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FDA Declines RP1 Approval Citing Flawed Trial Data

8/1/2026

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Story summary
  • FDA declined in April to approve RP1, citing reliance on a study without a control group and requesting more data.
  • Reviewers said trial design flaws make it hard to tell whether tumor shrinkage was due to RP1 and that response rates may be inflated.
  • An advisory committee voted 10-3 for approval, with several members describing the data as “really messy.”
  • The agency will decide on RP1’s accelerated-approval request by August 2.