April 10, 2023
Dear U.S. Duchenne Community,
Today the FDA announced that an advisory committee meeting related to the biologics license application for SRP-9001 (delandistrogene moxeparvovec), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy, is scheduled for May 12, 2023.
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.
Each advisory committee meeting dedicates time, in this case one hour, to conduct an open public hearing. During this allocated time, or through letters submitted in advance of the advisory committee meeting, representative members of the community including patients, families, advocates, and scientific/clinical experts share their perspective. A number of Duchenne and rare disease advocacy organizations have expressed an interest in collaborating closely together in this effort, as noted in recent blogs by Cure Duchenne and Parent Project Muscular Dystrophy.
If you are interested in helping in, or have questions about, the community’s collaborative effort that is underway in support of the advisory committee meeting, we suggest that you reach out to [email protected] .
We share our enduring gratitude for the individuals and families who have contributed toward the understanding of SRP-9001 through participation in clinical trials. Your continued participation in long term data collection is an incredibly important contribution to the field. Your perspective and voice, combined with other community and scientific voices, may inform this process as well.
The Sarepta Patient Affairs Team