STAT+: Following dispute with FDA, UniQure is cleared to submit Huntington’s treatment for approval

UniQure announced on Wednesday that the Food and Drug Administration (FDA) has reversed its prior opposition to AMT-130, an experimental treatment for Huntington's disease. This decision allows UniQure to proceed with filing for U.S. approval for the gene therapy. The biotech company plans to submit a marketing application in the third quarter, seeking accelerated approval based on a three-year analysis of an early-stage study. According to UniQure, the FDA deemed this analysis acceptable to support the application following a recent meeting. Previously, former FDA officials, including Commissioner Marty Makary and Vinay Prasad, had concluded that the same data was insufficient for a marketing application. This development marks a significant step forward for the treatment, which targets a rare and devastating neurological disorder.