Shares of uniQure N.V. (NASDAQ:QURE) dropped 45% on Monday after the company revealed that the U.S. Food and Drug Administration declined to support its proposed regulatory pathway for the approval of AMT-130, an experimental gene therapy targeting Huntington’s disease.
According to final minutes from a Type A meeting held on January 30, the FDA said it could not agree that results from Phase I/II trials — when compared with an external control group — provide sufficient primary evidence of effectiveness to support a marketing application for AMT-130. The agency instead strongly recommended that uniQure conduct a prospective, randomized, double-blind study incorporating a sham surgery control.
The company said it plans to continue discussions with the FDA regarding Phase III development requirements and intends to request a Type B meeting in the second quarter of 2026 to further explore potential clinical trial design options.
“While we did not reach alignment on a submission pathway based on the Phase I/II data, we believe the totality and durability of our data warrant continued substantive dialogue regarding how the FDA’s stated commitment to regulatory flexibility may be appropriately applied in this setting,” said uniQure Chief Executive Officer Matt Kapusta.
The FDA’s stance represents a significant delay to the company’s development timeline, as conducting a new controlled trial would require additional investment and extend the path toward potential commercialization.
AMT-130 is being developed as a treatment for Huntington’s disease, a hereditary condition that leads to the gradual degeneration of nerve cells in the brain.
uniQure describes itself as a gene therapy developer focused on advancing potentially transformative treatments for patients with serious and unmet medical needs.