QURE news

uniQure reports 2026 Q3 results; FDA granted Priority Review for AMT-130 with a PDUFA date of May 1, 2027; company expects cash runway into 2030.

uniQure announced FDA acceptance of the ifezuntirgene inilparvovec Biologics License Application with a Priority Review designation and a PDUFA target date of May 1, 2027. The news marks a significant industry milestone as ifezuntirgene could become the first disease-modifying treatment for the dise…

uniQure submitted a Biologics License Application to the FDA and a Marketing Authorisation Application to the MHRA for AMT-130, seeking accelerated approval for Huntington's disease. Both filings are supported by Phase I/II data showing a slowed disease progression.

uniQure announced preliminary six-month data from its Phase I/IIa trial of AMT-260 for refractory mesial temporal lobe epilepsy, with three of six patients achieving 79-100% seizure reductions and no serious adverse events reported. The favorable safety profile and early biological signals support c…

The FDA agreed that uniQure's 3-year Phase I/II data for AMT-130 can serve as the primary basis for accelerated approval of the gene therapy for Huntington's disease, with BLA submission expected in Q3 2026. This regulatory milestone significantly de-risks the pathway to market for the company's lea…