On July 22, 2025, Capricor Therapeutics provided a Community Letter with updates on the regulatory status of deramiocel, their investigational cell therapy for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy.
Please continue reading to view their Community Letter.
Dear Duchenne Community,
We want to update you on the regulatory status of deramiocel, our investigational cell therapy for the treatment of
cardiomyopathy associated with Duchenne muscular dystrophy (DMD). Recently, Capricor received a Complete
Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding its application to approve deramiocel. While the FDA’s response was not what we had hoped for and we understand how frustrating this may feel, we want to
reassure you that we remain fully committed to advancing deramiocel toward potential approval.
The FDA has informed us that our BLA, in its current form, does not meet the statutory requirement for substantial evidence
of effectiveness and has requested additional clinical data. This feedback came as a surprise to us, particularly given prior discussions with the Agency that encouraged us to proceed with our submission based on data from our HOPE-2 and HOPE-
2 OLE studies. While we respectfully disagree with the FDA’s interpretation of these data, we are fortunate to already have a Phase 3 clinical trial (HOPE-3) underway. We plan to propose including data from this pivotal study to strengthen and seek to amend the current BLA in support of the cardiomyopathy indication.
The FDA also noted some remaining technical items related to manufacturing. We believe that these are straightforward issues that we are already addressing, and we aim to complete this work in the coming weeks. We are currently preparing for a Type A meeting with the FDA which we have requested to discuss the best path forward for amending and resubmitting our BLA.
Capricor remains committed to continuing all ongoing clinical trials as planned, and all those receiving Deramiocel will continue have access to treatment until the trials are completed or otherwise closed.
We are extremely grateful for the continued support we’ve received from the DMD community—patients, families, clinicians, and advocates. Several of the nation’s leading neuromuscular and cardiac specialists have submitted a letter to the FDA expressing their support for deramiocel and their belief in its potential to meaningfully impact the lives of those living with DMD.
In the coming weeks, our team will participate in a PPMD-hosted webinar to provide further context, share insights into the
clinical data, and outline our next steps. We hope you’ll join us.
We recognize how much is at stake for families and individuals affected by DMD, and we are moving forward with focus,
urgency, and care to bring deramiocel one step closer to those who need it most. If you have questions, please speak with your
physician or contact our team here.
Sincerely,
Linda Marban, PhD Michael Binks, MD
Chief Executive Officer Chief Medical Officer
