Two separate U.S. FDA advisory committee reviews tightened regulatory scrutiny on cell therapies for Duchenne muscular dystrophy, with sharply different next steps for developers. Capricor’s deramiocel faced a contentious discussion centered on statistical analysis-plan questions and data interpretation during the CTGT meeting. The panel voted 9-3 against recommending approval, leaving Capricor to confront remaining efficacy evidence gaps before any potential FDA action. Separately, a broader DMD-focused cell-therapy pipeline context emerged as new Phase 3 data drew attention to HOPE-3 deramiocel clinical benefit signals. Published results in The Lancet describe functional slowing in boys and young men with advanced DMD, alongside increased allergic-type reactions. The juxtaposition of trial readouts and advisory-panel skepticism highlights how regulator-facing endpoints and analysis choices continue to drive outcomes. For biotech, the immediate operational takeaway is clear: teams preparing for FDA decisions must anticipate not only clinical effect size, but also the statistical handling that bridges trial design, unblinding, and evaluable endpoints. That focus is likely to shape how sponsors refine submission packages and how panels weigh single-arm and complex trial evidence.