Shares of uniQure N.V. (NASDAQ:QURE) fell sharply, dropping 45% on Monday after the U.S. Food and Drug Administration signaled it would not accept the gene therapy developer's proposed regulatory pathway for AMT-130.
According to final meeting minutes from a Type A meeting held on January 30, the FDA said it could not agree that data from the Phase I/II studies, when compared to an external control, would constitute the primary evidence of effectiveness required to support a marketing application for AMT-130. The agency strongly recommended that uniQure pursue a prospective, randomized, double-blind, sham surgery-controlled study.
The regulator’s stance was presented in the closed meeting minutes and represents a clear directive on the preferred design for pivotal testing, rather than acceptance of uniQure’s previously proposed approach. The FDA’s recommendation, if followed, would mean designing and executing a new controlled clinical study to meet the agency’s standards for primary effectiveness evidence.
uniQure said it plans to continue discussions with the FDA about Phase III development considerations and will request a Type B meeting in the second quarter of 2026 to further explore possible study design approaches. That meeting request is intended to advance conversations about how to structure additional trials in light of the FDA’s guidance.
In a statement included with the company’s disclosure, Matt Kapusta, chief executive officer at uniQure, said: "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."
The FDA’s position represents a setback for uniQure’s development timeline, since conducting a newly controlled study would require more time and resources before the company could seek marketing approval. AMT-130 is designed to treat Huntington’s disease, a genetic disorder that causes the progressive breakdown of nerve cells in the brain.
uniQure described itself as a gene therapy company advancing transformative therapies for patients with severe medical needs.
Summary of the regulatory status and next steps:
- The FDA did not accept uniQure’s proposal to rely on Phase I/II data versus an external control as primary evidence.
- The agency strongly recommended a prospective, randomized, double-blind, sham surgery-controlled Phase III study.
- uniQure intends to seek a Type B meeting in Q2 2026 to discuss potential Phase III study designs and will continue engaging with the FDA.