Today, the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) met to discuss Capricor Therapeutics’ Biologics License Application (BLA) for Deramiocel as a potential treatment for Duchenne muscular dystrophy (Duchenne).
Following a full day of presentations, discussion, and public testimony from patients, families and physicians, the committee voted 3 Yes – 9 No on the question of whether the available evidence provides substantial evidence of effectiveness of Deramiocel for the treatment of cardiomyopathy in patients with Duchenne.
While today’s vote is disappointing, the Advisory Committee’s recommendation is non-binding. The FDA will consider the committee’s discussion, the evidence presented, and the totality of the application before making its final regulatory decision by the Prescription Drug User Fee Act (PDUFA) action date of August 22, 2026.
Thank You for Raising Your Voice
Regardless of today’s outcome, one thing was abundantly clear: the Duchenne community showed up.
To every individual living with Duchenne, family member, caregiver, clinician, researcher, advocate, and partner who submitted written comments, spoke during the Open Public Hearing, attended the meeting, or followed along from home—thank you. Your voices reminded everyone in the room that behind every data point is a person, a family, and a community urgently waiting for new therapeutic options.
PPMD Written Comments:
In PPMD’s written comments to the Advisory Committee, we urged members to consider the totality of the evidence alongside the lived experiences of individuals and families living with Duchenne, emphasizing the urgent need for additional therapeutic options that preserve function, support cardiac health, and address the significant unmet needs of this community.
The voices of our community continue to shape how the FDA understands Duchenne—not only as a disease measured by clinical endpoints, but as the everyday experiences of those living with it. That perspective matters, and it was heard.
What Happened During Today’s Meeting?
Throughout the day, the committee heard presentations from both the FDA review team and Capricor before engaging in a robust scientific discussion.
FDA Presentation:
The FDA review team presented its independent assessment of Capricor’s application, outlining the evidence supporting Deramiocel as well as the key questions and uncertainties to be considered by the Advisory Committee. FDA reviewers discussed:
- The Agency’s independent evaluation of the evidence supporting Deramiocel, including a review of the clinical trial design, efficacy data, statistical analyses, and safety findings.
- Discussion of the HOPE-2, HOPE-2 OLE, and HOPE-3 trial results, including the primary and secondary endpoints assessing upper limb function, cardiac outcomes, and other measures of disease progression.
- Review of Deramiocel’s safety profile, including adverse events and the Agency’s assessment of the benefit-risk profile and remaining uncertainties as the Advisory Committee considered whether the evidence supports approval.
- Concerns regarding multiple changes to [the pre-specified statistical analysis plan, interpretation of efficacy results, handling of missing data, and whether the available evidence demonstrates substantial evidence of effectiveness for the proposed indication.
Capricor’s Presentation:
The focus of Capricor’s presentation during the Advisory Committee meeting was to provide additional context to the information provided in the briefing book submitted about Deramiocel to the voting members. Key themes included:
- Clinical efficacy: Capricor highlighted positive results from the Phase 3 HOPE-3 trial and supportive long-term data from HOPE-2 and its extension, noting that data demonstrated meaningful benefits in upper limb and cardiac function based on their final statistical analysis plan.
- Statistical analyses: The company provided details on its statistical approach, stating the primary analyses followed the final Statistical Analysis Plan established prior to unblinding and that the efficacy findings remained consistent across sensitivity analyses.
- Safety: Capricor highlighted Deramiocel’s favorable safety profile, emphasizing that the overall benefit-risk profile supports approval.
- Unmet need: The company emphasized the lack of therapies that address progressive cardiac disease and upper limb function in Duchenne.
Open Public Hearing:
The Open Public Hearing provided an opportunity for members of the Duchenne community to share their lived experiences with Duchenne and perspectives on the impact of Duchenne cardiomyopathy and upper limb function, and the need for additional therapeutic options. Common themes included:
- The urgent need for therapies that preserve cardiac function and slow disease progression.
- The importance of maintaining upper limb function to support independence, daily activities, and quality of life, particularly for individuals who are non-ambulatory.
- Lived experiences illustrating the impact of Duchenne and progressive cardiomyopathy on individuals and families.
- Testimony from individuals and families who have received Deramiocel, describing observed benefits in preserving cardiac and upper limb function.
- The importance of considering patient preferences, lived experience, and the totality of the evidence when evaluating therapies for Duchenne.
- Calls for regulatory flexibility that recognizes the serious, progressive nature of Duchenne and the significant unmet need for additional treatment options.
Voting & Discussion:
Committee members acknowledged the seriousness of Duchenne and the significant unmet medical need facing families, as well as the promising upper limb function data. However, many expressed concerns about changes to the statistical analysis plan, interpretation of efficacy results, missing data considerations, and whether the available evidence demonstrates substantial evidence of effectiveness.
After discussion, the committee voted 3 Yes – 9 No on the question of whether the available evidence provides substantial evidence of effectiveness of Deramiocel for the treatment of cardiomyopathy in patients with Duchenne.
What Happens Next?
Today’s vote is one important step in the FDA’s review process, but it is not the final decision. The FDA is not required to follow the Advisory Committee’s recommendation and will continue its independent review before issuing a decision by the PDUFA action date.
PPMD will continue to monitor the review process closely and will keep the Duchenne community informed as additional information becomes available.
Town Hall Webinar: August 4, 2026 at 1:00 PM ET
Join PPMD, the Best Day Ever Foundation, Kindness Over Muscular Dystrophy, Muscular Dystrophy Association, and the Muscular Dystrophy Family Foundation for a town hall webinar following the July 29, 2026, FDA Advisory Committee meeting for Deramiocel. We’ll review the committee’s discussion and vote, explain what the recommendation means (and what it doesn’t), discuss next steps leading up to the FDA’s August 22, 2026, PDUFA action date, and answer questions from the community. Register here.
A Defining Moment for the Duchenne Community
While today’s recommendation was not the outcome many had hoped for, the meeting represented something much larger than a single vote.
Over the past several weeks, the Duchenne community came together to ensure the lived experience of this disease was part of the regulatory decision-making. Families, individuals living with Duchenne, clinicians, researchers, and advocates shared their perspectives and reinforced the urgent need for meaningful therapeutic options.
That collective effort matters. Progress in Duchenne has never been defined by a single meeting or a single day. Every story shared and every opportunity to educate regulators helps shape future decisions and move the field forward.
Today’s discussion once again demonstrated the strength and unity of the Duchenne community. Thank you for lending your voice, your experience, and your advocacy. We remain committed to working alongside this community as we await the FDA’s final decision and continue pursuing better outcomes for everyone living with Duchenne.
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