The FDA advisory committee meeting on Capricor Therapeutics’ Duchenne muscular dystrophy therapy deramiocel ended with experts voting against the company, underscoring a high bar for statistical proof in advanced rare-disease programs. At issue was whether Capricor’s evidence set met FDA expectations for “substantial evidence” after a contentious review of the company’s analysis plan and endpoint separation. In parallel, FDA reviewers released briefing materials criticizing the data interpretation, arguing deramiocel failed to clearly separate from placebo on upper-limb and cardiac function endpoints in the phase 3 HOPE-3 dataset. The agency also said Capricor’s post-finalization statistical analysis-plan changes further muddied conclusions. For Capricor, the vote adds to an already strained review history and raises the likelihood that future interactions with FDA will require a clearer, pre-specified analytic approach aligned to regulatory expectations. The outcome also sets a benchmark for how other cell-therapy makers should structure SAP governance when trials rely on complex functional endpoints.