The FDA reversed course again on uniQure’s Huntington’s disease gene therapy, AMT-130, clearing the company to file for U.S. approval under an accelerated pathway. UniQure said it will submit a marketing application in the third quarter after the agency deemed three-year analysis data from an early-stage study acceptable for the proposed review. The decision follows prior FDA opposition linked to concerns around the sufficiency of external-control evidence and earlier regulatory disagreement between agency leaders and the evidence package. In the latest pivot, uniQure reported agreement on an accelerated BLA filing strategy supported by the longer analysis window. The renewed flexibility matters for sponsors across rare diseases because it signals a willingness to accept alternative evidence designs when confirmatory trial structures are aligned. For AMT-130, the next critical step is how FDA and uniQure structure the confirmatory development path and what endpoints and control elements will be required to sustain accelerated approval.