FDA Accepts Phase 1/2 Data for uniQure's AMT-130 BLA, Plans Q3 Submission
QURE has more than doubled off its 52-week low of $8.73 on light trading volume (0.4× avg).
Summary
uniQure announced that the FDA has agreed to accept 3-year Phase I/II study data as the primary basis for a Biologics License Application (BLA) for accelerated approval of AMT-130 in Huntington's disease. The company intends to submit the BLA in the third quarter of 2026. This is a significant positive development, potentially reversing the 'significant FDA setback' for AMT-130 reported on May 5th. It provides a clear regulatory path forward for uniQure's lead gene therapy candidate, substantially de-risking the program and moving it closer to commercialization. The FDA also seeks to align on the confirmatory study design prior to the BLA submission.
At the time of this announcement, QURE was trading at $27.50 on NASDAQ in the Life Sciences sector, with a market capitalization of approximately $1.7B. The 52-week trading range was $8.73 to $71.50. This news item was assessed with positive market sentiment and an importance score of 9 out of 10. Source: GlobeNewswire.