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# uniQure Files BLA for Huntington’s Gene Therapy
- URL: https://www.fdaweb.com/uniqure-files-bla-for-huntingtons-gene-therapy/
- Published: 2026-09-02T12:00:00.000Z
- Updated: 2026-09-14T12:05:19.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5161724

uniQure has submitted a BLA seeking accelerated approval of ifezuntirgene inilparvovec (AMT-130), an investigational gene therapy for treating Huntington’s disease. The company’s submission requested a priority review.

The filing is based on a three-year data analysis from a Phase I/II clinical study of ifezuntirgene inilparvovec, compared to a propensity score-matched external control derived from the Enroll-HD natural history database. The company expects to present a four-year data analysis from clinical studies before the end of the current 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://fdaweb.com/login.php?sa=v&aid=D5160771&cate=&stid=%241%24Cx2.jV1.%24QhAcy0l5qEo4x%2FHMcFjrJ1&ref=fdaweb.com)).

After Prasad’s departure, [FDA reversed course](https://fdaweb.com/login.php?sa=v&aid=D5161325&cate=&stid=%241%24Vg2.4h1.%24nf30baXvohPWxHWakqd%2F41&ref=fdaweb.com) and told UniQure that data from its ongoing Phase I/II study could serve as the primary basis for a BLA. FDA also requested alignment on the design of a post-approval confirmatory study before the filing is submitted.

AMT-130 is described as 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.