uniQure N.V. Shares Surge 100%

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

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

The agreement was finalized during a Regenerative Medicine Advanced Therapy (RMAT) Type B meeting with the FDA in late November. The FDA agreed that current Phase I/II study data, compared to a natural history external control, could serve as the primary basis for a Biologics License Application (BLA) under the Accelerated Approval pathway, potentially bypassing the need for further studies prior to BLA submission. The FDA also acknowledged that cUHDRS, an intermediate clinical endpoint, along with reductions in neurofilament light chain (NfL) levels in cerebrospinal fluid (CSF), could support 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 address unmet medical needs in treating Huntington’s disease. Interim data released in July 2024 showed promising results, with treated patients demonstrating a durable, dose-dependent slowing of disease progression and reductions in CSF NfL levels after 24 months.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.