May 24, 2023
Dear U.S. Duchenne Community,
Today Sarepta shared an update regarding the ongoing FDA review of the Biologics License Application (BLA) for SRP-9001 (delandistrogene moxeparvovec). As you are aware, we are seeking accelerated approval for SRP-9001 for use in individuals living with Duchenne Muscular Dystrophy who are ambulatory.
A summary of today’s update:
- The FDA has informed us that, subject to their completed review of the BLA and pending final label negotiations, the FDA may potentially grant accelerated approval for SRP-9001 for use in 4-5-year-olds living with Duchenne. The review is expected to be complete by June 22, 2023.
- EMBARK is the proposed confirmatory study. Data that comes from the fully enrolled EMBARK study is very important as it will inform FDA decisions moving forward regarding the potential of a broader label. Sarepta will work as quickly as possible to share data from EMBARK with the Agency as it becomes available.
In the coming weeks, we will be sharing more information on the ENVISION study, which will enroll older ambulatory and non-ambulatory individuals living with Duchenne. We are also advancing work to overcome pre-existing immunity to SRP-9001 and anticipate that those studies will be starting this year.
Our heartfelt appreciation goes to the families who are involved in clinical studies for SRP-9001. Your continued dedication to these studies will lend additional insight into the benefits and risks of this investigational therapy, and your contribution to this study effort will be carried into generations of Duchenne development work to come.
Thank you to every person, advocacy leader, and expert who shared written and spoken testimony that informed the advisory committee meeting for SRP-9001. Your words and calls to action echo in our work and decision making every day.
We continue in our efforts to bring more therapeutic opportunities to the Duchenne community broadly. We know that time is muscle, and time has incredible value for every family who has been impacted by this diagnosis.
The Sarepta Patient Affairs Team