--- title: 'Regulatory Daily Summary: What Happened on November 4, 2026' headline: FDA gives uniQure’s Huntington’s gene therapy Priority Review description: The agency set a May 1, 2027, target date and provisionally plans an advisory committee meeting. url: https://thebench.bio/news/regulatory/2026-11-04 topic: regulatory date: '2026-11-04' covers: - '2026-11-04' date_published: '2026-11-04T12:02:38Z' date_modified: '2026-11-04T12:02:38Z' tags: - Industry - Regulatory sources: 2 publisher: TheBench.Bio --- # Regulatory Daily Summary: What Happened on November 4, 2026 **FDA gives uniQure’s Huntington’s gene therapy Priority Review** The agency set a May 1, 2027, target date and provisionally plans an advisory committee meeting. ## Today in brief The FDA accepted uniQure’s application for its Huntington’s disease gene therapy and granted Priority Review, setting a May 1, 2027, target date. [1] The agency provisionally plans an advisory committee meeting, while uniQure prepares a randomized confirmatory study. [1] ## FDA sets May 1, 2027, target date for uniQure gene therapy The FDA accepted uniQure’s application for ifezuntirgene inilparvovec, also known as AMT-130, through the accelerated approval pathway and granted Priority Review. [1] Priority Review shortens the FDA’s review goal but does not mean the therapy is approved. [1] The agency set May 1, 2027, as its target action date and provisionally indicated it plans to hold an advisory committee meeting. [1] The application draws on 36-month results from a Phase 1/2 study compared with an external group in the Enroll-HD natural-history registry. [1] A four-year comparison reported by European Biotechnology on November 3 found progression was 44% slower in 12 high-dose patients than among external controls. The difference did not reach statistical significance. [2] UniQure and the FDA agreed on key parameters for a randomized trial of about 200 patients, comparing the therapy with standard care and measuring Total Functional Capacity at 36 months. [1] The company plans to begin screening US patients before the end of 2026. [1] The therapy is given once through MRI-guided surgery into the brain’s striatum, where it uses microRNA to silence the huntingtin gene. [1] ## The day in numbers - **May 1, 2027**: FDA target action date for uniQure’s application [1] - **About 200**: Planned enrollment in the confirmatory trial [1] ## Also published - [What do midterms mean for Trump's pharma and health agenda?](), pharmaphorum - [FDA needs a roadmap for disease-agnostic drug development — a Perspective](), BioCentury - [UniQure gets an FDA decision date; Teva scraps brain drug study](), BioPharma Dive - [uniQure set for May FDA verdict on Huntington's gene therapy](), pharmaphorum ## Sources 1. [FDA to review uniQure’s BLA for Huntington’s gene therapy](), Pharmaceutical Technology, 2026-11-04 2. [AstraZeneca targets $1.5B in savings; Ayrmid bids for Karyopharm; Sanofi pays $125M to Novavax](), European Biotechnology, 2026-11-03 --- TheBench.Bio wrote this from the reporting cited above, with a language model drafting and editing. [1]: [2]: