uniQure N.V. (NASDAQ: QURE) announced that the U.S. Food and Drug Administration has communicated that three-year analysis data from the company's Phase I/II study of AMT-130 would be acceptable as the primary basis for a Biologics License Application seeking accelerated approval for the treatment of Huntington's disease.

The FDA communicated this position during a recent Type B meeting with uniQure. The company said it intends to submit the BLA in the third quarter of 2026. uniQure expects to receive final minutes from the meeting within 30 days.

The FDA also indicated it seeks to align on the design of a confirmatory study prior to BLA submission, including consideration of a concurrent control on standard-of-care therapy instead of a sham procedure. The agency said it would work as expeditiously as possible with uniQure on this effort.

"The FDA has agreed that our current clinical data can support a near-term BLA submission and has committed to work expeditiously with us to align on the design of the required confirmatory study," said Matt Kapusta, chief executive officer at uniQure.

AMT-130 is a gene therapy delivered through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum. The U.S. Phase I/II study enrolled 26 patients with early manifest Huntington's disease. The European open-label study enrolled 13 patients. Additional cohorts have enrolled patients to evaluate AMT-130 with immunosuppression and in patients with lower striatal volumes.

AMT-130 holds Regenerative Medicine Advanced Therapy designation — which uniQure claims is the first such designation for Huntington's disease — as well as Breakthrough Therapy and Fast Track designations from the FDA.

Huntington's disease is a rare, inherited neurodegenerative disorder affecting approximately 75,000 people in the U.S., EU, and UK combined, according to the company. There are currently no approved therapies to delay onset or slow progression of the disease.