FDA Reviews Deramiocel for DMD
Capricor Therapeutics announced the FDA has accepted for review its Biologics License Application (“BLA”) seeking full approval for deramiocel, an investigational cell therapy, as a treatment for patients diagnosed with Duchenne muscular dystrophy (“DMD”) cardiomyopathy
Additionally, the FDA granted the BLA Priority Review with a Prescription Drug User Fee Act (“PDUFA”) target action date of August 31, 2025 and at this time, the FDA has not identified any potential review issues.
"We are thrilled to announce the acceptance of our BLA bringing us one step closer to providing this first-in-class treatment for Duchenne-cardiomyopathy, a condition for which there are no approved therapies" said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “If our application is successful, we expect deramiocel to be a lifelong treatment, administered quarterly, with the potential to be widely adopted across the DMD-cardiomyopathy treatment landscape. We want to extend our appreciation to the patients, their families and advocates who continue to work with Capricor and to the FDA for its commitment to accelerating treatments for DMD.”
The BLA submission is supported by Capricor’s existing cardiac data from its Phase II HOPE-2 and HOPE-2 Open Label Extension (“OLE”) trials compared to natural history data from an FDA-funded and published dataset on the implications of DMD cardiomyopathy and potential biomarkers of disease progression. The FDA also informed the Company they have not yet decided whether an Advisory Committee meeting is needed in relation to this application.
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