On 2 September 2026, uniQure announced the submission of a Biologics License Application (BLA) to the Food and Drug Administration in the US for the accelerated approval of AMT-130.

uniQure also announced that a Marketing Authorisation Application (MAA) has been submitted to the Medicines and Healthcare products Regulatory Agency in the UK.

These filings build on the trial data discussed at our November 2025 platform meeting: YouTube — video and EHDN Platform Meeting uniQure Topline Results — Report.
 
For details of all ongoing and completed EHDN-endorsed studies, click here.