Biologics

Split Vote on Sarepta Gene Therapy

FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee 5/12 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.

SUNY Health Sciences University neurology professor Steven Pavlakis voted yes to approve the therapy even though the biomarker micro dystrophin was not “proven to work” in the study, and he said the clinical data were not statistically effective. “The reason I voted yes, though, was I decided that there’s some very good clinicians here that think this really does work,” he said, adding that this is “how we do a lot of clinical science.”

UCLA professor Donald Kohn also voted yes and said most compelling efficacy data are the four-year follow up on the first four patients who had stable scores over the time period. He said allowing accelerated approval while a confirmatory study is being completed in September will provide patients access, and in this case it is best “to err on the side of giving patients the benefit of having access.”

In voting no, Johns Hopkins University professor Caleb Alexander agreed that patient videos of progress on therapy were “compelling,” but he said accelerated approval is more than that. Alexander said this therapy is not an instance where a single-arm, open label clinical study and external controls may suffice. “So, I think the totality of evidence and what we reviewed in the briefing simply doesn't rise to the threshold of substantial evidence that's required for accelerated approval.”

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Gupta Raises Concerns About Overton’s Independence, Ideology

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