The U.S. Food and Drug Administration has cleared the company to submit a Biologics License Application for AMT-130, its investigational gene therapy. During a recent meeting, regulators confirmed that three-year data from an ongoing study would suffice as the primary evidence for the application. UniQure expects to file the paperwork by the third quarter of this year.

Beyond the primary submission, the company is coordinating with the FDA to finalize the design of a confirmatory study. This alignment aims to smooth the path for accelerated approval of the drug, which targets the underlying causes of the rare, progressive neurodegenerative disorder.