uniQure cleared for accelerated approval BLA

• The FDA will accept ~3-year data from the Phase I/II study of AMT-130 as the basis for a Biologics License Application.
• Following the announcement, uniQure shares traded at $5.88 in public market sessions.
• The company stated it intends to submit the BLA for accelerated approval during the third quarter of 2026.

uniQure (NASDAQ:QURE) announced that the FDA cleared its clinical data package for AMT-130 to serve as the primary basis for a Biologics License Application (BLA).

The regulatory path allows for an accelerated approval submission using approximately three years of follow-up data from the initial Phase I/II Huntington’s disease study.

The FDA aims to reach an agreement with uniQure regarding the design of a required confirmatory study, potentially replacing sham procedures with concurrent control groups on standard-of-care therapy.

AMT-130 holds several regulatory designations, including Regenerative Medicine Advanced Therapy (RMAT), Breakthrough Therapy, and Fast Track.

The company stated it remains on track to finalize its submission materials for the BLA during the third quarter of 2026.