uniQure N.V. (QURE) said the U.S. Food and Drug Administration does not agree that data from its Phase I/II studies of AMT-130 are sufficient to support a marketing application for the investigational gene therapy for Huntington's disease.
The company said it received final meeting minutes from a Type A meeting held on January 30, 2026. In the minutes, the FDA stated that Phase I/II data compared to an external control would not provide the primary evidence of effectiveness required for approval.
The agency strongly recommended that uniQure conduct a prospective, randomized, double-blind, sham surgery-controlled study.
uniQure said it plans to continue discussions with the FDA regarding Phase III development and intends to request a Type B meeting in the second quarter of 2026 to further discuss potential study design approaches.
The company said it remains committed to advancing AMT-130 and engaging with regulators to determine an appropriate development and regulatory pathway.
For comments and feedback contact: editorial@rttnews.com
Business News
June 12, 2026 17:14 ET Major central bank action was the focus this week in economic news. The European Central Bank became the first major central bank to move in response to the rising inflationary pressures in the backdrop of the conflict in the Middle East. In North America, the U.S. inflation and trade data as well as Canada’s central bank decision gained attention. The Chinese trade data was the main news in Asia.