uniQure Secures FDA Agreement on Accelerated Approval Pathway for Huntington’s Disease Gene Therapy AMT-130
FDA Agreement:
The FDA has agreed with uniQure on key elements for an accelerated approval pathway for AMT-130, a gene therapy for Huntington's disease13.
Phase I/II Data:
The FDA will allow uniQure to use data from ongoing Phase I/II studies, compared to natural history external controls, as the primary basis for a Biologics License Application (BLA)13.
Clinical Endpoints:
The composite Unified Huntington’s Disease Rating Scale (cUHDRS) will be used as an intermediate clinical endpoint, and reductions in neurofilament light chain (NfL) levels in cerebrospinal fluid will serve as supportive evidence for therapeutic benefit13.
Regulatory Milestone:
This agreement marks a significant regulatory milestone, potentially reducing development time and costs by years and de-risking the approval pathway2.
Future Plans:
uniQure plans to engage further with the FDA in the first half of 2025 to discuss statistical analysis plans and technical CMC requirements, and will also meet with the European Medicines Agency to align on a path to approval135.
Impact:
This development not only advances uniQure's position but also provides a clear path for other drug developers working on Huntington's disease treatments5.
Sources:
1. https://www.biospace.com/drug-development/uniqure-clears-path-to-accelerated-approval-for-huntingtons-gene-therapy
2. https://www.stocktitan.net/news/QURE/uni-qure-announces-alignment-with-fda-on-key-elements-of-accelerated-do5298k3aq15.html
3. https://uniqure.gcs-web.com/news-releases/news-release-details/uniqure-announces-alignment-fda-key-elements-accelerated