The U.S. Food and Drug Administration (FDA) has scheduled an Advisory Committee meeting for July 29, 2026, to review Capricor Therapeutics’ Biologics License Application (BLA) for Deramiocel, an investigational cell therapy for the treatment of Duchenne muscular dystrophy. The application remains under review with a PDUFA target action date of August 22, 2026.
Advisory Committee meetings are a critical part of the FDA review process. They provide an opportunity for independent experts to evaluate the available data and for the Duchenne community – including patients, families, clinicians, and advocates – to have their voices heard. The meeting will be publicly accessible via live stream, and we strongly encourage community members to follow along and engage.
Deramiocel is supported by results from the Phase 2 HOPE-2 trial, long-term extension data, and the Phase 3 HOPE-3 trial, which reported positive outcomes across key skeletal and cardiac endpoints. As with all potential therapies, careful review of the totality of evidence -including safety and clinical benefit – will be essential.
At PPMD, we believe moments like this require a strong, coordinated, and informed community voice. We appreciate the FDA’s willingness to give the opportunity to the community to share their lived experiences as a part of the review and decision making process.
We have already begun outreach to Duchenne advocacy partners across the community to work together, align efforts, and ensure that patients and families are meaningfully represented throughout this process. Collaboration will be essential as we prepare for the Advisory Committee meeting and the broader FDA review.
If you are interested in getting involved, we invite you to fill out this form, and we will follow up with more information on how to participate. Stay tuned for additional updates, resources, and opportunities to engage in the coming weeks.
As always, PPMD remains committed to ensuring that the perspectives of those living with Duchenne are central to regulatory decision-making and that the community is informed, prepared, and empowered at every step.
We will continue to share updates as more information becomes available.
If you have any immediate questions, please contact Lauren Stanford at Lauren@parentprojectmd.org.
The post FDA Announces Advisory Committee Meeting to Review Capricor Therapeutics’ Biologics License Application for Deramiocel appeared first on Parent Project Muscular Dystrophy.