uniQure announces alignment with FDA on elements of approval path for AMT-130

uniQure (QURE) N.V. announced that the company reached agreement with the U.S. Food and Drug Administration, FDA, on key elements of an Accelerated Approval pathway for AMT-130. As part of uniQure’s Regenerative Medicine Advanced Therapy Type B meeting held in late November, the FDA agreed that data from the ongoing Phase I/II studies, compared to a natural history external control, may serve as the primary basis for a BLA submission under the Accelerated Approval pathway, avoiding the need for an additional pre-submission study. The FDA also agreed that cUHDRS may be used as an intermediate clinical endpoint and that reductions in neurofilament light chain measured in cerebrospinal fluid may serve as supportive evidence of therapeutic benefit in the application for accelerated approval.