New Haven-based biotech company Biohaven Ltd. has submitted a new drug application to the U.S. Food and Drug Administration for troriluzole, its potential treatment for Spinocerebellar Ataxia Type 3.
Patients with this rare, genetic neurodegenerative disease typically have progressive disability, problems with movement, frequent falls, loss of ambulation, and difficulty with speaking and swallowing. It can lead to premature death.
Biohaven leaders provided an overview of the company’s drug pipeline, including this new drug application, on Wednesday as part of the Yale Ventures’ Innovation Summit 2023.
Dr. Vlad Coric, CEO and chairman of Biohaven, said the new drug application for troriluzole represents approximately seven years of effort by the company to bring forward a potential treatment for this disease, which has no current therapy.
The company has been working closely with patient advocacy groups, including the National Ataxia Foundation, academic researchers and regulatory agencies, he said.
“Rare brain diseases are particularly challenging to research given relatively small populations of patients to participate in clinical trials,” Coric said, in an announcement.
Biohaven reported that its troriluzole studies showed positive results, such as a “robust reduction in fall risk.”
Spinocerebellar Ataxia Type 3 impacts approximately 10,600 people in North America, the European Union and Japan, according to Biohaven.
