Drugs

FDA Rejects Biohaven’s Troriluzole Application

FDA has issued Biohaven a complete response letter, rejecting the company’s NDA for Vyglxia (troriluzole) as a treatment for spinocerebellar ataxia (SCA), a rare and progressive neurodegenerative disease with no approved therapies. The agency letter cited concerns about potential bias and design limitations inherent in the real-world evidence (RWE) study that formed the primary basis for the company’s submission.

Biohaven’s filing was supported by Study 206-RWE, a three-year, externally controlled trial that showed troriluzole slowed disease progression by 50% to 70% compared with matched untreated controls, according to the company. The drug also appeared to reduce falls by more than 50% and delay loss of ambulation in SCA patients, it says.

Despite the study meeting its prespecified primary and secondary endpoints, FDA reportedly cited the reliance on external control data introduced the possibility of “bias, design flaws, lack of pre-specification, and unmeasured confounding factors,” the company says.

Biohaven says that the agency had previously advised the company that only a “large and robust treatment effect” could overcome the inherent limitations of a non-randomized trial. Biohaven says its results met that standard and were consistent across two independent, third-party-controlled data sets representing the largest natural history cohorts in the U.S. and Europe.

The company complains that FDA failed to apply the regulatory flexibility Congress has granted for rare diseases and missed opportunities to use tools such as advisory committee review, post-marketing studies, or accelerated approval pathways. The company also expressed frustration that the agency initially accepted the application for priority review, later planned but then canceled an advisory committee meeting just weeks before the expected date — a move Biohaven says deprived outside experts and patients of a public forum to weigh in.

Biohaven says it now plans to meet with FDA “as soon as possible” to discuss next steps and a potential path forward for Vyglxia.

Troriluzole is described by the company as a third-generation glutamate modulator designed to reduce excessive glutamate activity implicated in neurodegeneration. SCA affects about 15,000 people in the U.S. and 24,000 in Europe and the UK. The condition causes progressive loss of motor control, difficulty walking, and eventual wheelchair dependence.

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Gupta Raises Concerns About Overton’s Independence, Ideology

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