~ 3-year analysis from the Phase I/II study can serve as the primary basis of a Biologics License Application for accelerated approval with FDA ~

~ Company intends to submit the BLA in third quarter of 2026 ~

LEXINGTON, Mass. and AMSTERDAM, June 17, 2026 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that, during a recent Type B meeting with the U.S. Food and Drug Administration (FDA), the FDA communicated that the 3-year analysis from the Phase I/II study would be acceptable as the primary basis of a Biologics License Application (BLA) for the accelerated approval of AMT-130 in Huntington's disease. In addition, the FDA seeks to align on the confirmatory study design prior to the BLA submission, including consideration of concurrent control on standard-of-care therapy instead of a sham procedure. FDA communicated that they would work as expeditiously as possible with uniQure on this effort. The Company is committed to conducting the confirmatory study without delay and expects to further align with the FDA on the details of such a study prior to BLA submission. The Company intends to submit the BLA in the third quarter of 2026.

The Company expects to receive final minutes within 30 days of the recent Type B meeting.

AMT-130 has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA – the first RMAT designation for Huntington's disease – as well as Breakthrough Therapy designation and Fast Track designation.