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 treating cardiomyopathy in patients with Duchenne muscular dystrophy (DMD), with a vote of 3 for and 9 against. The recommendation is non-binding, and the FDA will consider it alongside the complete administrative record during its ongoing review of the Biologics License Application. The PDUFA target action date remains August 22, 2026. While the committee was directionally supportive regarding upper limb function data from the Phase 3 HOPE-3 trial, this negative vote on cardiac efficacy represents a significant regulatory setback for the company's lead asset. Capricor Therapeutics is a biotechnology company developing cell and exosome-based therapeutics for rare diseases.
