uniQure Hopeful In Accelerated Approval Potential For Huntington’s Disease

Updated interim data from uniQure N.V.’s Phase I/II study of its gene therapy AMT-130 in Huntington’s disease showed slowing of decline among treated patients compared with an external control, raising the company’s optimism that it may have a pathway to accelerated approval from the US Food and Drug Administration.

The data released 9 July show that patients receiving the high dose of AMT-130 had an 80% slowing of disease progression in the composite Unified Huntington’s Disease Rating Scale (cUHDRS)...