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Will uniQure submit BLA for AMT-130 to FDA by mid-2025?
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uniQure press releases or FDA submission records
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uniQure Gains FDA Alignment on Accelerated Approval Pathway Using cUHDRS for Huntington's Gene Therapy AMT-130
Dec 10, 2024, 01:04 PM
uniQure ($QURE) announced that it has aligned with the U.S. Food and Drug Administration (FDA) on key elements of an Accelerated Approval pathway for its gene therapy candidate AMT-130 in Huntington’s Disease. The FDA agreed that data from the ongoing Phase I/II studies, when compared to a natural history external control, may serve as the primary basis for a Biologics License Application (BLA) submission under the Accelerated Approval program. Additionally, the composite Unified Huntington's Disease Rating Scale (cUHDRS) may serve as an intermediate clinical endpoint. The company has previously presented 24-month data showing superiority over natural history controls. This regulatory alignment is significant as it could expedite the approval process for AMT-130, potentially offering a much-needed treatment for patients with Huntington's Disease. The announcement led to a substantial increase in uniQure's stock price, which more than doubled in pre-market trading.
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