uniQure reversal puts natural-history controls back in play for rare neuro
FDA’s U-turn on AMT-130 gives Huntington’s and other rare disease sponsors a counterweight to CBER’s earlier rejection of external-control data
FDA’s latest reversal on uniQure’s AMT-130 does more than revive one Huntington’s gene therapy. It gives rare disease sponsors weighing a natural-history comparator a fresh regulatory data point — one that cuts against the agency’s signals over the past year and gives renewed hope to disappointed patients.
The decision gives uniQure N.V. (NASDAQ:QURE) a near-term filing path for AMT-130, an AAV gene therapy delivered directly to the striatum to lower HTT. The company said Wednesday it plans to submit a BLA in 3Q26, with the three-year Phase I/II analysis of the gene therapy serving as the primary basis for an accelerated approval application, supported by a propensity score-matched comparison with Enroll-HD natural history data. The biotech’s shares rose 78% to $48.16 on the news, putting its market cap over $3 billion...