Biohaven Achieves Breakthrough in Spinocerebellar Ataxia Treatment with Positive Pivotal Study Results for Troriluzole

Positive Study Results:
Biohaven announced positive topline results from the pivotal Study BHV4157-206-RWE, demonstrating the efficacy of troriluzole in treating spinocerebellar ataxia (SCA).

Treatment Benefits:
Troriluzole showed a 50-70% slowing of disease progression, representing a 1.5-2.2 years delay in disease progression over the 3-year study period.

Statistical Significance:
Troriluzole achieved statistically significant superiority on 9 consecutive, prespecified primary and secondary endpoints, including improvements on the modified functional Scale for the Assessment and Rating of Ataxia (f-SARA) at years 1, 2, and 3.

FDA Submission:
Biohaven plans to submit a New Drug Application (NDA) to the FDA for troriluzole in the treatment of all SCA genotypes in Q4 2024, which is eligible for priority review due to orphan drug and fast-track designations.

Clinical Impact:
The study's findings suggest that troriluzole could provide a significant therapeutic benefit for SCA patients, potentially improving their quality of life by slowing disease progression.