UniQure has confirmed it will submit its Huntington’s disease gene therapy for regulatory review, after the US Food and Drug Administration (FDA) rescinded previous demands for extra clinical data.
If approved, uniQure’s AMT-130 would be the first ever therapy indicated for Huntington’s – a neurodegenerative disorder. Approximately 75,000 people in the US, Europe, and the UK have the inherited disease, which is fatal within 15 to 20 years of symptom onset.
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The FDA U-turn brings an apparent end to a tumultuous regulatory submission process for the gene therapy. The agency refused to accept uniQure’s filing for AMT-130 in November 2024, citing insufficient data from a Phase I/II trial. After meeting with the FDA in January, the biotech relayed that the FDA was demanding another study.
It is unclear why regulators at the agency have changed their tune, but uniQure stated in a 17 June release that the FDA now says the three-year analysis from the Phase I/II study is an acceptable data package that can form a biologics License Application (BLA) for the accelerated approval of AMT-130.
While uniQure confirmed it will submit its BLA in Q3 2026, it will still need to conduct a confirmatory study. The agency “seeks to align on the confirmatory study design prior to the BLA submission”, which includes a potential concurrent control on standard-of-care (SoC) therapy instead of a sham procedure.
“FDA communicated that they would work as expeditiously as possible with uniQure on this effort. [We are] committed to conducting the confirmatory study without delay,” the company added.
uniQure is currently conducting two multi-centre, dose-escalating, Phase I/II clinical studies (NCT04120493, NCT05243017) to explore the safety, tolerability, and efficacy of AMT-130 for the treatment of Huntington’s disease. The FDA agreed that the company can compare patients treated in the first two study groups with similar patients from Enroll-HD, the world’s largest observational study and natural history dataset for Huntington’s disease.
The biotech’s CEO, Matt Kapusta, said: “Today’s announcement reflects the outcome we have worked toward throughout our continued regulatory engagement with FDA, and we are deeply grateful for FDA’s genuine commitment to addressing the unmet need of Americans living with Huntington’s disease.
“The consistency and strength of the clinical data generated to date give us great confidence in the product’s potential to make a meaningful difference for patients. We remain focused on bringing AMT-130 to patients and families as quickly and responsibly as possible in the US and globally.”
AMT-130 is a gene therapy delivered directly into the brain through a single neurosurgical procedure. The one-off treatment targets the striatum, the brain region most damaged in Huntington’s disease, where it delivers a gene encoding miRNA that silences the human huntingtin protein.
William Blair analysts highlighted that, when taken together with a similar agency U-turn on Replimune’s combo therapy in advanced melanoma, the FDA is showing less rigidity with drug submissions.
“Given Vinay Prasad and Marty Makary’s public opinion on this filing and complete response letter (CRL) issuance and now the positive uniQure regulatory update for AMT-130, we believe the current FDA, largely in caretaker mode, appears to be more flexible on regulatory paths for applications where concerns were previously raised,” they said in a note.
Prasad left his role as head of the Center for Biologics Regulation and Evaluation (CBER) in March 2026, while Makary officially resigned from his post as commissioner in May. Prasad, especially, incited controversy due to his approach to cell and gene therapy approvals. His strict stance on clinical trial endpoints led to significant tensions with drug developers and rare disease charities.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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