FDA advisory panel votes against Deramiocel for DMD cardiomyopathy, 9 to 3

On July 29, 2026, the FDA's Cellular, Tissue and Gene Therapies Advisory Committee voted that available evidence did not support the effectiveness of Deramiocel for the treatment of cardiomyopathy in patients with Duchenne muscular dystrophy, a rare and life-threatening disease for which there is no approved treatment for this cardiac complication, with a vote of 3 for, 9 against, and 0 abstaining.¹ The Committee's vote is non-binding.¹

The Committee's voting question addressed a narrower indication than Capricor had proposed and did not include a vote on Deramiocel's overall benefit-risk profile.¹ In a separate discussion, the Committee's feedback was directionally supportive of the clinical evidence from the Phase 3 HOPE-3 trial, including results on its primary endpoint, PUL 2.0.¹

Capricor CEO Linda Marbán said the company remains committed to Deramiocel and to the patients who could benefit from it, and remains focused on working with the FDA toward approval ahead of the August 22, 2026, PDUFA target action date.¹

Per the filing, the Advisory Committee's recommendation is non-binding, and the FDA will consider it along with the complete administrative record in its ongoing review of the company's Biologics License Application for Deramiocel, with the PDUFA target action date remaining August 22, 2026.¹

DMD background: the disease affects approximately 15,000 individuals in the United States and primarily impacts boys, and over time deterioration of the heart muscle leads to cardiomyopathy and heart failure, with heart failure being the leading cause of death in DMD.¹