FDA

uniQure Soars After FDA's U-Turn Opens Accelerated Approval Pathway for Huntington's Therapy

Timur Mekhantev — Thu, 18 Jun 2026 22:27:26 +0000

Shares of uniQure N.V. (NASDAQ:QURE) pumped around 78% on Wednesday after the FDA cleared the path for a uniQure AMT-130 FDA approval filing under the accelerated pathway — a dramatic reversal from the agency’s prior stance that had sent the stock to a 52-week low of $8.73.

What Happened: The AMT-130 FDA Approval Path Opens

During a recent Type B meeting with the FDA, regulators communicated that the 3-year analysis from uniQure's Phase I/II clinical study of AMT-130 is acceptable as the primary basis for a Biologics License Application under the accelerated approval pathway. The company intends to submit the BLA in the third quarter of 2026.

These are the same AMT-130 data that were previously concluded as insufficient to support a marketing application. That shock had sent QURE shares to a 52-week low of $8.73. Wednesday's announcement means the regulatory overhang that has haunted the stock for months has been fully lifted.

The FDA also asked to finalize the design of the required confirmatory study before the BLA is filed, including the potential use of concurrent controls on standard-of-care therapy instead of a sham procedure — a notable shift in trial expectations for this rare neurodegenerative disorder.

"Today's announcement reflects the outcome we have worked toward throughout our continued regulatory engagement with FDA, and we are deeply grateful for FDA's genuine commitment to addressing the unmet need of Americans living with Huntington's disease".
— Matt Kapusta, ...

FDA

uniQure Soars After FDA's U-Turn Opens Accelerated Approval Pathway for Huntington's Therapy

What Happened: The AMT-130 FDA Approval Path Opens

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