FDA Setback Puts uniQure’s Huntington’s Gene Therapy Filing in Doubt

The agency signaled that Phase I/II results versus external controls would not provide the primary evidence for a BLA.

Overview

uniQure said a recent pre-BLA meeting indicated the FDA no longer agrees that AMT-130’s external-control Phase I/II data are adequate as primary evidence for approval.
The company expects final meeting minutes within about 30 days and described the stance as a key shift from prior guidance after earlier RMAT and Breakthrough designations.
The timing of a Biologics License Application is now unclear, and uniQure plans urgent engagement with the FDA to determine a path forward.
uniQure said it will also advance discussions with regulators in the European Union and United Kingdom as it evaluates next steps.
Shares fell about 67% in premarket trading following the update, according to financial reports.