FDA Denies Biohaven's VYGLXIA Application for Spinocerebellar Ataxia Treatment

Biohavens application for VYGLXIA, a treatment for spinocerebellar ataxia, was denied by the Food and Drug Administration due to concerns over study bias and design flaws.

• Biohaven Ltd. received a Complete Response Letter from the Food and Drug Administration rejecting its New drug application for VYGLXIA (troriluzole) aimed at treating Spinocerebellar ataxia.
• Despite meeting its prespecified primary and secondary efficacy clinical endpoint, the FDA expressed concerns regarding potential bias and design flaws in Biohavens real-world evidence study.
• The FDAs rejection highlights the ongoing challenges in demonstrating treatment effectiveness using real-world data, emphasizing the need for rigorous study designs in drug applications.