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FDA Advisers Find Replimune’s RP1 Trial Results Clinically Meaningful

The panel’s vote strengthens the case for accelerated approval by spotlighting an efficacy signal in a hard‑to‑treat group despite lingering agency doubts about the study’s reliability.

Overview

  • An FDA cellular, tissue and gene therapies advisory panel voted 10–3 on Thursday that the single‑arm IGNYTE trial results for RP1 plus nivolumab are evaluable and clinically meaningful, giving Replimune a major regulatory boost.
  • The committee’s question addressed only whether the trial’s efficacy readout was interpretable, not whether the drug should be approved, leaving the final decision to FDA officials.
  • FDA staff reviewers argued the trial’s response assessments were unreliable because many responses involved tumors injected with RP1 and could reflect local effects rather than a systemic drug benefit.
  • Company and agency calculations differ sharply: Replimune reported a 33.6% overall response rate and a 24.8‑month median duration of response, while FDA staff recalculated a 15.7% response rate and a 14.1‑month duration after excluding injected‑lesion effects.
  • The advisory vote drove shares sharply higher and raises pressure on the agency ahead of an accelerated‑approval target date of August 2, 2026, a decision that will determine whether patients gain earlier access despite unresolved safety and evidence questions.