FDA sets May 2027 review date for uniQure's Huntington's gene therapy

On November 2, 2026, the U.S. FDA accepted uniQure's application for its Huntington's disease gene therapy AMT-130, setting a decision deadline of May 1, 2027. The decision under the accelerated approval pathway follows a standoff where a senior regulator previously labeled the therapy a "failed product" after a four-year trial missed progression targets. UniQure plans to screen patients by late 2026 for a 200-patient confirmatory trial.

FDA review of uniQure's AMT-130

• ▪The U.S. Food and Drug Administration is reviewing uniQure's AMT-130 under its accelerated approval pathway based on early- to mid-stage trial data from 51 treated patients compared to external controls
• ▪The U.S. Food and Drug Administration's decision to review AMT-130 ended a standoff during which a senior agency official previously characterized the treatment as a "failed product."
• ▪The U.S. Food and Drug Administration is considering holding a meeting of outside expert advisors to discuss uniQure's AMT-130 application
• ▪The U.S. Food and Drug Administration accepted uniQure's application for its Huntington's disease gene therapy AMT-130, setting a decision review date of May 1, 2027

Stock market reaction and trial history

• ▪Following the FDA review acceptance announcement on November 2, 2026, U.S.-listed shares of uniQure rose 2.5% in premarket trading
• ▪Months prior to the review acceptance, uniQure disclosed that AMT-130 failed to slow disease progression in a four-year clinical trial, causing its stock to fall sharply