New Haven-based biotech company Biohaven Ltd. is hoping to convince the U.S. Food and Drug Administration to reconsider its decision that it won’t review Biohaven’s new drug application for troriluzole, its potential treatment for Spinocerebellar Ataxia Type 3.
This rare, genetic neurodegenerative disease often results in progressive disability, including problems with movement and falling, and difficulty with speaking and swallowing. It can lead to premature death, and there is no current treatment available.
Spinocerebellar Ataxia Type 3 impacts approximately 10,600 people in North America, the European Union and Japan, according to Biohaven.
This past week, Biohaven announced it had been informed by the FDA that it would not review its new drug application (NDA) for troriluzole, citing its failure to meet its primary endpoint in studies.
The FDA gave Biohaven 30 days to indicate if it wants a meeting.
Dr. Vlad Coric, CEO and chairman of Biohaven, said the company is requesting a meeting to address the FDA’s concerns, as the company wants to work closely with the FDA to bring troriluzole to people with this disease as quickly as possible.
“As a physician, I am deeply disappointed by the FDA’s decision not to review the submitted NDA, and not to give complete consideration of all available data that we believe show disease modifying effects for this genetic disorder,” Coric said.
According to Coric, the drug is well-tolerated, and data suggested an 80% reduction or 7-month benefit in disease progression over a one-year study period.
“The risk-benefit profile for troriluzole warranted careful consideration by the FDA for this ultra-rare disorder,” Coric added. “SCA is clearly a severely debilitating, life-threatening disease with substantial unmet need.”
Coric asserted these patients “at least deserved a thorough review of the data package submitted.”
“We believe the NDA package is compelling and shows that treatment with troriluzole leads to clinically meaningful treatment benefits, including significantly delaying disease progression and reduction in falls,” Coric said. “We stand by these data and analyses.”
Biohaven has several other candidates in its drug pipeline.
The company is continuing studies of BHV-7000 for epilepsy, for example. According to the company, early electroencephalogram data shows antiseizure efficacy without negative effects such as sleepiness and speech impairment.
Biohaven also anticipates beginning a Phase 2 clinical trial with BHV-8000 for Parkinson’s disease and potentially other neuroinflammatory diseases in 2024.
Enrollment in a Phase 3 trial for obsessive compulsive disorder for troriluzole is expected to be complete by the end of 2023.
Biohaven on Monday reported its cash, cash equivalents and marketable securities totaled $349 million as of June 30. The company reported a net loss for the quarter ending June 30 of $90.3 million, or $1.32 per share, compared to a loss of $203.3 million, or $5.16 per share, for the same period in 2022. Biohaven’s stock was trading at $19.52 a share as of Monday morning.
