uniQure Secures FDA Priority Review for Huntington’s Gene Therapy

Dutch gene therapy developer uniQure said the U.S. Food and Drug Administration (FDA) accepted its Biologics License Application (BLA) for ifezuntirgene inilparvovec, formerly known as AMT-130. The investigational gene therapy is intended to treat Huntington’s disease, a fatal neurodegenerative disorder with no approved disease-modifying treatments.

The FDA granted the application Priority Review and set a Prescription Drug User Fee Act (PDUFA) target action date of May 1, 2027. The application was submitted under the FDA’s Accelerated Approval pathway and is supported by data from a Phase I/II clinical study. The FDA said it may convene outside experts to discuss the application.

If approved, the therapy could become the first treatment to slow the progression of Huntington’s disease. The regulatory milestone follows an earlier uniQure trial update that initially raised doubts about the therapy’s efficacy.

Shares of uniQure (NASDAQ: QURE) rose approximately 2.5% in U.S. premarket trading after the announcement. The company plans to begin a larger confirmatory trial by the end of 2026 to verify the therapy’s clinical benefit.