On July 9th, the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) for Capricor’s Biologics License Application (BLA) for Deramiocel (CAP-1002), an investigational cell therapy for Duchenne cardiomyopathy. This decision means that the current application will not be approved in its present form, but it also presents a path forward by identifying areas where additional efficacy data and clarification in the Chemistry, Manufacturing, and Controls (CMC) section are needed.
Capricor plans to submit data from the Phase 3 HOPE-3 clinical trial, a randomized, double-blind, placebo-controlled clinical trial of 104 patients, with topline results expected in the third quarter of 2025. Deramiocel holds the potential to address the leading cause of death in Duchenne—cardiomyopathy—and may offer meaningful benefit across age, variant, and stage of progression. We are hopeful that data from HOPE-3 will provide the clarity and strength needed to support approval.
Capricor has indicated that it will engage further with the FDA on the path forward, and the agency has confirmed that it will restart the review clock upon resubmission. Every step in the regulatory process—whether approval or requests for more information—underscores the importance of strong evidence of safety and effectiveness. While a CRL is not the outcome many hoped for at this stage, it reflects the agency’s commitment to a thorough review and moves us closer to the therapies families urgently need.
At PPMD, we remain committed to supporting and working with the Duchenne community as the next regulatory steps are considered. Our priority is ensuring that any additional data reflect patient experiences and input from both the FDA and the broader community.
We appreciate the agency’s engagement and recognition of the serious needs of people living with Duchenne. The FDA’s fast, flexible, and patient‑centered approach, while maintaining patient safety, will continue to accelerate progress across the field.
Above all, we remain focused on supporting and amplifying the voices of individuals and families affected by Duchenne. Your lived experiences must help guide decisions about potential therapies. We will continue to work closely with Capricor, other partners, regulators, and families to ensure that the path forward is clear and collaborative, and will update you throughout this process. Together, we will keep driving progress forward.
Read Capricor’s press release here.
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