uniQure Announces Plan for BLA Submission for AMT-130 in Huntington’s Disease

UniQure to seek FDA approval for Huntington's disease gene therapy after previous clash with agency

The announcement comes months after UniQure became embroiled in a public debate with FDA leaders over the clinical trial data supporting its application.

uniQure sets course to US filing for Huntington gene therapy

Shares in uniQure have rocketed this morning, as it emerges that the FDA will not require it to run a new trial of Huntington's gene therapy AMT-130.

UniQure to file gene therapy for approval, reflecting major shifts at FDA

The decision marks another regulatory U-turn following the exits of Marty Makary and Vinay Prasad, suggesting to some analysts that current FDA leadership may be more flexible in certain cases.

Following dispute with FDA, UniQure is cleared to submit Huntington’s treatment for approval

UniQure will submit a marketing application in the third quarter seeking accelerated approval for the treatment, called AMT-130.

uniQure Announces Plan for BLA Submission for AMT-130 in Huntington’s Disease

~ 3-year analysis from the Phase I/II study can serve as the primary basis of a Biologics License Application for

uniQure shares steady after FDA momentum, Huntington’s filing on radar

uniQure surged 78.4% to $48.16 after the FDA said Phase I/II AMT-130 trial data could support an accelerated-approval filing for its Huntington’s disease gene therapy, with a Biologics License Application planned for Q3 2026; the FDA still requires a confirmatory study and could decide the data are insufficient, leaving the stock’s rally dependent on regulatory follow-through. The post uniQure shares steady after FDA momentum, Huntington’s filin…