Regulatory and investor attention stayed focused on Replimune’s RP-1 as FDA reviewers reiterated concerns about the company’s statistical approach and the adequacy of evidence in a single-arm melanoma trial ahead of a Cellular, Tissue, and Gene Therapies advisory committee meeting. Briefing materials characterized the company’s analysis as failing to prove that RP-1 works as intended in its intended population. In separate coverage, FDA staff issues described rejection-risk dynamics repeatedly for RP-1, tied to how objective response rate and duration of response were derived compared with FDA analyses. Replimune has reportedly already received multiple complete response letters from the FDA. The episode highlights how regulators are treating design-for-approval claims in complex, locally administered cell-gene therapy settings where robust control groups and endpoint validation can be central to potential clearance.