LEXINGTON, Mass. and AMSTERDAM - uniQure N.V. (NASDAQ: NASDAQ:QURE), a gene therapy company, has reached an agreement with the U.S. Food and Drug Administration (FDA) regarding an Accelerated Approval pathway for its experimental gene therapy, AMT-130, for the treatment of Huntington’s disease. This development is a significant step toward providing a new treatment option for patients with this genetic neurodegenerative disorder.
Under the agreement, data from ongoing Phase I/II studies, compared to a natural history external control, may be used as the primary basis for a Biologics License Application (BLA) submission for AMT-130. This potentially eliminates the need for additional pre-submission studies. The FDA also concurred that the composite Unified Huntington’s Disease Rating Scale (cUHDRS) could serve as an intermediate clinical endpoint, with reductions in neurofilament light chain (NfL) in cerebrospinal fluid (CSF) providing supportive evidence of therapeutic benefit.
The FDA granted uniQure Regenerative Medicine Advanced Therapy (RMAT) designation for AMT-130 in May 2024, recognizing its potential to address unmet medical needs in Huntington’s disease treatment. Interim data presented in July 2024 indicated that AMT-130 could slow disease progression based on cUHDRS and reduce CSF NfL levels.
The company is conducting two multi-center, dose-escalating, Phase I/II clinical studies to evaluate the safety, tolerability, and exploratory efficacy of AMT-130. The U.S. study involves 26 patients with early manifest Huntington’s disease, while the European Phase Ib/II study has enrolled 13 patients. An additional cohort is being recruited to explore the therapy in combination with immunosuppression.
Huntington’s disease affects approximately 70,000 people in the U.S. and Europe, with no approved therapies currently available to delay onset or slow progression. uniQure aims to engage further with the FDA in the first half of 2025 to discuss the statistical analysis plan and technical requirements for the BLA submission.
This announcement is based on a press release statement from uniQure Inc. and reflects the company's progress in the development of AMT-130 as a potential treatment for Huntington’s disease.
In other recent news, uniQure N.V. has initiated a Phase I/II clinical trial for its investigational treatment AMT-162, targeting ALS caused by SOD1 mutations. The FDA has granted Orphan Drug status and Fast Track designation to the therapy. Furthermore, the FDA has awarded Orphan Drug Designation to uniQure's investigational gene therapy AMT-191 for Fabry disease treatment.
In addition, the company has seen significant developments in its clinical portfolio. The FDA has granted Orphan Drug Designation to uniQure's investigational gene therapy, AMT-191, for Fabry disease treatment. Meanwhile, uniQure's Huntington's Disease gene therapy, AMT-130, has been producing compelling data.
Raymond (NS:RYMD) James resumed coverage on uniQure, assigning an Outperform rating, highlighting the company's robust clinical portfolio and financial runway extending to the end of 2027. H.C. Wainwright and Stifel have also maintained a Buy rating on uniQure.
Finally, uniQure has undergone significant organizational restructuring, including a 65% reduction in its workforce and the sale of its manufacturing facility in Lexington, Massachusetts. Genezen has acquired uniQure's commercial gene therapy operations in Lexington, enhancing its global gene therapy development capabilities. These are the recent developments in the company's operations.
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