May 24, 2023 / Clinical Trials

Sarepta Announces PDUFA Extension and Update for the FDA’s Review of SRP-9001





Sarepta Therapeutics announced today that they received an update from the FDA  on the Biologics License Application (BLA) for SRP-9001. The PDUFA date reportedly has been extended to June 22, 2023. 

Sarepta shared that the update from FDA includes: 

  • Subject to the completion of the BLA review, the FDA is working toward potentially granting an accelerated approval for SRP-9001, initially for use in Duchenne patients ages 4-5 years old.
  • In addition to confirming the results of the potential BLA approval for 4-5-year-olds, if the ongoing EMBARK study meets its objectives the Agency may entertain a non-age-restricted expansion of the SRP-9001 label based upon the review of the EMBARK data. EMBARK is fully enrolled, with top-line results expected in the fourth quarter of 2023.
  • The FDA has also informed Sarepta that more time is needed for final label negotiations and postmarketing commitment discussions, and that it anticipates that the review will be complete by June 22, 2023.

While this update is unprecedented in terms of addressing potential accelerated approval, for 4-5-year-olds, we appreciate the transparency and timeliness of this communication from the FDA. 

As our community echoed throughout their testimonies to the FDA Advisory Committee, time is muscle and time is precious in Duchenne

We are hopeful that this update means an approval for patients ages 4-5 by June 22 and an expedient collection and submission of EMBARK data  to provide the agency with additional evidence of SRP-9001’s effectiveness, and that the FDA will exercise regulatory flexibility as they weigh the benefits and risks of SRP-9001 for all patients.

While we are optimistic for a potential approval for 4-5-year-olds, we recognize the heartbreak that many families outside of this criteria feel today. We will continue to advocate for appropriate expanded eligibility. 

PPMD will continue to work alongside our community partners to discuss how we can further support the FDA’s review of SRP-9001, between now and this PDUFA extension date. As a community, we need to continue to rally around this effort. If you have not done so already, please submit your comments to the FDA. 

During this time, PPMD will also continue to ask for transparency and clarity from Sarepta and regulators, while ensuring that patient experiences and preferences are top of mind during the review process. We urge the FDA to use maximal regulatory flexibility to allow incremental innovations that also encourage new research in order to extend such advances. We also encourage the FDA to consider the Accelerated Approval criteria in gene therapy content developed by research, clinical, and advocacy leaders set forth in the Community-led draft drug development guidance submitted to the Agency in October 2022.

Today’s update is progress, a step forward. Our work continues to fight for treatment for every individual living with Duchenne.

Sarepta’s Community Letter: SRP-9001 Update on Regulatory Review

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

View Sarepta’s Community Letter

View Sarepta’s Press Release

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