Capricor Therapeutics recently announced in a press release that it has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) requesting more clinical data in response to the company’s Biologics License Application (BLA) for Deramiocel, the company’s cell therapy candidate for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD).
Deramiocel (CAP-1002) is a treatment that consists of allogeneic cardiosphere-derived cells (CDCs), which are a rare population of cardiac cells that are known to help preserve both cardiac and skeletal muscle function in dystrophinopathies like DMD.
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The FDA wrote in the CRL that it was unable to approve the application in its current form as it did not demonstrate substantial evidence of effectiveness. However, Capricor Therapeutics believes that certain outstanding items were addressed in prior communications.
The FDA offered Capricor Therapeutics the opportunity to meet and determine the appropriate next steps.
Previously, Dermamiocel was granted Priority Review in March 2025 based on promising data from the HOPE-2 trial and its open-label extension. The ongoing phase 3, HOPE-3 trial is a randomized, double-blind, placebo-controlled study involving 104 patients. Topline results from this trial are expected later this year; if these findings are positive and help demonstrate stabilization of cardiac health, skeletal muscle function preservation and a consistent safety profile, they can potentially resolve the questions raised by the FDA with regards to the effectiveness of this therapy in treating DMD-associated cardiomyopathy.
“While this was an unexpected decision by the FDA, we remain committed to the DMD community to get Deramiocel through the approval process,” Linda Marbán, CEO of Capricor Therapeutics, said.
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