As we approach August 22, the FDA’s current Prescription Drug User Fee Act (PDUFA) action date for Capricor Therapeutics’ Biologics License Application (BLA) for Deramiocel, PPMD recognizes how closely the Duchenne community is watching for an update.
On August 13, 2026, Capricor shared the company’s plans to submit an amendment to the application, which the FDA indicated may result in the current August 22 PDUFA date being extended. PPMD has requested an update from Capricor and will share information with the community as soon as we have any additional details.
We know how important this decision is to individuals and families affected by Duchenne, and we remain committed to keeping you informed throughout this process.
Background
Capricor’s original regulatory application focused primarily on cardiac benefit, particularly preservation of left ventricular ejection fraction (LVEF). The current review has shifted toward the PUL 2.0 data, specifically preservation of upper-limb function in people with Duchenne who already have upper-limb impairment. Capricor has submitted additional HOPE-3 open-label extension data and statistical analyses to support this narrower, function-based indication, which FDA is now evaluating.
Below is a brief refresher on the journey Deramiocel has taken to this point and the information PPMD has shared along the way.
Capricor Announces Plans to Submit BLA Amendment
Capricor has indicated that the company plans to submit an amendment containing additional data and analyses to support the ongoing FDA review. FDA has indicated to Capricor that the current August 22 PDUFA date may be extended once the amendment is submitted and received by the agency.
FDA Holds Advisory Committee Meeting for Deramiocel – The FDA’s Cellular, Tissue and Gene Therapies Advisory Committee met to discuss Capricor’s BLA for Deramiocel. The committee voted 3–9 that the available evidence did not support the effectiveness of Deramiocel for the treatment of cardiomyopathy in Duchenne. The vote was advisory and nonbinding; the FDA makes the final decision. PPMD participated in the Open Public Hearing and shared patient preference research and perspectives from the Duchenne community. Learn more.
Capricor Therapeutics Announces New PDUFA Date for Deramiocel – Following Capricor’s submission of additional data and supporting documentation from HOPE-3, the FDA lifted the previous CRL and resumed review of the BLA. The FDA established August 22, 2026 as the new PDUFA action date.
Capricor Therapeutics Announces Positive Data from HOPE-3 Study of Deramiocel – Capricor announced additional analyses and functional outcomes data from the Phase 3 HOPE-3 study. PPMD shared this information with the community as the FDA’s review continued.
Capricor Therapeutics Announces Positive Topline Results from HOPE-3 Study of Deramiocel – Capricor announced positive topline results from the Phase 3 HOPE-3 trial, and confirmed that the company would submit the data to FDA in response to the July 2025 CRL.
Capricor Therapeutics Receives Complete Response Letter Regarding Deramiocel – The FDA issued a Complete Response Letter (CRL) to Capricor, meaning the application could not be approved in its current form. The CRL also identified areas where additional efficacy data and clarification in the Chemistry, Manufacturing, and Controls (CMC) section were needed, and Capricor shared plans to submit data from the Phase 3 HOPE-3 clinical trial in response.
Capricor Therapeutics Announces FDA Acceptance and Priority Review of Biologics License Application for Deramiocel – The FDA accepted Capricor’s BLA for Deramiocel and granted Priority Review. The original PDUFA action date was August 31, 2025.
What Happens Next?
We recognize that waiting for a regulatory decision can be difficult, particularly after years of research, clinical trials, community advocacy, and anticipation. PPMD will continue to monitor developments closely, communicate with Capricor and other stakeholders, and share information with the Duchenne community as soon as it becomes available.
In the meantime, we encourage families to revisit the resources above for additional context on Deramiocel and the FDA review process.
The post Deramiocel: Where We Are Today appeared first on Parent Project Muscular Dystrophy.