approval of SRP-9001 outside of the US.


Sarepta Therapeutics submits Biologics License Application for SRP-9001 for the treatment of ambulant patients with Duchenne muscular dystrophy. News release. Sarepta Therapeutics; September 29, 2022.