On June 10, 2025, news from Lexington, Massachusetts and Amsterdam, the leading gene therapy company UniQure N.V. (NASDAQ: QURE) announced the regulatory progress of its investigational gene therapy AMT – 130 for Huntington’s disease. This news undoubtedly brings new hope to numerous patients suffering from Huntington’s disease and their families.Huntington’s disease is a rare and fatal neurodegenerative disorder that typically manifests in middle age and is an autosomal dominant genetic disorder. The offspring of patients have a 50% chance of developing the disease. Its pathogenic root is the abnormal expansion mutation of the CAG trinucleotide on the short arm of chromosome 4. Patients gradually develop symptoms such as choreiform involuntary movements, cognitive impairment, and mental disorders. As the disease progresses, they often lose the ability to take care of themselves. Currently, there is no effective cure.
UniQure Company has achieved remarkable results in recent B-class meetings with the US Food and Drug Administration (FDA) and subsequent guidance exchanges. The two parties have reached consensus on the statistical analysis plan and several key parts of the CMC information, which will strongly support the BLA submission of AMT – 130 expected in the first quarter of 2026.UniQure’s Chief Medical Officer, Dr. Walid Abi-Saab, said: “We are very pleased to maintain productive cooperation with the FDA and have made progress in advancing the BLA submission of AMT – 130 as planned. We are seeking an accelerated approval pathway based on years of clinical data support. This rigorous and targeted approach reflects the urgent need for treatment of Huntington’s disease patients and also demonstrates our determination to provide the first disease-modifying treatment option for this affected group. At the same time, we appreciate the continuous participation of the FDA and look forward to sharing the main data for three years in the third quarter of 2025.”
In the second quarter of 2025, UniQure and the FDA discussed the BLA submission plan for AMT – 130 in depth. The proposal to use external control data and a prospective-defined statistical analysis plan (SAP) became the key topic of discussion. The FDA continued to recognize the comprehensive unified Huntington’s disease assessment scale (cUHDRS) as an acceptable registration intermediate clinical endpoint for accelerated approval.According to the consensus reached, the main efficacy analysis of the BLA will focus on evaluating the 3-year changes in cUHDRS in patients receiving high-dose AMT – 130 compared to the propensity score-adjusted external control group. UniQure plans to use the propensity score-weighted external control from the ENROLL-HD dataset for the main analysis and submit certain sensitivity analyses, including the use of propensity score-matched external control analysis, as additional supporting evidence.
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