> ## Content Index
> Fetch the complete content index at: https://www.fdaweb.com/llms.txt
> Use this file to discover other available public pages before exploring further.

# FDA Shifts Stance on AMT-130 BLA Data
- URL: https://www.fdaweb.com/fda-shifts-stance-on-amt-130-bla-data/
- Published: 2025-11-03T12:00:00.000Z
- Updated: 2026-09-14T15:25:35.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5160150

Gene therapy company uniQure says that FDA indicated during a recent pre-BLA meeting that the agency may no longer view the company’s Phase 1-2 data for its Huntington’s disease (HD) therapy AMT-130, analyzed against an external control, as sufficient to serve as primary evidence to support a planned submission.

The company says that while final meeting minutes have not yet been received, the feedback marks a “key shift” from prior communications over the past year, during which the agency had suggested the current data could form the basis for an accelerated approval filing. As a result, the timeline for a BLA submission is now unclear, it says.

In September, the company reported favorable data from the study. The study met its prespecified primary endpoint, with high-dose AMT-130 demonstrating a statistically significant (75%) slowing of disease progression as measured by the composite Unified Huntington’s Disease Rating Scale at 36 months compared to a propensity score-matched external control, according to the company.

“We are surprised by the FDA’s feedback at the recent pre-BLA meeting, which is a drastic change from the guidance provided in November 2024,” CEO Matt Kapusta is quoted in a release as saying. “We remain fully committed to working with the FDA to determine the best path forward.”