UniQure's shares soared by more than 40% in premarket trading on Wednesday following an announcement that the FDA has agreed to consider an accelerated approval application for its Huntington's disease gene therapy, AMT-130, based on the existing Phase 1/2 clinical trial data. This marks a significant turnaround after the FDA previously recommended a costly and time-consuming Phase 3 trial.
The biotechnology company had faced a precipitous drop in stock value earlier in the year when the FDA initially deemed the existing data insufficient and demanded a sham-controlled Phase 3 study. This earlier decision had cast doubt on the near-term future of AMT-130, which had shown promising results in slowing disease progression.
The FDA's change of heart comes after further discussions with UniQure regarding the regulatory pathway for AMT-130. The company indicated it would now prepare to submit its application for accelerated approval, a pathway that allows therapies for serious conditions to reach patients sooner based on surrogate endpoints or an effect on a clinical endpoint that is reasonably likely to predict clinical benefit. This development is seen as a major win for UniQure and offers renewed hope for patients awaiting treatment options for the rare neurodegenerative disease.
