UniQure AMT-130 Leading the Way for More Gene Silencing Drugs

On March 2, 2026, uniQure announced that it has received final meeting minutes from the U.S. Food and Drug Administration (FDA) following a Type A meeting held on January 30, 2026 to discuss AMT-130, its investigational gene therapy for Huntington’s disease (HD). uniQure says they were told by the FDA that their current data are insufficient for the FDA to approve AMT-130. This is disappointing news for the HD community.

Jan. 26, 2026 The Huntington’s disease (HD) community has shown its collective power over the past two months, rallying together to advocate for the continued advancement of AMT-130, uniQure’s investigational gene therapy, in the United States. This sustained advocacy effort appears to have contributed to the scheduling of uniQure’s Type A meeting with the Food and Drug Administration (FDA) to discuss the US regulatory pathway for AMT-130. 

On January 22, people living with Huntington’s disease (HD), alongside their families and supporters, gathered outside the U.S. Food and Drug Administration (FDA) campus in Silver Spring, Maryland, to deliver a clear message: the HD community cannot wait. Nearly 48,000 people signed two Change.org petitions urging the FDA to provide a fair and equitable review AMT-130. The petitions were formally submitted off-site and shared with FDA leadership, amplifying the urgent call for regulatory flexibility and meaningful engagement with the HD community. 

Bi-Monthly Advocacy Newsletter: Visit HDSA.org/Takeaction to read the New Bi-Monthly Advocacy Newsletter for updates.