The U.S. FDA Cellular, Tissue, and Gene Therapies Advisory Committee voted against Capricor Therapeutics’ allogeneic cell therapy deramiocel for Duchenne muscular dystrophy-related cardiomyopathy after a meeting marked by a statistical plan dispute. The panel’s decision centered on whether the evidence met the FDA’s “substantial evidence” threshold. According to meeting coverage and FDA briefing materials, regulators criticized Capricor’s choice of statistical analysis plan versions used to interpret HOPE-3 endpoints, while the company argued the study’s final plan should govern interpretation. Capricor’s position that it had already demonstrated benefit in Phase 3 has not carried the day with the advisory experts. The outcome adds to regulatory uncertainty for rare-disease cell therapies that rely on complex endpoint and SAP methodology. For developers, it underscores how documentation and plan versioning can become central to efficacy debates, not just safety or raw effect sizes.