The FDA is signaling continued doubts about Capricor Therapeutics’ Duchenne muscular dystrophy cell therapy deramiocel ahead of a Cellular, Tissue and Gene Therapies Advisory Committee meeting. In a publicly posted review, agency analysts said the therapy failed to separate from placebo on key upper-limb and cardiac endpoints, and criticized post hoc changes Capricor made to the statistical analysis plan. Capricor’s deramiocel had previously been positioned as a therapy for boys and young men with Duchenne who have lost the ability to walk, with Phase 3 results reported last December as meeting primary and secondary objectives. The upcoming adcom marks another high-stakes inflection point for a program the company has argued addresses major unmet need in a difficult-to-treat population. In a separate development also tied to FDA advisory scrutiny, briefing materials ahead of a meeting on Replimune Group’s RP-1 in advanced melanoma again highlighted concerns about the single-arm trial design. FDA staff reiterated that the evidence is insufficient to determine whether RP-1 itself is effective and whether any systemic benefit is demonstrated when given locally with Opdivo. The parallel FDA messages underscore an ongoing focus on trial design rigor, endpoint separation, and statistical analysis integrity as these therapies seek their first approvals or attempts to clear prior regulatory hurdles.