FDA Accepts NDA for Troriluzole

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). Based on FDA Priority Review timelines and 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 from Study BHV4157-206-RWE (NCT06529146), 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 (f-SARA), 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. SCA patients treated with troriluzole showed a 50-70% slower rate of decline, representing 1.5-2.2 years delay in disease progression, over the 3-year study period.