The FDA has shared live stream access information for the May 12th Advisory Committee for SRP-9001 (delandistrogene moxeparvovec), Sarepta Therapeutics’s investigational gene therapy, which has applied for FDA approval through the Accelerated Approval pathway with the requested indication for the treatment of ambulatory patients with Duchenne.
While community participation in the Advisory Committee meeting will be limited to those who have already been selected by FDA to present during the Open Public Hearing (OPH) portion, we encourage all members of the community to watch along with us for this historic meeting, the first ever FDA Advisory Committee meeting for a gene therapy specifically to treat Duchenne.
PPMD will share an update following the conclusion of the meeting discussing what we’ve learned. We also invite you to join our Virtual Town Hall, hosted alongside our community partners, on Thursday, May 18th, at 4 PM ET where we will debrief and provide next steps leading up to the SRP-9001 PDUFA date on May 29th.
Friday, May 12th: Live Stream Information
The live stream will be available here, planned to run from 9 AM – 6 PM ET. View the agenda provided by FDA. The meeting presentations will be heard, viewed, captioned, and recorded.
Please note that while this meeting will include presentations and discussions followed by a vote on that discussion, the result of the votes during the meeting should be viewed as Committee advice to Center for Biologics Evaluation and Research (CBER) and do not reflect the final decision of the FDA. Discussions from the Advisory Committee meeting will be used to help further inform regulators before a decision is made, on or prior to SRP-9001’s PDUFA date of May 29th.Join the live stream >
Meeting materials including briefing documents have also been released. PPMD will review all of the materials the FDA has released ahead of the Advisory Committee meeting and will continue working with our Duchenne community partners to make sure that the meeting is as impactful as possible.
What Happens During an Advisory Committee Meeting?
PPMD recently hosted a discussion with FDA expert Tim Franson around what Advisory Committee meetings are all about. Watch the video below to familiarize yourself with the basics of these meetings and their role in the regulatory review process.
Virtual Town Hall
PPMD, along with our other community partners, will host a Virtual Town Hall on Thursday, May 18th, at 4 PM ET where we will debrief on the FDA Advisory Committee meeting and provide next steps leading up to the SRP-9001 PDUFA date on May 29th. You can register to participate here and submit questions in advance.