FDA extends Sarepta Duchenne gene therapy review

May 30, 2023

Last week, Sarepta announced that the FDA will require additional time to complete the review of its Biologics License Application (BLA) for SRP-9001, a gene therapy treatment for Duchenne muscular dystrophy (DMD).

The agency first accepted Sarepta’s BLA back in November, granting it Priority Review and assigning an initial PDUFA date of May 29. The agency is now pushing the timeline back by approximately one month, giving Sarepta a new regulatory action date of June 22.

A few weeks ago, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee narrowly voted to back the drug's approvalDuring the committee’s meeting, members expressed apprehension regarding the treatment’s usage of engineered viruses for delivery, sharing concern over alternative treatments for patients if Sarepta's therapy were to be deemed ineffective in the future.  

Now, the agency has communicated to Sarepta that “it requires modest additional time to complete the review, including final label negotiations and postmarketing commitment discussions.”  

In its recent statement, Sarepta said the FDA may potentially grant accelerated approval for the treatment in a smaller subset of patients — those between the ages of 4 and 5 years old.

SRP-9001 — a driving force behind Roches $1.5 billon acquisition of Sarepta in 2019 — is slated to be manufactured by Catalent should it win approval.