Sarepta Therapeutics today shared an update regarding SRP-9001 (delandistrogene moxeparvovec), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy, for which a Biologics License Application (BLA) is currently under review by the U.S. Food and Drug Administration (FDA).
Sarepta has been informed by the FDA Center for Biologics Evaluation and Research (CBER), Office of Therapeutic Products that an advisory committee meeting will be scheduled prior to the PDUFA (or regulatory action) date of May 29, 2023. The FDA has not yet announced the date or format of the advisory committee meeting.
What is the FDA Advisory Committee?
Part of the FDA’s mission is to evaluate new therapies and determine which are safe and effective for their intended use. This complex job often involves many areas of expertise, and sometimes FDA turns to outside experts for counsel.
Advisory committees weigh the available evidence and provide scientific and medical advice on the safety, effectiveness, and appropriate use of products that FDA regulates. FDA advisory committees are just that, advisory, in nature. Therefore the advice that it receives from the committee does not represent the position of the FDA, rather, the FDA weighs the advice that it receives when taking actions on medical products. FDA makes the final decisions on all matters that come before the committee.
It is Sarepta’s understanding that as one of the first gene therapy BLAs founded on a surrogate endpoint, the advisory committee will primarily relate to the totality of evidence supporting the conclusion that the SRP 9001 dystrophin is reasonably likely to predict clinical benefit, the standard for accelerated approval.
How can the Duchenne community participate?
The FDA has not yet shared the date or format of the advisory committee meeting. Once announced, the public, and the Duchenne community, will be invited and encouraged to participate. PPMD will continue to disseminate information about the meeting and how the Duchenne community can participate as it becomes available.
Read the Community Letter From Sarepta:
March 16, 2023
Dear U.S. Duchenne Community,
Today we are sharing an update regarding SRP-9001 (delandistrogene moxeparvovec), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy, for which a Biologics License Application (BLA) is currently under review by the FDA.
We have been informed by the FDA Center for Biologics Evaluation and Research (CBER), Office of Therapeutic Products that an advisory committee meeting will be scheduled prior to the PDUFA (or regulatory action) date. While this is a change from what was previously communicated, we had begun preparations upon submission of our BLA last fall in anticipation of such a meeting and will continue doing so. We look forward to presenting all the evidence available in support of SRP-9001 at the advisory committee meeting. We also believe that the BLA review will be well-informed from hearing the perspectives of Duchenne experts, clinicians, clinical investigators and the patient community.
An advisory committee meeting is a public forum that brings together independent external experts for the purpose of providing advice and recommendations to FDA on scientific, technical, and policy matters, including on specific products under review to help FDA make sound decisions based on the available science. Here is a helpful resource that describes an advisory committee:
When more is known about the advisory committee meeting details we will communicate as appropriate with the community. We are thankful that CBER moved quickly to schedule the advisory committee meeting in advance of the May 29, 2023 PDUFA date once the decision was made to hold this meeting. We anticipate confirmation of the advisory committee meeting date within the next week. Until then, we invite you to reach out to firstname.lastname@example.org with any questions.
We are continually thankful for the ongoing collaboration offered by people living with Duchenne who participate in clinical studies, their families, health care providers, regulatory agencies, and advocacy leaders- each of you help to inform our decisions and our work. And for members of the Duchenne community who await the outcome of this review and the opportunity for treatment, we are very aware that Duchenne does not wait. Our colleagues across Sarepta continue to work with urgency to advance treatment options for people living with Duchenne and other rare diseases.
The Sarepta Patient Affairs Team