Biohaven Announces FDA Acceptance and Priority Review of Troriluzole New Drug Application for the Treatment of Spinocerebellar Ataxia

Biohaven Announces FDA Acceptance and Priority Review of Troriluzole New Drug Application for the Treatment of Spinocerebellar Ataxia News provided by Biohaven Ltd. Feb 11, 2025, 07:30 ET Share this article Share to X Share this article Share to X Spinocerebellar Ataxia (SCA) is a rare, genetic, life-threatening neurodegenerative disease with no available treatment. Troriluzole demonstrated a 50-70% slowing of SCA disease progression on the primary and secondary outcome measures at the 3-year endpoint in a real-world evidence (RWE) study. Troriluzole has a well-established safety profile and if approved, would be the first and only FDA-approved treatment for SCA; subject to receipt of FDA approval, Biohaven is prepared to commercialize troriluzole for SCA in the US in 2025. NEW HAVEN, Conn. , Feb. 11, 2025 /PRNewswire/ -- Biohaven Ltd. (NYSE: BHVN ) (Biohaven or the Company), today announced that the US Food and Drug Administration (FDA) has accepted for review the Company's New Drug Application (NDA) for troriluzole for the treatment of adult patients with spinocerebellar ataxia (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). Based on FDA Priority Review timelines and if ultimately approved, Biohaven is prepared to commercialize troriluzole for SCA in the US in 2025. Continue Reading Troriluzole vs Matched US Natural History External Control Shows 50% Slowing of Disease Progression at Year 3 Troriluzole vs Independent Matched EU Natural History External Control Shows 70% Slowing of Disease Progression at Year 3 Troriluzole vs Matched Global Natural History External Control Shows 60% Slowing of Disease Progression at Year 3 Troriluzole-treated Subjects vs Placebo Show Disease Stability in SCA3 Genotype at 1 Year Troriluzole-treated Subjects vs Placebo Show a Substantial Risk Reduction in Falls Across AlI SCA genotypes 1: Untreated SCA Patients Have Greater Risk of Significant Disease Worsening