STAT+: Why a Huntington’s patient isn’t fazed by the waning effect of UniQure’s gene therapy — SkimNews
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- UniQure's gene therapy AMT-130 slowed Huntington's disease progression by 44% in a four-year analysis of 12 treated patients, down from a 75% slowing reported at the three-year update last year
- Lauren Holder, an HD advocate living with early-stage Huntington's disease, told STAT she isn't fazed by the declining treatment effect, reflecting the patient community's willingness to accept an imperfect option in a fatal disease with no approved disease-modifying therapies
- The treatment effect of AMT-130 appears to have waned between year three and year four, a shift that has raised outside questions about the therapy's long-term durability
- UniQure is not alarmed by the year-four decline, though independent observers have flagged durability concerns, setting up a tension between company interpretation and external skepticism as regulatory discussions advance
Why it matters: With no approved disease-modifying treatment for Huntington's, the 31-percentage-point drop in AMT-130's apparent efficacy from year three (75%) to year four (44%) gives regulators and clinicians a central durability question to weigh against the therapy's still-substantial slowing of a fatal disease — and Holder's response suggests the patient community may accept meaningful but imperfect benefit over no option at all.
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