The FDA is not convinced that UniQure's experimental AMT-130 gene therapy for Huntington's disease provides benefit to patients[1][2]. After meeting with the FDA in late January 2026, plans to file for approval remain blocked[1]. The regulator considers the data from the completed single-arm phase 1/2 clinical trial to be insufficient to support the marketing application[1][5]. The FDA strongly recommended a prospective, randomized, double-blind, sham-controlled trial[1]. In a phase 1/2 study, no treatment effects were seen after 12 months compared to the sham control[2]. UniQure shares fell more than 40% in premarket trading[2]. The firm plans to request another meeting with the FDA in the second quarter of 2026 to discuss the Phase 3 study design[2]. This is a change from previous agreements, including the RMAT and Breakthrough Therapy statutes[4][6].