Capricor Therapeutics Receives Negative FDA Advisory Panel Vote On Deramiocel For DMD Cardiomyopathy, Final Decision Still Pending

Capricor Therapeutics (NASDAQ: CAPR) announced that the U.S. Food and Drug Administration's Cellular, Tissue and Gene Therapies Advisory Committee voted that the available evidence does not support the effectiveness of its investigational therapy, Deramiocel, for treating cardiomyopathy in patients with Duchenne muscular dystrophy (DMD). The committee voted 9 against, 3 in favor, with no abstentions. While the outcome is a setback for the company, the recommendation is non-binding, and the FDA will make the final decision on the therapy's approval.

According to Capricor, the advisory committee's voting question focused on a narrower treatment indication than the company had originally proposed. The company also noted that the panel did not vote on Deramiocel's overall benefit-risk profile, which will continue to be considered as part of the FDA's review process.

The committee also held a separate discussion on Deramiocel's impact on upper limb function in patients with Duchenne muscular dystrophy. Capricor said the feedback was generally supportive of the clinical evidence from its Phase 3 HOPE-3 trial, including results related to the study's primary endpoint, the Performance of Upper Limb (PUL 2.0) assessment. The company believes these findings continue to demonstrate the therapy's potential benefits beyond cardiomyopathy.

Commenting on the advisory committee meeting, Linda Marbán, Ph.D., Chief Executive Officer of Capricor Therapeutics, said the company remains committed to advancing Deramiocel for patients with Duchenne muscular dystrophy. She said the meeting provided an important opportunity to present the clinical evidence and added that the company was encouraged by the committee's discussion of the therapy's positive impact on skeletal muscle function.

Marbán also pointed to the open public hearing during the meeting, where patients, family members, and healthcare professionals shared their experiences with Deramiocel and highlighted the significant unmet medical need for treatments targeting DMD-related cardiomyopathy, a life-threatening condition for which there are currently no approved therapies.

Despite the advisory committee's vote, Capricor said it remains confident in the strength of the Phase 3 HOPE-3 clinical data and will continue working with the FDA throughout the review process. The company is now awaiting the FDA's decision on Deramiocel, with the Prescription Drug User Fee Act (PDUFA) target action date set for August 22, 2026.