The U.S. Food and Drug Administration (FDA) staff have issued briefing documents ahead of advisory committee meetings that cast serious doubt on the efficacy data for both Capricor Therapeutics' deramiocel (for cardiomyopathy in Duchenne muscular dystrophy) and Replimune's RP1 (for advanced melanoma), despite the companies having previously aligned with the agency on trial design and analyses. For Capricor, the FDA reviewers question the statistical approach, population characteristics, and overall evidence of benefit; for Replimune, the agency maintains that the single‑arm IGNYTE study remains 'not interpretable' and insufficient to demonstrate substantial evidence of effectiveness. These re‑evaluations of the same data sets—now framed through a stricter regulatory lens—underscore that a fresh look by the FDA does not automatically lead to a new regulatory conclusion and have reminded investors that re‑evaluation is not the same as reversal.
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