UniQure will submit its AMT-130 gene therapy BLA in Q3 2026 after FDA rescinded demands for additional clinical data, accepting the three-year Phase I/II analysis for accelerated approval of the Huntington’s treatment. The FDA and uniQure will align on a confirmatory study design using a concurrent standard-of-care control before filing.
The FDA has rescinded its November 2024 refusal and now agrees that the three-year analysis from uniQure’s Phase I/II study provides an adequate data package to support a Biologics License Application for AMT-130 under accelerated approval.
UniQure will file the AMT-130 BLA in Q3 2026 and work with the FDA to finalize a confirmatory trial design that may use a concurrent control on standard-of-care therapy instead of a sham procedure before submission.
The company is conducting two multicenter, dose-escalating Phase I/II trials to evaluate safety, tolerability and efficacy, with the FDA agreeing to compare treated patients against matched controls drawn from the Enroll-HD observational dataset.
