Sarepta recently announced that the FDA Advisory Committee meeting for SRP-9001 (delandistrogene moxeparvovec), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy, will be held on May 12th. The event will be completely virtual and a live stream will be available.

The Duchenne community is working closely together to prepare for the Advisory Committee meeting for SRP-9001. With the announcement of the meeting, PPMD and other leaders in the Duchenne community, including representatives from the Best Day Ever Foundation, CureDuchenne, Little Hercules, Muscular Dystrophy Association, Muscular Dystrophy Family Foundation, and Team Joseph, met to plan how we can collaborate and work best together to deliver a clear, concise message about the importance of gene therapy and its relevance to treatments for Duchenne.

We need your voice on May 12th. Join us in working together to ensure patient and scientific voices are represented in order to best inform FDA and its Advisory Committee in support of a fair and favorable review of SRP-9001. Whether you plan to submit written testimony or join our organized effort for public testimonies – this community is strongest when we work together and collaborate.

How to Submit Written Testimony

Submitting a written testimony to the committee is a crucial way to advocate and have your voice heard. Written comments are compiled and shared with the FDA Advisory Committee members prior to the meeting, and are a critical part of helping to provide context to the Advisory Committee. We encourage all members of the community – families, physicians, researchers – to submit a written testimony.

Written testimonies must be submitted here on or before May 5th. All submissions received must include the Docket No. FDA-2023-N-1190 for “Cellular, Tissue, and Gene Therapies Advisory Committee; Notice of Meeting; Establishment of a Public Docket; Request for Comments.”

What types of information should be included in written testimony?

The written testimony you submit should reflect your direct experiences with Duchenne and the product under review. The Advisory Committee will be presented with a vast amount of data about the experimental product and is being asked to determine whether enough safety and efficacy data has been made available to warrant access to the public.

By providing your written comments to the Advisory Committee members, you will have an opportunity to make sure that FDA understands the priorities of people with Duchenne and take those into account when reviewing SRP-9001.

PPMD and our partner organizations have compiled a written comment guide which outlines topics you may find helpful to consider when drafting your comments, as well as important links for submitting your testimony.

While all individuals must submit their written testimony directly to the FDA as referenced above, PPMD and our partner organizations are happy to help review your submission following the suggestions of the written comment guide. Please email a draft to lauren@parentprojectmd.org by April 28th if you would like us to review.

FDA Advisory Committee Meetings 101: A Conversation with Tim Franson, MD

What exactly is an FDA Advisory Committee meeting? Following the news of the Advisory Committee meeting planned for SRP-9001 (delandistrogene moxeparvovec), Sarepta’s investigational gene therapy for Duchenne, PPMD hosted an important discussion with FDA expert Tim Franson around what Advisory Committee meetings are all about.

Watch the Meeting

The event will be completely virtual and a live stream will be available for public viewing on May 12th. However, public participation in the meeting will be limited to those selected by FDA to participate in the OPH. Additional details for how to access the live stream are available here.

Questions?

Together, the Duchenne community will continue to work to make sure that this Advisory Committee meeting is as impactful as possible. Please email lauren@parentprojectmd.org if you have any additional questions.

Get Involved >

Together, the Duchenne community will continue to work to make sure that this Advisory Committee meeting is as impactful as possible. Please email lauren@parentprojectmd.org to learn how you can best make your voice heard.