FDA Reverses Course; Will Hold Adcomm for Sarepta’s DMD Gene Therapy

Pictured: A Sarepta building with logo/courtesy of Sarepta

Pictured: A Sarepta building with logo/courtesy of Sarepta

In a reversal of course, the FDA will hold an advisory committee meeting for Sarepta’s investigational gene therapy for Duchenne muscular dystrophy (DMD) ahead of its May 29 action date.

Doug Ingram, president and CEO, Sarepta, previously said the regulator had found no “significant safety issues of concern” with SRP-9001 (delandistrogene moxeparvovec) and did not plan to convene an advisory committee meeting.

There is currently no set date for the adcomm. In reaction to the news, Sarepta’s shares were down around 20% in after-hours trading Thursday.

In an investor call Thursday, Ingram said the candidate’s safety is not a “significant issue” and that the primary focus of the meeting “will be the design of the 9001 construct and the evidence supporting the conclusion that 9001 dystrophin is reasonably likely to predict clinical benefit.”

The adcomm was also not spurred by new data or evidence regarding delandistrogene moxeparvovec, Ingram said. Sarepta does not expect it will need to file a major amendment to the candidate’s Biologics License Application (BLA), nor does it anticipate delays in the target action date.

Sarepta does not see the adcomm meeting as a setback, Ingram said, as the company had always assumed that a panel would be convened to discuss delandistrogene moxeparvovec and had prepared accordingly.

DMD is a rare neuromuscular disorder arising from mutations in the dystrophin gene, which manifest as developmental delays and muscle weakness. Delandistrogene moxeparvovec is an investigational gene therapy designed to address DMD by delivering a functional copy of the dystrophin gene to the muscle tissues.

The FDA accepted Sarepta’s BLA for delandistrogene moxeparvovec in November 2022 and granted the submission priority review.

Data from three studies formed the BLA: SRP-9001-101, SRP-9001-102 and SRP-9001-103. With a total of over 80 patients enrolled, these studies demonstrated the therapy’s efficacy through different measures and across various time points.

Early results from the studies showed Sarepta’s candidate could increase dystrophin expression, though its effect on functional endpoints were sometimes mixed. Later results, however, showed promising functional outcomes from delandistrogene moxeparvovec.

In a late-cycle meeting, the FDA told Sarepta it did not identify significant safety issues with the gene therapy and “does not anticipate the need for a risk management program,” Ingram said during the call.

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