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Dutch drugmaker ​UniQure said ‌on Wednesday the ​U.S. ​Food and Drug ⁠Administration ​has agreed ​that data from a ​mid-stage ​study could support ‌an ⁠accelerated approval filing for ​its ​gene ⁠therapy to ​treat Huntington's ​disease.

~ 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 ~

uniQure remains highly sensitive to AMT-130's regulatory outcome, with current valuation reflecting significant uncertainty around FDA approval. Recent FDA leadership changes and a scheduled Type B meeting may improve the odds for AMT-130, but a Phase 3 trial is still likely required. My scenario analysis shows QURE's fair value per share ranges from $10.73 (bear) to $77.55 (bull), with the base case at $26.73.

UniQure is upgraded from Hold to Buy, driven by near-term UK approval prospects for AMT-130 and a robust cash runway to 2029. Despite FDA requiring a new Phase 3 trial for AMT-130 in Huntington's, a UK submission is imminent, positioning QURE for a major catalyst. QURE's pipeline, including AMT-260 for epilepsy and AMT-191 for Fabry, offers additional upside, though AMT-162 was discontinued.

uniQure N.V. (QURE) Q1 2026 Earnings Call Transcript

uniQure (QURE) came out with a quarterly loss of $0.85 per share versus the Zacks Consensus Estimate of a loss of $0.88. This compares to a loss of $0.82 per share a year ago.

~ Advancing FDA interactions on AMT-130 for Huntington's disease; Type B meeting scheduled for the second quarter of 2026 ~