UniQure won a key FDA reversal that clears the path to file for accelerated approval of its Huntington’s disease gene therapy, AMT-130. The company said the agency agreed that a three-year analysis of early clinical data is “acceptable” to support a marketing application, after a previously more restrictive stance tied to additional study requirements. With the FDA now aligned on what data can be used, UniQure plans to submit its marketing application in the third quarter. The company also highlighted that recent discussions with FDA officials included alignment on a confirmatory trial design that may reduce the need for a sham-controlled element. The decision is closely watched by rare-disease sponsors because it signals that FDA expectations for external control data and accelerated pathways may be shifting again for gene therapies. UniQure’s stock reaction underscored how much regulatory language and accepted evidence can swing timelines in CBER-aligned programs. Separately, the broader UniQure narrative is being framed as part of a wider period of FDA flexibility following leadership changes, but UniQure’s near-term focus remains on converting the FDA’s acceptance into an actual filing.