In September, Sarepta Therapeutics filed a BLA (Biologics License Application) for the approval of SRP-9001 (delandistrogene moxeparvovec), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy, using the FDA’s accelerated approval pathway.

During the marketing application’s review process, FDA shared that it would hold an Advisory Committee Meeting, a meeting which allows the FDA to include a variety of perspectives for consideration when making the final marketing decision for a therapy under investigation, prior to deciding on the company’s BLA by its regulatory action date of May 29, 2023.

While the date for this meeting has not yet been announced, the Duchenne community is in a pivotal moment, similar to our work in 2016 when the first exon-skipping drug was under review and FDA scheduled an Advisory Committee Meeting. We have another opportunity to make a lasting impact.

Last night, PPMD met with leaders in the Duchenne community to plan how we can collaborate and work best together to get the most effective message in front of the FDA. The meeting included representatives from the Best Day Ever Foundation, CureDuchenne, Little Hercules, Muscular Dystrophy Association, Muscular Dystrophy Family Foundation, and Team Joseph.

Focusing Our Voices

Within the next few weeks, we will have another opportunity to show up and to raise our collective voices. With this meeting, there are some differences that are important to consider, reflecting the need for our community to coordinate a unified effort to ensure the patient voice and scientific voice are equally balanced to best inform FDA and its Advisory Committee.

1. Sarepta’s gene therapy product is under the review of a new office, CBER’s Office of Therapeutic Products (OTP).
   Previously approved Duchenne therapies were reviewed by the FDA Center for Drug Evaluation and Research (CDER) because they were not gene or cell therapies. Gene and cell therapies, including Sarepta’s gene therapy product, are reviewed by the FDA Center for Biologics Evaluation and Research (CBER), and specifically CBER’s new Office of Therapeutic Products (OTP). The Advisory Committee Meeting for SRP-9001 will be led by members of OTP. The CBER Advisory Committee for OTP is entirely different from the Advisory Committee which reviewed the prior Duchenne applications in CDER.
2. OTP is focused on science and data.
   Science and data will drive OTP’s decision to approve Sarepta’s gene therapy and all gene and cell therapeutics in the future. We understand much of the discussion may center on the evidence supporting that the proposed surrogate endpoint of SRP-9001 dystrophin is reasonably likely to predict clinical benefit, which is the standard for accelerated approval.
3. The patient voice is critical to give meaning to the science and data.
   Under accelerated approval, FDA also accounts for severity, rarity, and unmet need and weighs factors like the community’s willingness to accept risk and uncertainty in their benefit-risk decision-making.

Our Action Plan

For these reasons, we are hopeful this community will join us in working together. PPMD is taking the lead on:

1. Submitting unified messaging from Duchenne leaders to FDA
   PPMD will deliver a letter to FDA supporting a fair and favorable review of SRP-9001, signed by Duchenne community leaders and other Key Opinion Leaders.
2. Encouraging patient and scientific voices to be equally balanced
   We have sent a letter to FDA requesting their consideration to add individuals with scientific and medical expertise in Duchenne to their committee. We already understand they will be adding people with Duchenne as well.
3. Preparing community members for testimony delivery
   We will schedule Zoom meetings for anyone interested in contributing to the Advisory Committee Meeting, including through written and/or video testimony. Those who have reached out about participating will receive more information about this in the coming days. If you are interested in participating, please email Lauren Stanford at lauren@parentprojectmd.org.
4. Planning an organized effort for public testimonies
   We will attempt to avoid having so many people register independently for live testimony during the Advisory Committee Open Public Hearing that the FDA would be forced to hold a ‘lottery system’, which may lead to the Advisory Committee not hearing from clinicians and those with direct impact of SRP-9001.

We Need Your Voice

The Advisory Committee will hear from the Duchenne 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. It is paramount that we avoid the OPH going to a ‘lottery system’ where we will not be able to tell our stories in the most effective and impactful way.

Our goal is to represent a balance of patient and scientific voices during the meeting, including:

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

Together, we must work to deliver a clear, concise message about the importance of gene therapy and its relevance to treatments for Duchenne. This is about all of us, a community committed.

Get Involved >

If you are interested in participating, please email Lauren Stanford at lauren@parentprojectmd.org.