Biohaven on Thursday said the Food and Drug Administration declined to review its experimental treatment for the rare neurodegenerative disease spinocerebellar ataxia in a setback to one of the most advanced prospects in the biotechnology company’s pipeline.
Biohaven, which was spun out of the similarly named biotech Pfizer bought for $12 billion last year, had submitted the treatment for approval in people with a common form of the disease known as SCA Type 3. Though the drug, called troriluzole, failed to meet its main goal in a Phase 3 study, the Connecticut-based company pointed to subsequent analyses demonstrating a “numerical treatment benefit” in that particular subgroup.
The agency has expressed a willingness to be flexible with neurological disease treatments before. Earlier this year, it cleared a Biogen medicine for amyotrophic lateral sclerosis that had failed a late-stage trial, for instance.
Biohaven, though, said that the agency responded to its request with a “refuse to file” letter, indicating it wouldn’t review its application because the primary endpoint of its key study wasn’t met. The company has requested a meeting to address the agency’s concerns, with CEO Vlad Coric, in a statement, pointing to the lack of available treatments for SCA and the “disease modifying effects” observed in testing.
The disorder causes problems with movement, speaking, and swallowing. It affects about 6,000 people in North America, according to Biohaven.
"We stand committed to serving people suffering from SCA and will continue to work with the FDA to request further consideration of all the available data,” Coric said.
Troriluzole is one of several medicines that were spun into the new version of Biohaven following the Pfizer buyout. It previously failed trials in general anxiety disorder and Alzheimer’s disease, but is now being tested in Phase 3 studies in obsessive compulsive disorder.
Biohaven also has drugs in development for epilepsy, pain and cancer, and a drug in late-stage clinical trials for spinal muscular atrophy.
The company’s shares fell about 28%, to around $17 apiece, in midday trading Thursday.