Clinical Trial Update: On 10th December, uniQure shared the exciting news about alignment with the US Food and Drug Administration (FDA) on a path forward for AMT-130 gene therapy in Huntington’s disease.

The FDA agreed that the basis for a Biologics License Application for Accelerated Approval of AMT-130 may include:

1. Data from ongoing phase I/II studies compared to data from a natural history study serving as an external control, avoiding the need for a further pre-marketing study

2. The composite Unified Huntington’s Disease Rating Scale as an intermediate clinical endpoint

3. Levels of neurofilament light chain in spinal fluid (a biomarker of neuronal damage) as supportive evidence for clinical benefit

Walid Abi-Saab, uniQure’s chief medical officer, explained, ‘This is an important milestone for the Huntington’s disease community as it puts us on the most rapid and efficient pathway to deliver a potentially life-changing therapy.’ The press release is available here: uniqure.gcs-web.com/news-releases

For details of all ongoing and completed EHDN-endorsed studies, click here.