FDA advisory meeting on SRP-9001 gene therapy livestreams May 12

Committee will vote on whether to support accelerated approval of SRP-9001

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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An advisory committee of the U.S. Food and Drug Administration (FDA) will be meeting  Friday to discuss SRP-9001 (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular dystrophy (DMD) that’s up for possible approval.

The meeting, which will be livestreamed on YouTube, is scheduled from 9 a.m. to 6 p.m. Eastern time, according to the draft agenda.

SRP-9001’s developer Sarepta Therapeutics applied to the FDA late last year seeking accelerated approval of SRP-9001 to treat DMD patients who can walk.

Under the accelerated approval pathway, the FDA can grant marketing authorization to therapies based on early trial data suggesting the therapy is likely to benefit patients. Developers of therapies given accelerated approval are mandated to conduct additional testing to prove the treatment is effective.

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SRP-9001 designed to deliver gene encoding micro-dystrophin to muscle cells

SRP-9001 is designed to deliver to muscle cells a gene encoding micro-dystrophin, a shortened version of the dystrophin protein, which is a protein that is lacking in DMD patients. Sarepta’s application is specifically based on data suggesting the therapy can increase micro-dystrophin levels as designed.

The application is supported by data from a proof-of-concept Phase 1/2 study (NCT03375164), which showed that four boys with DMD experienced improvements in motor function following one-time treatment with SRP-9001.

The application also includes data from a Phase 2 trial (NCT03769116) that tested SRP-9001 in 41 boys with DMD, as well as from an open-label Phase 1 study called ENDEAVOR (NCT04626674). Although the placebo-controlled Phase 2 study failed to hit its main clinical outcome, data from these trials have generally suggested that boys with DMD who are treated with SRP-9001 show improved motor function compared to what is typically seen in DMD boys not given the gene therapy.

During the advisory meeting, experts will discuss these data in depth. Ahead of the meeting, the FDA has published a full list of discussion topics and a roster of who will be attending, as well as briefing documents from the FDA and from Sarepta.

After discussions, the advisory committee will vote on the following question: “Do the overall considerations of benefit and risk, taking into account the existing uncertainties, support accelerated approval of SRP-9001, using as a surrogate endpoint expression of Sarepta’s micro-dystrophin at Week 12 after administration, for the treatment of ambulatory patients with DMD with a confirmed mutation in the DMD gene?”

Committee members will have the option to vote yes or no, or to abstain from the vote. While the FDA will consider the committee’s vote in its review of SRP-9001, the agency is not obligated to abide by the committee’s decision.

Patients and their families are waiting for the tipping point … to move forward with dramatic and life-changing interventions, such as gene therapy for those with unmet need and who live with relentless decline from Duchenne.

PPMD hosts virtual town hall May 18 to discuss FDA meeting

At 4 p.m. Eastern time May 18, Parent Project Muscular Dystrophy (PPMD) will be hosting a virtual town hall to discuss the committee meeting and next steps for the FDA decision expected by May 29. People can register for this town hall and submit their questions in advance.

PPMD announced last week that it had given the FDA new unpublished data from a study that assessed views from the DMD community about the risk and benefits for a potential treatment. Results generally showed that community members are willing to accept a fairly high amount of risk if a therapy can meaningfully slow disease progression.

“We strongly urge the FDA to take these results into account when reviewing SRP-9001. It is paramount that the agency acknowledges that this community is willing to accept significant risk in exchange for the slowing of disease progression as they evaluate potential therapies for Duchenne,” the organization stated in a press release.

Pat Furlong, founding president and CEO of PPMD, was one of many in the community who submitted written testimony to the FDA committee, writing that SRP-9001 “is reasonably likely to provide benefit in our view.”

“Patients and their families are waiting for the tipping point … to move forward with dramatic and life-changing interventions, such as gene therapy for those with unmet need and who live with relentless decline from Duchenne,” Furlong wrote. “We ask that you thoughtfully weigh and factor in the voice and needs of the patient, and of our families, when making your decision.”