Biohaven shares jump 39% after FDA accepts resubmitted ataxia drug application
Key Facts
The U.S. Food and Drug Administration accepted Biohaven's resubmitted application for VYGLXIA, known as troriluzole, to treat spinocerebellar ataxia, according to a company announcement reported by Dow Jones on 22 October 2026. The company's shares rose 39% to $16.35 in premarket trading, according to the news agency's report, as the application returned to review following its earlier rejection. Biohaven said the resubmission included "new positive supportive analyses," describing the additions it submitted with the application. It added that the FDA deemed the resubmission a "complete response" to its previous rejection letter. The acceptance puts the treatment application back before the regulator, while an approval decision remains a later stage in the review process.
The FDA's previous letter concluded that "substantial evidence of effectiveness had not been established" for troriluzole in treating spinocerebellar ataxia. The agency said the real-world evidence study underpinning the application had design flaws and methodological limitations that introduced bias into the study. Its observations also covered the original trial, which failed to achieve statistical significance on its prespecified primary endpoint. The regulator objected to treating post hoc analyses as confirmatory evidence of the treatment's effectiveness. The previous rejection thus addressed both the original trial's outcome and the methodological basis of the real-world evidence study submitted to support the application.
The drug targets a disease that Biohaven estimated affects about 15,000 people in the United States, according to its February 2025 statement. The company estimated that about 24,000 people have the disease in Europe and the United Kingdom. It describes spinocerebellar ataxia as a group of inherited neurodegenerative disorders that cause a "progressive loss of voluntary motor control." The disease's effects include atrophy of the cerebellum and brainstem, and it can progress to loss of the ability to walk. Patients may also experience difficulty speaking and swallowing, according to the company's description. Biohaven says troriluzole's primary mechanism of action is to lower glutamate levels at synapses.
On spending, Biohaven announced a restructuring plan on 4 November 2025 aimed at cutting annual direct research and development spending by about 60%. The spending base covered by that target excluded personnel costs and stock-based compensation. The plan also included delaying lower-priority research programs, according to the company's statement following the regulatory rejection. In its annual report filed with the U.S. Securities and Exchange Commission, Biohaven said net cash used in operating activities totaled $609.4 million in 2025. That compares with $582.5 million used in operating activities in 2024, representing a year-on-year increase in operating cash outflows.
The previous rejection was accompanied by a 44% drop in Biohaven shares, according to a report published by Investing.com on 5 November 2025. Following that decision, Baird cut its price target on the stock to $42.00 from $52.00, according to another report by the website. The firm maintained its Outperform rating despite lowering its price target. In the current review, the FDA plans to convene an advisory committee meeting, according to Dow Jones, citing the company. A date for the meeting is expected to be set soon. The agency set a target decision date in the first quarter of 2027 for the resubmitted application.