UniQure NV's experimental gene therapy AMT-130 slowed Huntington's disease progression by 44% after four years in a clinical trial, but the benefit waned from 75% at three years and failed to reach statistical significance. Despite the softening results, UniQure applied for FDA approval in September 2026 after the agency reversed its stance on data sufficiency. The news sent UniQure's U.S.-listed shares down 48% as investors questioned the treatment's long-term durability.
Efficacy results on the cUHDRS scale
- ▪The 44% slowing of Huntington's disease progression on the cUHDRS scale after four years of AMT-130 treatment did not reach statistical significance
- ▪UniQure NV stated that control-group patients in the AMT-130 trial who discontinued follow-up appeared to experience faster disease progression than those who remained, which may have understated the potential benefit of AMT-130
- ▪UniQure NV's experimental gene therapy AMT-130 slowed Huntington's disease progression by 44% in 12 high-dose patients after four years of treatment on the cUHDRS scale compared to an external control group, down from 75% after three years
Total Functional Capacity scale results
- ▪UniQure NV's AMT-130 showed a 61% slowing of disease progression at four years on the Total Functional Capacity scale, which assesses a patient's ability to perform everyday activities
- ▪Guggenheim analyst Debjit Chattopadhyay stated that a steady measure of Total Functional Capacity counterbalances the four-year softening observed in AMT-130's cUHDRS scale results
Regulatory review
- ▪The U.S. Food and Drug Administration is beginning to review UniQure NV's marketing application for its experimental Huntington's disease treatment AMT-130
- ▪UniQure NV applied for U.S. Food and Drug Administration approval of AMT-130 in September 2026, after the agency reversed its position that there was insufficient data on the treatment
Debatable claims
- ▪External control groups are reliable for evaluating experimental gene therapies
- ▪The FDA should approve UniQure's AMT-130 for Huntington's disease
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