FDA Agrees On Accelerated Approval Pathway For AMT-130 In Huntington's Disease; Stock Up 89%

(RTTNews) - Gene therapy company uniQure N.V. (QURE) announced Tuesday 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 (RMAT) 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 Biologics License Application (BLA) submission under the Accelerated Approval pathway, avoiding the need for an additional pre-submission study.

The FDA also agreed that the composite Unified Huntington's Disease Rating Scale (cUHDRS) may be used as an intermediate clinical endpoint and that reductions in neurofilament light chain (NfL) measured in cerebrospinal fluid (CSF) may serve as supportive evidence of therapeutic benefit in the application for accelerated approval.

The company said it initiated BLA readiness activities and looks to further engage with the FDA in the first half of 2025 to discuss its statistical analysis plan and the technical CMC requirements.

The FDA granted uniQure RMAT designation for AMT-130 in May 2024, stating that preliminary clinical data from the ongoing Phase I/II studies indicate AMT-130 has the potential to address unmet medical needs for the treatment of Huntington's disease.

In July 2024, uniQure presented interim data at 24 months showing durable, dose-dependent slowing of disease progression based on the cUHDRS of treated patients compared to a propensity-weighted natural history. These data also showed reductions in CSF NfL, a measure of neurodegeneration, in treated patients at 24 months compared to baseline.