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# UniQure Seeking Accelerated Approval Path for Gene Therapy
- URL: https://www.fdaweb.com/uniqure-seeking-accelerated-approval-path-for-gene-therapy/
- Published: 2024-07-09T12:00:00.000Z
- Updated: 2026-09-14T14:34:57.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5157314

UniQure says it will meet soon with FDA to present updated interim data, including up to 24 months of follow-up data from 29 treated patients, from its ongoing U.S. and European Phase 1-2 clinical trials of gene therapy AMT-130 for treating Huntington’s disease. The company says the Type B meeting request will discuss potential expedited clinical development pathways and accelerated approval.

The company says the updated data show that AMT-130 “achieved statistically significant, dose-dependent, and durable evidence of potential therapeutic benefit.” Patients receiving high-dose AMT-130 showed 80% slowing of disease progression at 24 months compared to a propensity score-weighted external control, it says. The therapy also achieved statistically significant lowering of biomarker cerebrospinal fluid (CSF) neurofilament light protein (NfL) compared to baseline at 24 months.

“We are very pleased with these new data demonstrating a statistically significant, dose-dependent slowing of the progression of Huntington’s disease and lowering of NfL in the CSF at 24 months,” a company release says. “We believe this is the first clinical trial of any investigational medicine for Huntington’s disease to show evidence of a potential long-term clinical benefit and reduction of a key marker of neurodegeneration. Moreover, given the one-time administration of AMT-130, we are in a unique position to continue accumulating longer-term patient outcomes from the Phase I/II studies to support the emerging therapeutic benefit.”