uniQure Faces FDA Demand for Sham-Controlled Phase III Study for AMT-130
The FDA rejected uniQure’s Phase I/II data against an external control as sufficient evidence for AMT-130 and recommended a prospective randomized double-blind sham surgery-controlled study. uniQure will request a Type B meeting in Q2 2026 to discuss Phase III study design and align on an efficient regulatory pathway.
1. FDA Meeting Outcome
On January 30, 2026, uniQure and the FDA held a Type A meeting on AMT-130 for Huntington’s disease. The FDA concluded that data from Phase I/II studies compared to an external control do not meet the primary evidence requirement for a marketing application, and it strongly advised a prospective randomized double-blind sham surgery-controlled study for Phase III development.
2. uniQure’s Next Regulatory Steps
uniQure intends to continue discussions with the FDA, including a request for a Type B meeting scheduled in Q2 2026. The company plans to present potential study designs aimed at achieving a clear, scientifically valid approval pathway while leveraging any available regulatory flexibility.
3. Program and Financial Implications
The requirement for a sham-controlled Phase III trial may extend AMT-130’s development timeline and increase clinical costs, potentially delaying market entry for Huntington’s disease. uniQure’s ability to secure additional funding and align on trial parameters will be critical for advancing this therapy.