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FDA Rejects Biohaven’s Vyglxia for Spinocerebellar Ataxia

The decision cites flaws in Biohaven’s reliance on real‑world evidence with external controls.

Overview

  • The FDA issued a complete response letter declining approval and flagged potential bias, design limitations, and unmeasured or missing data in the submission.
  • Biohaven’s shares fell more than 40% on Wednesday following the rejection.
  • Biohaven said it will meet the FDA to discuss a potential path forward, as analysts caution that additional controlled clinical data will likely be required.
  • The company announced a restructuring that targets about a 60% reduction in annual direct research and development spending.
  • Vyglxia is being developed for spinocerebellar ataxia, which currently has no approved therapies, after a 2022 late-stage study missed its main goal and a 2023 filing was declined for review.