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UniQure announces alignment with FDA on key elements of accelerated approval pathway for AMT-130

Yesterday, uniQure issued a press release announcing alignment with the US Food and Drug Administration (FDA) on key elements of an accelerated approval pathway for uniQure‘s AMT-130 program in Huntington’s disease. The key takeaways are as follows:

· The U.S. Food and Drug Administration (FDA) agrees that data from ongoing Phase I/II studies compared to a natural history external control may serve as the primary basis for a Biologics License Application (BLA) for Accelerated Approval

· FDA agrees that the composite Unified Huntington’s Disease Rating Scale (cUHDRS) may serve as an intermediate clinical endpoint for Accelerated Approval

You can read the full press release here.

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