Biohaven Ltd. announces positive topline results from pivotal Study BHV4157-206-RWE (NCT06529146) demonstrating the efficacy of troriluzole on the mean change from baseline in the f-SARA after 3 years of treatment. The study achieved the primary endpoint and showed statistically significant improvements on the f-SARA at years 1 and 2.

Biohaven Ltd.宣布了关键研究BHV4157-206-RWE（NCT06529146）的阳性结果，证明了曲利鲁唑对治疗3年后f-SARA基线平均变化的疗效。该研究达到了主要终点，并在第1年和第2年显示了f-SARA的统计学显着改善。

SCA is a rare, progressively debilitating neurodegenerative disease that affects approximately 15,000 people in the United States and 24,000 in Europe and the United Kingdom. There are no FDA approved treatments for SCA.

SCA是一种罕见的，逐渐衰弱的神经退行性疾病，在美国约有15000人受到影响，在欧洲和英国约有24000人受到影响。没有FDA批准的SCA治疗方法。

Collectively, data across multiple analyses demonstrate a robust and clinically meaningful slowing of disease progression in SCA patients. These treatment benefits translate into a 50-70% slower rate of decline compared to untreated patients, representing 1.5-2.2 years delay in disease progression over the 3-year study period.

总的来说，多项分析的数据表明，SCA患者的疾病进展速度强劲且具有临床意义。。

Additionally, in a responder sensitivity analysis, disease progression when defined by a 2 point or greater worsening on the f-SARA at 3 years showed an odds ratio (OR) of 4.1 (95% CI: 2.1, 8.1) for the untreated external control arm versus troriluzole treated subjects (p < 0.0001; pooled analysis)..

此外，在应答者敏感性分析中，当以3年时f-SARA恶化2分或更大定义时，未经治疗的外部对照组与曲利鲁唑治疗的受试者的比值比（or）为4.1（95%CI:2.1，8.1）（p<0.0001；汇总分析）。。

Based upon the topline data from Study BHV4157-206-RWE, and previous safety and efficacy data from the troriluzole development program in SCA, Biohaven plans to submit a New Drug Application (NDA) to the FDA in Q4 2024. The troriluzole development program has generated the largest clinical trial dataset in SCA and now has follow-up in some patients treated with troriluzole for over 5 years.

。曲利鲁唑开发计划已经产生了SCA中最大的临床试验数据集，现在对一些接受曲利鲁唑治疗超过5年的患者进行了随访。

Biohaven has previously received both Fast-Track and Orphan drug designation (ODD) from the FDA, and ODD from the European Medicines Agency, for troriluzole in SCA. An NDA with ODD is eligible for priority FDA review. Biohaven will be prepared to commercialize SCA in the US in 2025, if ultimately approved, based on potential priority review timelines..

Biohaven之前曾因SCA中的曲利鲁唑而获得FDA的快速通道和孤儿药指定（ODD），以及欧洲药品管理局的ODD。具有ODD的NDA有资格获得FDA的优先审查。如果最终获得批准，Biohaven将根据潜在的优先审查时间表，准备于2025年在美国将SCA商业化。。

Condition: Spinocerebellar Ataxia

条件：脊髓小脑共济失调

Type: drug

类型：药物