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.
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April 24, 2026 15:15 ET Economics news flow was relatively light this week even as the conflict in the Middle East continued, raising concerns for policymakers. In the U.S., spending data, initial jobless claims and pending home sales were the highlights. Business confidence in the biggest euro area economy was in focus in Europe. Inflation data from Japan gained attention in Asia.