Troriluzole FDA Approval Status
Last updated by Judith Stewart, BPharm on Feb 19, 2025.
FDA Approved: No
Generic name: troriluzole
Company: Biohaven Ltd.
Treatment for: Spinocerebellar Ataxia
Troriluzole is a glutamate modulator in development for the treatment of adult patients with spinocerebellar ataxia.
- Spinocerebellar ataxia (SCA) is a rare, genetic, neurodegenerative disease characterized by the progressive loss of voluntary motor control and atrophy of the cerebellum and brainstem. It is thought to be caused by mutations in the Purkinje cells in the cerebellar cortex. Patients with SCA experience significant morbidity, including impaired gait leading to falls, loss of ambulation and progression to a wheelchair, inability to communicate due to speech impairment, difficulty swallowing, and premature death.
- Troriluzole is a tripeptide prodrug of the active metabolite riluzole, which is a modulator of the excitatory neurotransmitter glutamate. It works by increasing glutamate uptake from the synapse to reduce synaptic levels of glutamate. Glutamate deregulation is thought to contribute to neurodegeneration and Purkinje cell dysfunction in patients with SCA.
- A New Drug Application (NDA) has been submitted to the U.S. Food and Drug Administration (FDA) for troriluzole for the treatment of adult patients with spinocerebellar ataxia. Positive topline results in a pivotal study of troriluzole in SCA were reported in September 2024, demonstrating that troriluzole slowed disease progression by 50-70% over the 3-year study period.
- Troriluzole has also been studied in a number of other diseases associated with excessive glutamate, including Alzheimer's disease, generalized anxiety disorder, and obsessive-compulsive disorder.
Development timeline for troriluzole
Further information
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