uniQure shares surge on FDA accelerated approval pathway accord

Shares of uniQure N.V. (NASDAQ: NASDAQ:QURE ) experienced a notable 100% increase in pre-market trading today following the company's announcement of an agreement with the U.S. Food and Drug Administration (FDA) regarding the Accelerated Approval pathway for its gene therapy product, AMT-130, targeting Huntington’s disease.

The gene therapy leader, uniQure, has confirmed its alignment with the FDA on pivotal aspects of AMT-130's Accelerated Approval pathway. The FDA's Center for Biologics Evaluation and Research (CBER) has been in collaborative discussions with uniQure, leading to this consensus. "Our alignment reflects the strength of our data and collaborative discussions with the staff and senior management at FDA’s CBER," expressed Walid Abi-Saab, M.D., the chief medical officer of uniQure. He further stated the significance of this milestone for those affected by Huntington’s disease, emphasizing the company's commitment to delivering this potentially transformative therapy.

The agreement was reached during a Regenerative Medicine Advanced Therapy (RMAT) Type B meeting with the FDA in late November. The FDA concurred that current Phase I/II study data, benchmarked against a natural history external control, could form the primary foundation for a Biologics License Application (BLA) under the Accelerated Approval pathway. This arrangement potentially eliminates the need for additional studies before the BLA submission. The FDA also acknowledged that cUHDRS, an intermediate clinical endpoint, and reductions in neurofilament light chain (NfL) levels in cerebrospinal fluid (CSF) could be used as supportive evidence of AMT-130's therapeutic benefit.

uniQure received RMAT designation for AMT-130 from the FDA in May 2024, indicating the therapy's potential to meet the unmet medical needs in Huntington’s disease treatment. Interim data presented in July 2024 showed promising results, with treated patients demonstrating a durable, dose-dependent deceleration of disease progression and reduction in CSF NfL levels after 24 months.

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Source: Investing.com