FDA approves UniQure path for Huntington gene therapy

Biotechnology company UniQure announced on Wednesday, June 17, 2026, that the U.S. Food and Drug Administration has agreed to a path for an accelerated approval of its experimental gene therapy for Huntington’s disease, a major reversal from the agency's previous position. The company's stock surged more than 70% following the news.

The FDA communicated that three-year data from an ongoing Phase I/II study of the therapy, AMT-130, would be acceptable as the primary evidence to support a Biologics License Application (BLA). As a result, UniQure plans to submit that application in the third quarter of 2026. An FDA official confirmed the agreement on a regulatory pathway, stating the agency remains committed to working with the company while upholding scientific standards.

This decision marks a significant shift from earlier this year. In March, the regulator had told UniQure its clinical trial data would not support an application, with a senior official previously having criticized the evidence. The company had compared patient outcomes to an external database of disease progression, arguing that a placebo-controlled trial requiring a sham brain surgery would be unethical. The...