On May 12, the FDA held an Advisory Committee meeting for SRP-9001, a gene therapy intended to treat Duchenne. The Advisory Committee meeting was used by FDA to obtain independent expert advice on scientific, technical, and policy matters.

The Duchenne community played a very active role, both in providing written comments on SRP-9001 and in testimony during the Open Public Hearing portion of the meeting.

With a favorable vote from the Advisory Committee, our Duchenne community is optimistic and ready to support the continuing progress on SRP-9001’s ongoing review through the PDUFA regulatory review date of May 29.

What’s Next?

The Duchenne community is encouraged to provide additional written comments to the FDA following the Advisory Committee discussion. This is an opportunity to reinforce any earlier comments and reflect on the topics that were covered in the Advisory Committee meeting as the FDA is making its decision. Comments should be submitted as soon as possible so they can be considered by FDA prior to the SRP-9001 expected action date of May 29, 2023.

Written comments should be submitted by email to ocod@fda.hhs.gov. Please put “DMD Gene Therapy” in the subject line of your email so it can be easily identified by FDA staff for this purpose. We recommend these emails be as brief as possible. 

Suggested Topics 

• Your name, when you/your family member was diagnosed with Duchenne, and if your family member participated in the SRP-9001 clinical trials. 
• You/your child’s current stage of progression of symptoms and the impact it is having on the individual and your family’s life. 
• How you view the importance of a gene therapy that offers the chance of benefits compared with the risks outlined in the Advisory Committee meeting. 
• What a delay in accessing a new therapy, including for a few months, means in terms of preserving muscle function and quality of life (time is muscle). 

Topics to Avoid 

• Other unapproved therapies – please keep the focus on the therapy being reviewed. 
• Cost and coverage of therapy – kindly avoid focusing on the cost or health plan coverage of the therapy. Although this is an important concern for many people, the FDA is not responsible for setting therapy costs or coverage. 
• Criticism of FDA – these sessions are expected to be respectful and professionally appropriate. 

Thank you again for your active participation in Duchenne advocacy. PPMD will continue to work alongside our community partners as we hope for a positive outcome on May 29.

For additional information, please contact PPMD’s Lauren Stanford at lauren@parentprojectmd.org.

WATCH: Duchenne Community Town Hall

Duchenne community partners held a Virtual Town Hall on Thursday, May 18 to debrief on the Advisory Committee meeting and provide next steps leading up to the PDUFA date. If you missed it, the recording is available.