New York, June 17, 2026, 09:08 EDT
- uniQure said the FDA told the company it will review three-year Phase I/II results as the primary evidence behind a planned accelerated approval submission for AMT-130 in Huntington’s disease.
- uniQure is bringing back a U.S. filing route after the FDA previously pushed back, and AMT-130 could become the first disease-modifying treatment for Huntington’s if approved.
- uniQure shares surged in the premarket session after a trading halt, up 78%, according to Seeking Alpha.
uniQure said Wednesday the U.S. Food and Drug Administration will let it use three-year data from its early Huntington’s gene therapy trial as the basis for an accelerated approval filing, changing course in the dragged-out regulatory battle over the therapy. The company will go ahead with a biologics license application (BLA) in the third quarter. That application is the standard FDA process for marketing a biologic drug in the U.S.
The move is significant now as it gives AMT-130 a U.S. path forward in the near term, after the agency earlier cast doubt on whether its overall data would be enough for an approval filing. Reuters said FDA officials late last year saw the data as lacking, and quoted a senior regulator who called the therapy a “failed product.” Reuters
The trial lands in a disease with no approved drugs to delay or slow symptoms. Huntington’s is a rare genetic brain disorder with movement, thinking and behavior changes. uniQure puts the number of patients at about 75,000 in the U.S., EU and UK.
AMT-130 aims to reduce activity of the huntingtin gene, which is responsible for making the mutant huntingtin protein tied to the disease. The one-time treatment uses MRI-guided stereotactic neurosurgery to deliver the drug into the striatum, a brain region key for movement.
uniQure said the FDA told the company after a Type B meeting that its filing can use the three-year Phase I/II data as the main support. The regulator also wants to settle on the confirmatory trial design before uniQure submits its application, including the possible use of standard-of-care patients as controls instead of a sham group.
“Today’s announcement reflects the outcome we have worked toward,” Chief Executive Matt Kapusta said. He said the FDA agreed the company’s current clinical data is enough for a near-term BLA submission. uniqure BV
The new regulatory step is based on uniQure’s September data. At 36 months, the high dose of AMT-130 slowed Huntington’s disease progression by 75% on cUHDRS, which measures movement, cognition, and daily living, against a matched external control. The same update reported a 60% slowing on Total Functional Capacity, a daily independence score.
Sarah Tabrizi, a clinical neurology professor at University College London and director of its Huntington’s Disease Center, last year called the uniQure data “the most convincing in the field to date” after the company published its 36-month results. She wasn’t referring to Wednesday’s FDA meeting. uniqure BV
But an approval isn’t locked in. uniQure is waiting for final FDA meeting minutes, which it expects in the next 30 days. The company warns that the FDA could decide the Phase I/II data fall short, that newer patient data might alter how the data are seen, or that post-approval demands could be expensive if there’s an accelerated approval.
uniQure shares surged in premarket trading, up 78% according to Seeking Alpha after a brief halt, and more than 70% higher per Reuters. The jump is tied to AMT-130’s regulatory outlook.
The move also changes Huntington’s drug development landscape. Novartis has licensed PTC Therapeutics’ PTC518, which is an oral HTT mRNA splice modulator, as part of a deal totaling up to $2.9 billion. Wave Life Sciences is working on WVE-003, its allele-selective silencing therapy targeting mutant huntingtin, now in clinical development.
uniQure is facing a procedural step next. The company needs to finalize the confirmatory trial and have its BLA accepted for review. Accelerated approval offers earlier access for serious conditions with unmet needs, but usually relies on evidence that needs later confirmation.
