uniQure’s (QURE) stock rocketed more than 70% in premarket trading on Wednesday after the FDA reversed course and agreed that existing trial data could support an accelerated-approval filing for AMT-130, its Huntington’s disease gene therapy.
The jump came after uniQure announced the outcome of a recent formal Type B meeting with the regulator. The FDA confirmed that three-year follow-up data from the company’s ongoing Phase I/II study would be acceptable as the primary dataset for a future BLA submission.
That is a sharp turn from where things stood just months ago. In late 2024, FDA officials indicated the available data was not enough to support a filing. One senior regulator went as far as describing AMT-130 as a “failed product.” Wednesday’s news effectively puts that characterization to rest.
uniQure said it plans to file the BLA in the third quarter of 2026.
The FDA has signalled it wants to work quickly with uniQure to nail down the design of a post-approval confirmatory study before the BLA is submitted. One key discussion point is whether the control group in that study would receive standard-of-care treatment rather than a sham surgical procedure — a change that would ease the burden on trial participants.
uniQure said it is committed to launching the confirmatory study as soon as possible and expects to continue those conversations ahead of submission.
The company also expects to receive official meeting minutes from the FDA within 30 days of the Type B meeting.
The U.S. trial enrolled 26 patients with early manifest Huntington’s disease. Patients were randomised to either AMT-130 or a sham procedure, with the therapy delivered as a single dose directly into the striatum via MRI-guided neurosurgery.
Three-year data published last year showed patients on the high dose saw a 75% reduction in disease progression, based on a widely used clinical scale.
AMT-130 already carries heavy regulatory weight. It holds Regenerative Medicine Advanced Therapy (RMAT), Breakthrough Therapy, and Fast Track designations from the FDA. The RMAT designation was the first ever granted to a Huntington’s disease treatment candidate.
RBC Capital Markets analysts called the news “great news for QURE,” and suggested it may indicate the FDA is moving back toward regulatory flexibility following leadership changes at the agency.
Acting FDA Commissioner Kyle Diamantas met with rare disease advocacy groups earlier this month, with one attendee describing the meeting as a “breath of fresh air.”
Huntington’s disease is a fatal genetic disorder with no currently approved disease-modifying therapy. The accelerated approval pathway is designed to speed access to treatments for serious conditions where no good alternatives exist.
uniQure is running two multicenter Phase I/II studies evaluating AMT-130’s safety and efficacy.
The BLA submission is targeted for Q3 2026.
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