March 22, 2023 / Clinical Trials

How You Can Be Involved in the First FDA Ad Comm Meeting for a Gene Therapy Product for Duchenne

Last week, Sarepta announced that the FDA Center for Biologics Evaluation and Research (CBER), Office of Therapeutic Products had informed the company that an advisory committee meeting will be scheduled prior to the PDUFA (or regulatory action) date of May 29, 2023 for SRP-9001 (delandistrogene moxeparvovec), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy.

While FDA has not yet shared the date of the advisory committee meeting, we expect the date to soon be posted on the Federal Registrar.

You will recall that we celebrated on November 30, 2022 when Sarepta announced that FDA had accepted their Biologics License Application (BLA) and granted priority review for SRP-9001. Sarepta’s BLA submission for an accelerated approval, along with the FDA’s acceptance of that BLA for filing and review, is a tremendously important milestone in our Duchenne community.

Those of us who were involved in our first FDA advisory committee meetings in Washington, DC years ago, remember the anticipation, the energy, the hope that permeated our community. Our community gathered and with one voice, agreed that expressing a truncated dystrophin with exon skipping technology was reasonably likely to predict benefit. Our voice was heard, and we celebrated that first approval. The Center for Drug Evaluation and Research (CDER) led that meeting.

Now we stand at the forefront of another step forward for Duchenne and this time with CBER. Gene Therapy, delivering a micro-dystrophin, which we agree, is again reasonably likely to predict benefit, again utilizing the accelerated approval pathway.

As a community, we have anticipated and supported the development of micro-dystrophin which has been years in development. We have watched our families participate in trials, conducted preference studies to understand benefit and risk, met with CBER to talk about what we are seeing post-gene therapy and to share our enthusiasm around micro-dystrophy.

Our community will come together once again, collectively with one voice, highlighting the science, the clinical changes and the stories, our stories, and the firm belief that this is a new day, an incredible step forward to change the trajectory of Duchenne.

Get Involved

While we await the date of the meeting, PPMD will continue to work closely with our Duchenne community partners, as well as our partners at Sarepta, to prepare for this critical event. Please contact if you are willing to join forces and are interested in helping us with this effort.

We can do this. We will do this. We will celebrate together the next major step forward for Duchenne.

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