Biologics

FDA Extends Review of Sarepta Gene Therapy

FDA has extended by three weeks its review of a Sarepta Therapeutics BLA for SRP-9001 (delandistrogene moxeparvovec), an adeno-associated virus vector-based gene therapy product for treating ambulatory patients with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. The submission is currently under review for accelerated approval. The agency told Sarepta the review extension is needed to complete the review, which has now transitioned to a consideration of a narrower indication for use in Duchenne patients ages 4-5 years old, according to the company. Previously, the submission was under consideration for patients without any age limitations.

Sarepta also says it was told by FDA that the extension will allow it to complete final label negotiations and postmarketing commitment discussions, and that it expects that the review will be complete by 6/22. The previous user fee review action target date was 5/29.

Additionally, Sarepta says that if granted accelerated approval, FDA will consider removing the age limitation if the company’s confirmatory study (EMBARK) meets its objectives to support its expanded use. “EMBARK is fully enrolled, with top-line results expected in the fourth quarter of 2023,” it says.

Earlier this month, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee voted 8 to 6 to recommend approval of a Sarepta BLA seeking accelerated approval for its SRP-9001 (delandistrogene moxeparvovec), an adeno-associated virus vector-based gene therapy product, to treat ambulatory patients with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. A briefing document issued in advance of the meeting said agency medical reviewers found that “the clinical studies conducted to date do not provide unambiguous evidence that SRP-9001 is likely beneficial for ambulatory patients with DMD.”

The document said it is challenging to conclude with reasonable certainty from the data provided by Sarpeta either that SRP-9001 is likely effective for younger patients or that it is likely ineffective for older patients or those with somewhat poorer functional status. It also said FDA reviewers have concerns about the safety of possibly administering an ineffective gene therapy.

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