**uniQure Reaches Agreement with FDA on Accelerated Approval Pathway for AMT-130 in Huntington’s Disease**

On December 10, 2024, uniQure N.V. (NASDAQ: QURE) disclosed in a press release that it has successfully concluded discussions with the U.S. Food and Drug Administration (FDA) regarding critical elements of the Accelerated Approval pathway for AMT-130. The agreement signals progress towards submitting a Biologics License Application (BLA) for AMT-130 through this expedited pathway.

As outlined in the press release, uniQure will be hosting an investor call and webcast commencing at 8:30 a.m. Eastern Time on the same date to elaborate on these developments. To access the virtual event, interested parties can visit the Events and Presentations section of uniQure’s website at https://www.uniqure.com/investors-media/events-presentations. Additionally, a replay of the event will be available for 90 days post the initial broadcast.

The key highlights of the agreement with the FDA include the acceptance of data from ongoing Phase I/II studies compared to an external natural history control as the primary foundation for the BLA submission under the Accelerated Approval pathway for AMT-130. The FDA also acknowledged the composite Unified Huntington’s Disease Rating Scale (cUHDRS) as an intermediate clinical endpoint for Accelerated Approval. Furthermore, reductions in neurofilament light chain (NfL) measured in cerebrospinal fluid (CSF) can serve as supporting evidence of therapeutic benefit in the application for accelerated approval.

In her statement, Jeannette Potts, Chief Legal and Compliance Officer at uniQure, expressed satisfaction with the alignment reached with the FDA, emphasizing the significance of the milestone for the Huntington’s disease community. She mentioned that this agreement places uniQure on a swift and efficient path to delivering potentially life-altering therapy to individuals affected by this debilitating neurodegenerative disorder.

The regulatory progress for AMT-130 began with the RMAT designation granted by the FDA in May 2024, recognizing the therapy’s potential to address unmet medical needs in Huntington’s disease. Subsequent interim data presented in July 2024 demonstrated promising results, including a dose-dependent slowing of disease progression based on the cUHDRS and reductions in CSF NfL in treated patients compared to baseline.

UniQure is conducting Phase I/II clinical studies to evaluate the safety, tolerability, and efficacy signals of AMT-130 in Huntington’s disease. Through these studies, the company aims to address the urgent medical requirements of individuals grappling with this disorder. The company is also working on a pipeline of gene therapies for other severe diseases like refractory temporal lobe epilepsy, ALS, Fabry disease, and more.

The press release contained forward-looking statements based on management’s beliefs and available information. These forward-looking statements elaborated on the Company’s ongoing clinical trials, regulatory discussions, BLA-readiness plans, therapeutic benefits of AMT-130, and future engagement with regulatory bodies. The Company advised caution with undue reliance on these statements due to inherent risks and uncertainties associated with clinical development and regulatory processes.

Investors can access further details and updates through uniQure’s website, www.uniQure.com.

Contact information for uniQure:
– For Investors: Chiara Russo, Direct: 617-306-9137, Mobile: 617-306-9137, [email protected]
– For Media: Tom Malone, Direct: 339-970-7558, Mobile: 339-223-8541, [email protected].

This article was generated by an automated content engine and was reviewed by a human editor prior to publication. For additional information, read uniQure’s 8K filing here.

About uniQure

uniQure N.V. develops treatments for patients suffering from rare and other devastating diseases. It offers HEMGENIX that has completed Phase III HOPE-B pivotal trial for the treatment of hemophilia B. The company also develops AMT-130, a gene therapy that is in Phase I/II clinical study for the treatment of Huntington's disease.