First-in-Class Drug May be Coming to You, SCA Patients!

Biohaven Pharmaceutical Holding Company Ltd. announced at the end of last month that the company has completed enrollment in its Phase 3 clinical trial of trigriluzole in spinocerebellar ataxia (SCA) patients.

Trigriluzole has already received both an Orphan Drug Designation and a Fast Track Designation from the US FDA as a potential treatment option for spinocerebellar ataxia.

Spinocerebellar ataxia, or SCA, is a group of rare neurological conditions that are characterized by problems with the movement control part of the brain. Treatment for SCA is currently focused on merely symptom management, and there are no approved medications to help the condition at its core. To learn more, click here.

Trigriluzole is a third-generation tripeptide prodrug, i.e. a biologically inactive group of peptides that can be metabolized in the body to produce a drug. It would work to treat the underlying biological causes of SCA.

The Phase 3 clinical trial is placebo-controlled to evaluate the safety and efficacy of using trigriluzole once-daily. One hundred eighty adults with SCA will be tested with the hopes that at the end of eight weeks of treatment, these patients will see a significant change in movement abilities, measured from baseline on the objective Scale for the Assessment and Rating of Ataxia (SARA).

The results from this study are expected to be released early next year, and if positive, Biohaven plans on submitting a New Drug Application (NDA) to the U.S. FDA the same year.

If you or someone you know has SCA or other rare forms of ataxia and want to learn more about this novel new drug, click here!


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