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uniQure Announces Alignment with FDA on Key Elements of Accelerated Approval Pathway for AMT-130 in Huntington’s Disease
uniQure has 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. This follows the RMAT designation granted in May 2024 and promising interim data from July 2024 showing durable, dose-dependent slowing of disease progression.
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. This follows the RMAT designation granted in May 2024 and promising interim data from July 2024 showing durable, dose-dependent slowing of disease progression.
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