Biohaven’s troriluzole NDA in spinocerebellar ataxia accepted by the FDA

Biohaven (BHVN) announced that the FDA has accepted for review the company’s New Drug Application, or NDA, for troriluzole for the treatment of adult patients with spinocerebellar ataxia, or SCA, and has granted Priority Review. This designation is assigned to applications for drugs that would offer a significant improvement over other available treatments for a given disorder or would provide a treatment option where none exists. In the case of SCA, a rare, genetic, neurodegenerative disease, troriluzole would be the first and only FDA-approved treatment for this life-threatening disorder. The FDA’s decision regarding the NDA is expected within 6 months of filing – during 3Q2025 -. If ultimately approved, Biohaven is prepared to commercialize troriluzole for SCA in the US in 2025. The NDA submission was based, in part, on positive topline results in which troriluzole 200 mg dosed orally in patients with SCA met the study’s primary endpoint of change from baseline on the functional Scale for the Assessment and Rating of Ataxia in all SCA genotypes at 3 years compared to an external control arm. Troriluzole showed statistically significant superiority across 9 consecutive, prespecified primary and secondary endpoints with highly consistent, sustained, robust and clinically meaningful treatment effects. Biohaven previously received both Fast-Track and Orphan Drug Designation from the FDA, and ODD from the European Medicines Agency, where a troriluzole MAA is currently under review.