Uniqure, Inc.

UniQure's gene therapy AMT-130 for Huntington's disease may qualify for FDA accelerated approval, based on ongoing trial data compared to an external control group. The FDA suggested using a known scale for Huntington's progression and neurofilament light chain reductions as evidence of effectiveness. UniQure's stock doubled following this news.

UniQure aligned with FDA on accelerated approval pathway for Huntington’s disease gene therapy AMT-130, using Phase I/II studies and composite Unified Huntington’s Disease Rating Scale as primary basis for Biologics License Application (BLA).

Founded in 1967 by Marjorie Guthrie to combat Huntington’s disease, the organization continues her mission by uniting the community to support affected families and fight HD.

FDA agrees on Accelerated Approval pathway for AMT-130 using Phase I/II data vs. natural history control, with cUHDRS as an intermediate endpoint. uniQure initiates BLA readiness activities for Huntington's disease therapy.

uniQure reached an agreement with the FDA on key elements for an Accelerated Approval pathway for AMT-130, their Huntington's disease treatment. The FDA agreed that data from ongoing Phase I/II studies compared to natural history external control can serve as the primary basis for a Biologics License Application (BLA), eliminating the need for additional pre-submission studies. The FDA accepted the composite Unified Huntington's Disease Rating Scale (cUHDRS) as an intermediate clinical endpoint and acknowledged that reductions in neurofilament light chain (NfL) in cerebrospinal fluid may support evidence of therapeutic benefit.

uniQure's shares surged 100% in pre-market trading due to an FDA agreement on Accelerated Approval for its gene therapy AMT-130 targeting Huntington’s disease. The FDA's CBER collaboration led to this alignment, potentially eliminating the need for additional studies before a Biologics License Application submission.

uniQure N.V. secured FDA agreement on Accelerated Approval pathway for AMT-130, allowing Phase I/II study data, compared to natural history, as primary BLA submission basis. FDA also accepted cUHDRS as an intermediate endpoint and neurofilament light chain reductions as supportive evidence.