Sarepta: FDA finds ‘no significant safety issues of concern’ with DMD gene therapy

By Jane Byrne

- Last updated on GMT

© GettyImages/IvelinRadkov
© GettyImages/IvelinRadkov

Related tags Sarepta Therapeutics Roche Gene therapy DMD

Sarepta Therapeutics would seem to be edging closer to accelerated FDA approval of its gene therapy for Duchenne muscular dystrophy (DMD).

An investigational gene therapy for Duchenne being developed in partnership with Roche, SRP-9001 is designed to treat the proximate cause of Duchenne by delivering to muscle a gene that codes for a shortened, functional form of dystrophin.

Last week, Sarepta reported that the US Food and Drug Administration (FDA) will not be holding an advisory committee meeting to discuss its gene therapy, SRPT-9001. Such panels typically review and evaluate data about the safety, effectiveness, and appropriate use of certain drugs.

Announcing the company’s financial results for the fourth quarter and full-year 2022, Doug Ingram, Sarepta’s CEO, said: “2023 will be a bellwether year for the patient community we serve and for the promise of gene therapy. Sarepta continues to work toward the May 29, 2023, action date for our gene therapy SRP-9001 Biologics License Application, answering questions, preparing for scheduled pre-approval inspections and preparing for launch."

The US agency posed chemistry, manufacturing, and control (CMC) questions, to which Sarepta responded, and the FDA has formally confirmed that there are no significant safety issues of concern, he added.

In November, the FDA accepted Sarepta’s BLA seeking accelerated approval of SRP-9001 for the treatment of ambulant individuals with DMD, and the therapy has been granted priority review by the US regulator. 

Supporting data

In addition to a wealth of pre-clinical evidence, the BLA for SRP-9001 included efficacy and safety data from Study SRP-9001-103, also known as ENDEAVOR, as well as from Studies SRP-9001-101 and SRP-9001-102, and an integrated analysis across these three clinical studies comparing functional results to a propensity-score-weighted external control (EC).

In clinical results from more than 80 treated patients, the company confirmed that SRP-9001 has demonstrated positive results at multiple time points, including one-, two- and up to four-years after treatment, in addition to demonstrating a consistent safety profile.

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