Biohaven Told That FDA Won't Review NDA Application for Troriluzole

By Robb M. Stewart

Biohaven has been told that U.S. regulators won't review the clinical-stage biopharmaceutical company's recently submitted new drug application for a treatment of patients with spinocerebellar ataxia troriluzole since the study's primary endpoint wasn't met, and that they wouldn't permit a substantive review.

The company's shares were sharply lower after the news, falling more than 30% in premarket trading Thursday after ending the previous session at $23.80, up more than 70% so far this year.

Biohaven said the communication from the Food ad Drug Administration indicated the company may request a "type A" meeting within 30 days

The company said it was committed to working closely with the FDA to bring troriluzole to people with SCA3 as quickly as possible, given that no therapy is currently approved for the ultra-rare genetic disorder, and it is requesting a type A meeting to comprehensively address the regulator's concerns.

"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 that has no approved treatments," said Vlad Coric, chief executive and chairman.

Coric said troriluzole's active metabolite has a known safety profile and is well-tolerated, and its risk-benefit profile warranted careful consideration by the FDA for the disorder.

SCA is a debilitating, life-threatening disease and the company said about 200 patients have been treated with troriluzole for up to three years and the troriluzole-treated cohorts have remained stable compared with untreated natural history cohorts who clearly show marked disease progression over a similar time period.

Spinocerebellar ataxia is a group of dominantly inherited disorders characterized by progressive ataxia affecting the coordination of hands, arms and legs as well as balance and speech. Patients experience significant morbidity, including progression to a wheelchair, impaired gait leading to falls, inability to communicate due to speech impairment, difficulty swallowing, and premature death.

Write to Robb M. Stewart at robb.stewart@wsj.com

(END) Dow Jones Newswires

July 27, 2023 08:27 ET (12:27 GMT)

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