May 1, 2023 / Clinical Trials

PPMD & Community Partners Submit Letter to FDA Encouraging Appropriate Regulatory Flexibility in SRP-9001 Review

PPMD along with community partners and key opinion leaders and physicians in the Duchenne space have submitted a letter to the US Food and Drug Administration (FDA) and FDA’s Center for Biologics Evaluation and Research (CBER) regarding the upcoming regulatory review of SRP-9001 (delandistrogene moxeparvovec), Sarepta Therapeutics’ investigational gene therapy for Duchenne, through the Accelerated Approval (AA) pathway.

This collaborative letter brings attention to the desperate need the Duchenne community has for new and innovative treatments. It calls on the FDA to not delay access to SRP-9001 if it meets the approval standard for accelerated approval.

We also commend the FDA for its commitment to appropriate regulatory flexibility and innovation in confronting the devastation of Duchenne as well as other rare disorders.

Finally, we are urging the FDA to consider the AA criteria in gene therapy content developed by research, clinical, and advocacy leaders set forth in the updated Community-led draft drug development guidance submitted to the Agency in October 2022.

PPMD looks forward to continuing to work with our community partners leading up to the May 12th FDA Advisory Committee meeting for SRP-9001.

View the letter >


If you haven’t already, there is still time to submit a written testimony to the committee to make sure your voice is heard in this process. Click here for additional information on how to submit your testimony ahead of the May 5th deadline.

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