FDA staff reiterated concerns that Capricor’s Duchenne muscular dystrophy stem-cell therapy deramiocel did not meet Phase 3 objectives, according to FDA briefing materials ahead of an advisory committee meeting. The agency’s critique challenges the company’s earlier readout that deramiocel met primary and key secondary endpoints in a large randomized study. In parallel, FDA documents for Replimune’s RP-1 (suvolimogene oderparepvec) add pressure ahead of another Cellular, Tissue and Gene Therapies advisory committee session. Regulators questioned whether the single-arm phase 2 Ignyte design sufficiently demonstrates the investigational treatment’s efficacy, including potential systemic benefit when given locally with Opdivo (nivolumab). For investors and clinicians, both cases underscore a widening emphasis on trial design sufficiency—particularly single-arm frameworks and the ability to isolate effect attributable to the investigational product—when the agency is deciding whether the evidence supports approval.