UniQure N.V. shares surged around 75%–80% in premarket trading after the FDA reversed its stance and agreed that existing trial data from a phase 1/2 study of its Huntington's disease gene therapy AMT-130 can serve as the primary basis for an accelerated approval filing
The FDA had previously demanded additional data and a new trial, causing UniQure's stock to drop sharply, but now says a three-year analysis of the current study is sufficient for a Biologics License Application (BLA) under the accelerated approval pathway
UniQure plans to submit the BLA in the third quarter of 2026 and is working with the FDA to align on the design of a confirmatory study ahead of the filing
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