Today Sarepta shared that the FDA has announced that the planned Advisory Committee meeting for SRP-9001 (delandistrogene moxeparvovec), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy, will be held on May 12, 2023. The event will be completely virtual and a live stream will be available.

The Duchenne community is working closely together to prepare for the meeting. PPMD recently met with 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, 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.

We will be hosting an informational webinar on Thursday, April 13th at 7 PM ET. Please contact lauren@parentprojectmd.org for more information on how you can participate and how you can get involved.

Make Your Voice Heard

1) 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. We will update the community with more details regarding written testimony preparation and submission soon.

2) Request to speak during the Open Public Hearing

The Advisory Committee will hear from the community during the Open Public Hearing (OPH) portion of the Advisory Committee Meeting. The OPH is typically just 60 minutes which means there will be very limited speaking opportunities. With the understanding that science and data will drive decisions from the FDA Center for Biologics Evaluation and Research (CBER), Office of Therapeutic Products (OTP) in their review of Sarepta’s gene therapy and all gene and cell therapeutics in the future, our goal is to represent a balance of patient and scientific voices during the OPH that will be most meaningful to the Committee, including:

1. Parent representatives speaking for groups of families who share certain perspectives related to Duchenne and experience with SRP-9001;
2. Physicians speaking on behalf of the families and children under their care and that of their colleagues who have clinical insights; and
3. Scientists who are experts on dystrophin, including the development and utility of shortened forms of this vital protein.

The Duchenne community is working closely together to organize our public testimony in order to avoid the FDA implementing a potential ‘lottery system’ where we will not be able to tell our stories in the most effective and impactful way. We appreciate the submissions from the community we’ve received so far, and encourage additional representatives with scientific and medical expertise in Duchenne to join us in this process as well.

For those interested in collaborating to ensure messages are not duplicated and are most impactful please consider contacting us to speak about the Open Public Hearing

3) 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. We expect additional details for how to access the live stream to be made available by FDA closer to the event.

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.