UniQure Advances AMT-130 Gene Therapy for Huntington's Disease with FDA Support for Accelerated Approval

UniQure has reached a significant milestone in the development of its investigational gene therapy, AMT-130, targeting Huntington's disease. Following a positive engagement with the FDA, the company is cleared to utilize ongoing Phase I/II study data and natural history external controls as the primary foundation for a Biologics License Application, bypassing the necessity for additional trials[1][2]. The FDA's approval of using the Unified Huntington’s Disease Rating Scale as an intermediate clinical endpoint, alongside neurofilament light chain as supportive evidence, demonstrates a strategic alignment with metrics that have previously expedited ALS treatment approvals[1][2]. AMT-130, designed to mitigate the production of a pathological protein by targeting the mutant huntingtin gene, has shown efficacy in reducing disease progression in preliminary trials, significantly bolstering UniQure's stock value post-announcement[1][2].