uniQure Aligns with FDA for BLA Submission and Accelerated Approval of AMT-130 in Phase I/II Huntington's Disease Studies

Dec 10, 2024, 12:18 PM

uniQure has announced that the FDA has agreed that data from its ongoing Phase I/II studies for AMT-130, a treatment for Huntington's Disease, may serve as the primary basis for a Biologics License Application (BLA) submission under the Accelerated Approval pathway. This alignment with the FDA is seen as a significant step forward for the company, as it establishes the use of natural history external control data in the review process. The FDA's flexibility in review standards for gene therapy applications is also noted, with emphasis on the importance of biomarkers like mHTT and NfL being deprioritized in this context. The announcement has generated positive sentiment among investors, drawing comparisons to previous successful approvals in the biotech sector.