FDA hands Biohaven’s ataxia program a refuse-to-file letter, shares drop

Biohaven said Thursday it received a refusal-to-file letter from the FDA over its NDA for troriluzole in an announcement that sent its share price down more than 26% in market trading.

The regulator reportedly told the company it would not review the drug because the primary endpoint in the study it submitted was not met. Biohaven said in May 2022 that troriluzole missed the primary endpoint in a Phase III last year that enrolled 213 patients with different types of spinocerebellar ataxia (SCA).

However, at that time, Biohaven touted a subgroup analysis, showing benefit in a certain subset of SCA patients, and the company later shared a post hoc analysis showing the drug reduced the risk of falls by 58% in treated patients.

The biotech has now said it will request a Type A meeting with the regulator to address the agency’s concerns about the application for its SCA treatment. Biohaven had filed the NDA for troriluzole in this indication sometime in Q2.

Vlad Coric

“We stand by these data and analyses,” CEO Vlad Coric said in the press release.

The FDA’s action surprised Cowen analysts, who also said in a note to investors on early Thursday morning that they are disappointed by the FDA’s rejection.

“Due to the lack of a pre-NDA meeting, expectations for an acceptance and eventual approval by investors were quite low,” the analysts said. “This should have been a review issue and not something that prohibited the acceptance of the filing assuming that it was properly constructed.”

The drug has failed a number of trials in recent years, missing in anxiety and Alzheimer’s.

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