An FDA advisory committee vote rejected Capricor’s evidence for deramiocel in Duchenne muscular dystrophy after a meeting dominated by dispute over statistical analysis and how the Phase 3 endpoints separated from placebo. Outside experts ultimately voted against “substantial evidence” that the therapy works, with balloting 9-3. Separate reporting highlights that FDA reviewers criticized the company’s conclusions, saying the agency’s own analysis found deramiocel failed to separate from placebo on key upper-limb and cardiac function endpoints. The agency also pointed to changes Capricor made to the statistical analysis plan after it was completed. The dispute matters for the broader cell-therapy field because the panel’s discussion centered on how sponsors should lock study plans for inferential integrity in rare-disease trials, particularly when therapies target advanced functional decline. Capricor’s next path likely hinges on resolving the FDA’s concerns around the interpretation framework rather than on expanding the dataset alone.