uniQure Stock Drops 32% After FDA Demands Sham-Controlled AMT-130 Trial
uniQure shares tumbled 31.8% in a week after FDA feedback warned that AMT-130 phase I/II data versus external controls are insufficient as primary efficacy evidence. The regulator strongly urged a prospective, randomized, double-blind study with sham surgery control, prompting uniQure to plan a Type B meeting in Q2 2026.
1. Regulatory Feedback Impacts AMT-130 Development
In the FDA’s final minutes from the Jan. 30 Type A meeting, agency reviewers concluded that AMT-130 phase I/II data against external controls are inadequate for primary efficacy evidence. The FDA strongly recommended a prospective, randomized, double-blind trial with sham surgery control to confirm therapeutic benefit.
2. Stock Market Reaction
uniQure shares plunged 31.8% over one week following the FDA’s guidance update, extending a six-month loss of 38.9% compared with 19.6% industry growth. The sharp sell-off reflects investor concern over increased trial requirements and extended regulatory timelines.
3. Planned Next Steps
uniQure plans to pursue a Type B meeting with the FDA in Q2 2026 to discuss phase III study design options and establish a clearer registration pathway for AMT-130. Management remains confident in the candidate’s long-term potential based on durability and existing biomarker data.
4. Pipeline and Background
Beyond AMT-130 for Huntington’s disease, uniQure’s pipeline includes gene therapy programs for epilepsy, ALS and Fabry disease, as well as commercialized Hemgenix for hemophilia B in partnership with CSL Behring. The company retains RMAT and Breakthrough Therapy designations for AMT-130.