FDA cited real-world evidence flaws to reject VYGLXIA (troriluzole) for spinocerebellar ataxia in November 2025. Six months later, Marty Makary was gone.
The complete response letter for Biohaven’s ataxia drug cited potential bias, design flaws, and unmeasured confounding factors in the real-world evidence study, despite troriluzole hitting all nine prespecified hierarchical endpoints and showing 50-70% slowing of disease progression against external controls. CEO Vlad Coric said prior FDA leadership had reviewed the protocol and indicated approval would follow strong efficacy results. Makary’s FDA didn’t agree. The rejection forced Biohaven into a ~60% cut to annual R&D spend.
Coric didn’t absorb it quietly. He began building a covert push that reached the White House and Kennedy’s HHS office, framing the rejections as systemic and citing parallel CRLs against other rare disease programs. Makary went on television defending “rigorous” standards while growing isolated from the political circle that had put him there.
In late April, Coric secured a meeting with Makary. Trump signed off on Makary’s departure shortly after, on May 12.
What made this campaign land where single-company lobbying rarely does: multiple CEOs with parallel grievances funneling complaints through the same HHS channel simultaneously. Biohaven’s troriluzole CRL wasn’t the only kindling. It was the one that lit it.
Kyle Diamantas was named acting commissioner on May 12. The troriluzole CRL stands.
— Sarah Chen