An FDA advisory committee voted 9–3 against recommending approval of Capricor’s deramiocel for Duchenne muscular dystrophy cardiomyopathy, capping off a meeting marked by statistical disagreement over which analysis plan should guide the Phase 3 HOPE-3 efficacy readout. Panelists cited data fragility and said evidence of benefit was insufficient. Capricor argued the FDA improperly relied on an outdated draft statistical analysis plan, while the company’s reported success was based on a finalized version used before unblinding. During the meeting, committee members weighed the question of whether the evidence provides substantial effectiveness for cardiomyopathy endpoints. Even with the unmet-need backdrop, the panel rejection leaves deramiocel facing the next FDA review step without an adcom endorsement, underscoring how trial analytics and SAP governance can determine rare-disease cell therapy outcomes.