The FDA has reversed course on UniQure’s Huntington’s disease gene therapy, clearing the company to pursue an accelerated approval filing for AMT-130. UniQure said the agency accepted that a three-year analysis from an earlier phase I/II study is “acceptable” to support a marketing application, after previously requiring more data. UniQure now plans to submit its BLA in the third quarter, framing the decision as a regained willingness to use existing clinical datasets for accelerated pathways. The update follows earlier FDA skepticism tied to external-control evidence, which had constrained sponsors seeking approval. Separate reporting notes that UniQure and FDA aligned on a confirmatory trial design that may avoid a sham-controlled element, following a recent Type B meeting. Markets reacted positively, with UniQure shares jumping more than 70% in morning trading after the disclosure. For the gene therapy sector, the decision is another signal that FDA review posture—particularly around evidence standards for rare disease accelerated approvals—may be shifting toward greater flexibility, potentially changing timelines for other Huntington’s and rare neuro programs.