UniQure seeks FDA approval for Huntington's gene therapy after reversal

Dutch biotech uniQure has submitted a marketing application to the FDA seeking accelerated approval for AMT-130, which could become the first approved gene therapy to slow Huntington's disease progression. The submission follows a turbulent year of regulatory friction, during which the FDA initially rejected uniQure's three-year dataset and demanded a controversial trial involving invasive sham brain surgeries. Following the departures of FDA vaccine chief Vinay Prasad and Commissioner Marty Makary, the agency reversed its stance in June 2026, allowing uniQure to apply using its existing Phase 1/2 data.

UniQure FDA submission for AMT-130

• ▪The Dutch gene therapy developer uniQure requested a priority review from the U.S. Food and Drug Administration for its AMT-130 gene therapy application, which could shorten the review period to six to eight months
• ▪The Dutch gene therapy developer uniQure submitted a marketing application for its experimental Huntington's disease gene therapy, AMT-130, to the medicines regulator of Great Britain
• ▪The Dutch gene therapy developer uniQure submitted a marketing application to the U.S. Food and Drug Administration seeking accelerated approval of its experimental Huntington's disease gene therapy, AMT-130

FDA leadership turnover

• ▪Marty Makary resigned as the chief commissioner of the U.S. Food and Drug Administration in May 2026
• ▪Kyle Diamantas took over as the acting commissioner of the U.S. Food and Drug Administration in May 2026
• ▪Vinay Prasad, the former top regulator of vaccines and gene therapies at the U.S. Food and Drug Administration, left the agency in April 2026

Regulatory reversal on approval pathway

• ▪The U.S. Food and Drug Administration reversed its previous stance in June 2026, agreeing to accept uniQure's existing Phase 1/2 trial data for an accelerated approval review of AMT-130
• ▪Prior to its June 2026 reversal, the U.S. Food and Drug Administration had informed uniQure that its existing clinical data was insufficient to support a marketing application for AMT-130
• ▪An unnamed senior official at the U.S. Food and Drug Administration previously characterized uniQure's experimental gene therapy, AMT-130, as a "failed product" during discussions with journalists

Clinical trial design dispute

• ▪In March 2026, the U.S. Food and Drug Administration requested that uniQure conduct a new double-blind trial for AMT-130 that included a sham surgery control group
• ▪The U.S. Food and Drug Administration's demand for a new trial with a placebo-surgery control group faced criticism because it required patients to undergo invasive brain procedures without receiving the gene therapy

Three-year efficacy data

• ▪The Dutch gene therapy developer uniQure compared its AMT-130 clinical trial results against an external natural history database called Enroll-HD instead of an in-study control group
• ▪The Dutch gene therapy developer uniQure's marketing application is backed by three-year clinical trial data showing that AMT-130 slowed Huntington's disease progression compared to patients in an external database
• ▪The Dutch gene therapy developer uniQure expects to present four-year follow-up data for its AMT-130 gene therapy by the end of September 2026

Analyst outlook on approval

• ▪Leerink Partners analyst Joseph Schwartz stated that avoiding a sham surgery control procedure in the confirmatory study removes a meaningful ethical and execution hurdle for uniQure
• ▪William Blair analyst Sami Corwin noted that some regulatory risk remains for uniQure's AMT-130 application due to ongoing leadership changes at the U.S. Food and Drug Administration

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