Regulatory Daily Summary: What Happened on November 4, 2026

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

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