Shares of uniQure NV (QURE) surged 5.16% in intraday trading on Wednesday, following the company's announcement that it has reached an agreement with the U.S. Food and Drug Administration (FDA) on key elements of an accelerated approval pathway for AMT-130, its investigational gene therapy for the treatment of Huntington's disease.

According to the news, uniQure reported that the FDA has agreed that the data from the ongoing early-to-mid-stage studies of AMT-130, compared to a natural history external control, are sufficient to serve as the primary basis for a Biologics License Application (BLA) for accelerated approval. Furthermore, the FDA has also agreed that certain clinical and biomarker endpoints, such as the cUHDRS (a sensitive measurement of clinical progression) and reductions in neurofilament light chain (a biomarker of neurodegeneration), can be used as supporting evidence of therapeutic benefit in the application.

This alignment with the FDA marks a significant milestone for uniQure, as it positions the company on the fastest and most efficient pathway to potentially bring a revolutionary therapy to patients with Huntington's disease, a rare and debilitating genetic disorder. The company plans to engage further with the FDA in the first half of 2025 to discuss the statistical analysis plan and technical requirements for the BLA submission.