UniQure had planned to file for approval of its AMT-130 gene therapy to treat Huntington's disease, but was blocked by the FDA[1][3]. According to the final minutes of the Type A meeting on January 30, 2026, data from the completed single-arm phase I/II study are insufficient for a marketing application[1][2]. The FDA rejected the use of external control data and strongly recommended that a prospective, randomized, double-blind study be performed with control of sham surgery[1][2][3]. An anonymous senior FDA official stated that the sham group would involve anesthesia and 1-3 scalp incisions without drilling into the skull[2][4]. UniQure's stock fell more than 40% after the announcement[3]. The company plans to request a Type B meeting in the second quarter of 2026 to discuss the design of the new study[2][3]. CEO Matt Kapusta affirmed his commitment to working with the FDA and supporting the Huntington's community[2].