UniQure Shares Drop After More Negative News From FDA on Huntington's Disease Drug
By Josée Rose
UniQure shares fell Thursday after the gene therapy company said its Huntington's disease drug hit more regulatory roadblocks.
Shares of the Lexington, Mass., company fell 15% early Thursday, to $21.25, though the stock is up 44% year-to-date.
UniQure said the U.S. Food and Drug Administration said data submitted from the Phase I and Phase II studies of its drug AMT-130 are unlikely to provide the primary evidence to support a biologics license application submission. A BLA is a formal request to introduce a biologic product and the FDA reviews it to determine safety and performance before full authorization.
UniQure said it's carefully evaluating the feedback and will urgently request a follow-up meeting with the FDA for the first quarter of 2026.
"We are committed to collaborating with the FDA to advance AMT-130 to patients and their families as rapidly as possible," said Chief Executive Matt Kapusta.
AMT-130 is being developed to slow the progression of Huntington's disease. The FDA previously granted AMT-130 breakthrough-therapy designation based on data from the studies.
In November, the company said the FDA no longer agreed that data from the studies of AMT-130 are adequate to support a biologics license application. "Consequently, the timing of the BLA submission for AMT-130 is now unclear," the Amsterdam-based company said at the time.
Write to Josée Rose at josee.rose@wsj.com
(END) Dow Jones Newswires
December 04, 2025 09:17 ET (14:17 GMT)
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