Capricor Therapeutics Completes FDA Mid-Cycle Review Meeting For Deramiocel In Duchenne Muscular Dystrophy-Associated Cardiomyopathy

Capricor Therapeutics, a biotechnology company specializing in innovative cell and exosome-based treatments for rare diseases, has announced the successful completion of a mid-cycle review meeting with the U.S. Food and Drug Administration (FDA) regarding its Biologics License Application (BLA) for deramiocel. This investigational cell therapy is being developed as a potential treatment for cardiomyopathy in patients with Duchenne muscular dystrophy (DMD)—a serious and progressive genetic disorder affecting muscle function.

During the meeting, the FDA informed Capricor that no significant deficiencies were identified in the BLA by the Review Committee at this stage. Additionally, the regulatory review remains on schedule, with a Prescription Drug User Fee Act (PDUFA) target action date set for August 31, 2025. The FDA has also indicated its plan to convene an advisory committee meeting to further evaluate the application, although the specific date for this meeting has not yet been determined.

Linda Marbán, Ph.D., Chief Executive Officer of Capricor, stated, “The successful completion of our mid-cycle review meeting along with the upcoming advisory committee meeting represents major milestones on the path towards approval of deramiocel. Deramiocel is a first-in-class cellular therapy with the potential to halt or slow the progression of DMD-cardiomyopathy, and we are pleased to have the opportunity to present the efficacy and safety data to the advisory committee. We have been actively preparing for an advisory committee meeting, and we look forward to providing the physician and patient perspectives to highlight the weight of evidence supporting the transformative potential of deramiocel in treating DMD-cardiomyopathy.”

Capricor’s BLA submission is primarily based on cardiac data from its Phase 2 HOPE-2 trial and the HOPE-2 Open Label Extension (OLE) study. These results were compared against patient-level data from an FDA-funded natural history study of DMD-related cardiomyopathy, which includes valuable insights into disease progression and relevant biomarkers. It’s important to note that data from the ongoing HOPE-3 trial have not been included in the current BLA package. The progress of deramiocel marks a significant step forward in addressing the urgent needs of patients suffering from DMD cardiomyopathy, a life-threatening complication of the disease for which there are limited treatment options.