The FDA’s Cellular, Tissue and Gene Therapies advisory committee is set to review Capricor Therapeutics’ Duchenne muscular dystrophy cell therapy deramiocel (deramiocel). Ahead of the meeting, an FDA expert critique disputed Capricor’s Phase 3 conclusions, arguing the evidence did not cleanly separate from placebo on upper-limb and cardiac function endpoints and that the company made multiple post hoc changes to the statistical analysis plan. Capricor has positioned deramiocel for boys and young men with DMD who have already lost the ability to walk, with the company previously citing statistically significant results across primary and secondary goals.