Biohaven Pharmaceutical Holding Company announced the U.S. Food and Drug Administration (FDA) has granted the company Fast Track Designation for Biohaven's product candidate trigriluzole (BHV-4157) for the potential treatment of Spinocerebellar Ataxia (SCA). Trigriluzole previously received Orphan Drug Designation from the FDA for the treatment of SCA in 2016. Biohaven is currently conducting a Phase 2/3 clinical trial in patients with SCA, with topline results expected in 2018.
SCA is a rare, debilitating neurodegenerative disorder that is estimated to affect approximately 22,000 people in the United States. Standard of care treatment is supportive and no medications are currently approved for patients with this debilitating condition.
"This Fast Track Designation from the FDA recognizes the high unmet medical need in patients with SCA," Vlad Coric, M.D., CEO of Biohaven said. "Fast track designation will help facilitate Biohaven's development of trigriluzole, and potentially expedite future regulatory review of trigriluzole for patients with SCA."
Biohaven initiated a randomized clinical trial of trigriluzole in patients with SCA in December 2016. Target enrollment is approximately 120 patients, with topline results expected in the first quarter of 2018. The study is designed to evaluate the safety and efficacy of trigriluzole for acute symptomatic treatment in this patient population. We believe that, if successful, this Phase 2/3 clinical trial will be the only pivotal trial necessary to support regulatory approval in SCA.
Trigiluzole is a novel tripeptide prodrug being developed by Biohaven. The company anticipates trigriluzole will be designated as a new chemical entity (NCE) and has received orphan drug designation from the FDA for the treatment of SCA.
As one of its Expedited Programs for Serious Conditions, the FDA grants Fast Track Designation to "facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need" in order to make important new drugs available to patients earlier.