Troriluzole Priority Review Granted for Spinocerebellar Ataxia

The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for troriluzole for the treatment of adults with spinocerebellar ataxia (SCA).

Troriluzole is a third generation prodrug of the glutamate modulating agent, riluzole. By increasing glutamate uptake from the synapse, troriluzole is expected to improve the glutamate deregulation commonly seen in neurodegenerative disorders such as SCA.  

The NDA is supported by pivotal data from the BH4157-206-RWE study (ClinicalTrials.gov Identifier: NCT06529146), as well as supportive data from the BHV4157-201 (ClinicalTrials.gov Identifier: NCT02960893) and BHV4157-206 (ClinicalTrials.gov Identifier: NCT03701399) studies. 

The BH4157-206-RWE study evaluated the effectiveness of troriluzole after 3 years of treatment in patients with SCA. Troriluzole data from the 3 year open-label extension study BHV4157-206 was compared with external real world control data collected from the Clinical Research Consortium for the Study of Cerebellar Ataxia (CRC-SCA; US SCA Natural History cohort) and the European Integrated Project on Spinocerebellar Ataxias (EUROSCA; European SCA National History Cohort). The primary endpoint was the change from baseline in the modified functional Scale for the Assessment and Rating of Ataxia (f-SARA). 

Findings showed troriluzole statistically significantly improved f-SARA scores after 1, 2 and 3 years of treatment. At year 3, disease progression (measured by f-SARA total score change from baseline) was slowed by 50% (1.5 year delay), 70% (2.2 year delay), and 60% (1.9 year delay) when comparing troriluzole with CRC-SCA data, EUROSCA data, and pooled CRC-SCA and EUROSCA data, respectively. Notably, disease progression, when defined as a 2-point or greater worsening on the f-SARA at 3 years, demonstrated an odds ratio of 4.1 (95% CI, 2.1-8.1) for the pooled untreated control arm vs the troriluzole arm (P <.0001).

“The robust clinical data presented in the NDA demonstrate sustained and compelling treatment benefit in SCA patients treated with troriluzole, a once-daily, oral pill,” said Melissa Beiner, MD, SCA Clinical Development Lead at Biohaven. “We look forward to working closely with the FDA throughout the review process to bring the very first treatment to patients and families suffering from SCA.”

According to Biohaven, the FDA’s decision on the NDA is expected during the 3rd quarter of 2025. If approved, troriluzole for SCA will be available in 2025. 

This article originally appeared on MPR