July 29, 2022
Dear United States Duchenne Community,
Today, Sarepta announced its intention to submit a Biologic License Application (BLA) for SRP-9001 (delandistrogene moxeparvovec) this fall using the U.S. Food and Drug Administration’s accelerated approval pathway. This letter provides some background on the accelerated approval process and Sarepta’s current assumptions around timing for regulatory submission and review.
FDA has a number of ways to accelerate the availability of treatments for serious diseases. Nearly 30 years ago, the FDA established the Accelerated Approval pathway to expedite the availability of novel treatments that address urgent and unmet medical needs of patients with serious and often life-threatening diseases. Accelerated approval has been used to bring forward treatments for many diseases, including cancer, HIV, and rare diseases such as Duchenne.
As part of its BLA seeking accelerated approval in ambulant individuals with Duchenne, Sarepta plans to provide both biologic and functional data from its studies of SRP-9001. Accelerated approval requires that a post-marketing confirmatory trial is performed to verify the predicted benefit. Importantly, the company is working with clinicians, study participants, and their families to complete the EMBARK study, a phase 3 pivotal trial evaluating SRP-9001 for the treatment of Duchenne, which is anticipated to serve as the confirmatory trial. The company continues to study SRP-9001 in patients with Duchenne who are non-ambulatory and plans to initiate a phase 3 study later this year.
In addition to pursuing the accelerated approval pathway for SRP-9001, the company will also seek priority review. Priority review is a designation given, if certain criteria are met, to a regulatory application with a goal of completing a review of the application within 6 months of file acceptance. It can be used in combination with other FDA expedited programs such as accelerated approval to expedite the availability of treatments for serious diseases.
Should the FDA accept the company’s BLA submission and agree to priority review, we anticipate the FDA will review the application in the first half of 2023.
We are grateful to all of the patients and families participating in clinical studies, as it is your commitment that helps advance science. If you are a member of the U.S. Duchenne community and have any questions about this communication, please contact Patient Affairs at: [email protected].
The Sarepta Patient Affairs Team