UniQure to seek approval of Huntington’s gene therapy after FDA U-turn - Pharmaceutical Technology

uniQure confirmed it will submit its BLA in Q3 2026, though there is still a required confirmatory study to conduct. uniQure confirmed it will submit its BLA in Q3 2026, though there is still a required confirmatory study to conduct. The FDA U-turn brings an apparent end to a tumultuous regulatory submission process for the gene therapy. Credit: Tada Images/Shutterstock.com. UniQure has confirmed it will submit its Huntington’s disease gene therapy for regulatory review, after the US Food and Drug Administration (FDA) rescinded previous demands for extra clinical data. If approved, uniQure’s AMT-130 would be the first ever therapy indicated…

Source: Pharmaceutical Technology