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
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
Write to Robb M. Stewart at firstname.lastname@example.org
(END) Dow Jones Newswires
July 27, 2023 08:27 ET (12:27 GMT)
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