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The Food and Drug Administration was more than likely correct to reject Biohaven Pharmaceuticals’ treatment for spinocerebellar ataxia, a rare and debilitating neurodegenerative disease. At the very least, the decision announced Tuesday night was not a surprise to anyone paying attention. Approval was a coin flip, at best.
The complete response letter has not yet been posted to the FDA website, but according to Biohaven, the agency rejected its drug, called troriluzole, due to issues that can be “inherent to real-world evidence and external control studies, including potential bias, design flaws, law of pre-specification and unmeasured confounding factors.”
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