FDA accepts Biohaven's troriluzole resubmission for ataxia and plans an advisory committee
- The FDA accepted Biohaven's resubmission of troriluzole for spinocerebellar ataxia as a response to the November 2025 complete response letter, with a decision due in Q1 2027.
- The rejection centered on bias in a real-world-evidence comparison against a natural history database; the new filing adds supportive analyses.
- The agency intends to convene an advisory committee, date to be announced. Shares jumped about 30% on the news after falling nearly 45% on the rejection.
💡 Points
Acceptance is a procedural step, not a verdict. The core problem, an external-control comparison where the primary endpoint was missed, hasn't changed, so the advisory committee is the real test of whether new analyses can offset that bias. Analysts call it the key de-risking event. For rare-disease sponsors the case is a template for how far natural-history data can carry an application.
Biohaven shares pop as FDA accepts resubmission for ataxia drug with adcomm expected ↗