Biopharmaceutical company Biohaven Ltd (NYSE:BHVN) announced on Tuesday that the US Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for troriluzole and granted it Priority Review for the treatment of spinocerebellar ataxia (SCA).
The FDA's decision is expected in the third quarter of 2025. If approved, troriluzole would become the first and only FDA-approved treatment for this rare, life-threatening neurodegenerative disease.
Troriluzole demonstrated a 50-70% reduction in SCA disease progression over three years in a real-world evidence study. The drug also met primary and secondary endpoints across multiple studies, showing significant disease stabilization in the SCA3 genotype and a reduction in falls across all SCA genotypes.
Biohaven previously received Fast Track and Orphan Drug Designation from the FDA, as well as Orphan Drug Designation from the European Medicines Agency, where a marketing authorization application is under review. An expanded access protocol is currently enrolling eligible SCA patients to provide early access to the treatment.
Subject to FDA approval, Biohaven plans to commercialize troriluzole in the US in 2025.
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