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# Sarepta Submits BLA for Duchenne Gene Therapy
- URL: https://www.fdaweb.com/sarepta-submits-bla-for-duchenne-gene-therapy/
- Published: 2022-09-29T12:00:00.000Z
- Updated: 2026-09-14T18:01:15.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5152855

Sarepta Therapeutics has submitted a BLA seeking accelerated approval of SRP-9001 (delandistrogene moxeparvovec) to treat ambulant patients with Duchenne muscular dystrophy. SRP-9001 is a gene therapy being developed in partnership with Roche.

The submission relies on the expression of SRP-9001 dystrophin protein, “an internally shortened and functional version of dystrophin, as a surrogate endpoint reasonably likely to predict clinical benefit,” according to the [company](https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-submits-biologics-license-application-srp?%5Fga=2.84892816.889259431.1664467844-1224349600.1662471987&ref=fdaweb.com). “In clinical trials, SRP-9001 demonstrated positive results at multiple time points, including one-, two- and four-years after treatment, in addition to a consistent safety profile.” Sarepta also says it has proposed its fully-enrolled study EMBARK as its confirmatory study to support the accelerated approval.