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.

Community Webinar

PPMD, in collaboration with Duchenne advocacy partners Best Day Ever Foundation, CureDuchenne, Jett Foundation, Kindness Over Muscular Dystrophy, Inc., Little Hercules Foundation, Muscular Dystrophy Association, Muscular Dystrophy Family Foundation, and Team Joseph, invites you to join an upcoming webinar on July 7th from 2:00-3:00 PM ET, focused on how our community can engage in the upcoming FDA Advisory Committee (Ad Comm) meeting for Deramiocel.

This Ad Comm represents a critical moment for individuals and families living with Duchenne. The FDA will be evaluating Deramiocel, and the voices of the Duchenne community play a vital role in helping regulators understand the real-world impact of this disease and the urgent need for new treatment options. During this webinar, we will walk through what to expect from the Ad Comm, why it matters, and the different ways you can get involved. This includes opportunities to submit public comments, request to join a speaking group during the Open Public Hearing, and help elevate the patient and caregiver perspective throughout the process.

Whether you are new to advocacy or have participated in past FDA meetings, this session will provide clear, actionable guidance to help you make your voice heard. Join us to learn how you can be part of this important moment for the Duchenne community. Register here.