UniQure Shares Drop After More Negative News From FDA on Huntington's Disease Drug

Dow Jones12-04

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 09:02 ET (14:02 GMT)

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