uniQure NV has reached an agreement with the US Food and Drug Administration to proceed with a marketing authorisation application for its gene therapy for Huntington’s disease, a rare, inherited neurodegenerative disorder. The application is expected to be filed in the third quarter of this year, the company announced on 17 June.
This follows a demand by the FDA in January for data from a Phase 3 sham-surgery controlled study to support uniQure’s regulatory submission. The company appealed against this. A sham study is an inactive, placebo-like study designed to mimic a medical procedure in order to eliminate bias.
The gene therapy, AMT-130, is intended to be administered directly to the brain. The therapy uses an adeno-associated virus to deliver microRNA directly to the stratum in order to induce the silencing of a mutant huntingtin gene and the subsequent toxic proteins.
uniQure argued that a three-year analysis from a Phase 1/2 study of the therapy should be sufficient as the basis for an application. After further discussions, the FDA agreed to consider an accelerated approval of AMT-130 on the basis of this data, including an external control derived from a natural history study. The condition is that uniQure conduct a confirmatory study of the data without delay.
In a prepared statement, uniQure’s chief executive Matt Kapusta said the company is “deeply grateful for the FDA’s genuine commitment to addressing the unmet need of Americans living with Huntington’s disease.”
Huntington’s is an autosomal dominant condition where a genetic mutation leads to the production of abnormal protein in the brain. Approximately 75,000 people are said to have the disease in the US, EU and the UK.
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