Biohaven Revives Troriluzole Hopes with Positive Update for Spinocerebellar Ataxia

Clinical Trial Success:
Biohaven's pivotal study (BHV4157-206-RWE) demonstrated that troriluzole significantly slowed disease progression in patients with spinocerebellar ataxia (SCA), showing a 50-70% slower rate of decline compared to untreated patients over a 3-year period.

Treatment Benefits:
Troriluzole-treated patients experienced a 1.5-2.2 year delay in disease progression, offering additional years of independence and quality of life.

Statistical Significance:
The study achieved its primary endpoint, with statistically significant improvements on the modified functional Scale for the Assessment and Rating of Ataxia (f-SARA) at years 1 and 2.

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

Impact:
Troriluzole is the first treatment to show a delay in disease progression for SCA, offering hope for patients and families affected by this debilitating and progressive neurodegenerative disease.

Background:
Spinocerebellar ataxias are rare, inherited neurological diseases affecting movement and leading to increasing disability over time, with no FDA-approved treatments available.