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# FDA Backs Accelerated Pathway for UniQure Huntington's Therapy
- URL: https://www.fdaweb.com/fda-backs-accelerated-pathway-for-uniqure-huntingtons-therapy/
- Published: 2026-06-17T12:00:00.000Z
- Updated: 2026-09-14T13:41:21.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5161325

FDA has reversed course and has told UniQure that data from its ongoing Phase I/II study of AMT-130 could serve as the primary basis for a BLA seeking accelerated approval for the investigational Huntington's disease gene therapy. The regulatory feedback was provided during a recent Type B meeting with FDA, according to the company. UniQure said it now plans to submit a BLA for AMT-130 during the third quarter.

Earlier this year, UniQure was tangled in a dispute with the agency over then-CBER director **Vinay Prasad**’s insistence on a new randomized trial, saying such designs are necessary in diseases like Huntington’s where outcomes can vary widely and subjective endpoints may be influenced by expectations ([see earlier story](https://www.fdaweb.com/fda-official-pushes-back-in-uniqure-dispute/)).

Under the agency's new guidance, a three-year analysis from the Phase I/II clinical program would be acceptable as the primary evidence package supporting an accelerated approval application. FDA also requested alignment on the design of a post-approval confirmatory study before the filing is submitted.

According to uniQure, the agency is considering a confirmatory trial that would use a concurrent standard-of-care control group rather than a sham-surgery control arm. The company said it expects to finalize details of the study design with FDA prior to the BLA submission.

"This reflects the outcome we have worked toward throughout our continued regulatory engagement with FDA," said Chief Executive Officer **Matt Kapusta** in a statement.

The company's clinical program includes U.S. and European Phase I/II studies evaluating low- and high-dose administration of AMT-130 delivered directly into the striatum through MRI-guided neurosurgery. The proposed filing would rely on data from treated patients compared with a propensity score-matched external control group derived from the Enroll-HD natural history database under a pre-specified statistical analysis plan.

AMT-130 is an investigational one-time gene therapy designed to lower production of the mutant huntingtin protein that causes Huntington's disease, a rare inherited neurodegenerative disorder characterized by progressive motor, cognitive, and psychiatric decline. No therapies are currently approved to slow disease progression.