uniQure N.V. announced on September 2, 2026, that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for the accelerated approval of ifezuntirgene inilparvovec (AMT-130) to treat Huntington’s disease. Concurrently, the company submitted a Marketing Authorisation Application (MAA) to the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) for the same therapy.
The company has requested priority review for the BLA. If granted, the FDA review cycle would be shortened to six months following the standard 60-day filing review period. The applications are supported by a three-year data analysis from the Phase I/II clinical study, which demonstrated a slowing of disease progression compared to a control group derived from the Enroll-HD natural history database.
AMT-130 is a gene therapy candidate designed to silence the huntingtin gene. It utilizes a proprietary miQURE platform and is administered via a single MRI-guided, convection-enhanced stereotactic neurosurgical procedure directly into the striatum.
The therapy has received Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA, as well as Fast Track designation. The company plans to present a four-year data analysis from the ongoing Phase I/II studies before the end of the current third quarter.
The Phase I/II program included a U.S. randomized study enrolling 26 patients, and a European open-label study enrolling 13 patients. Additional cohorts evaluated the therapy in combination with immunosuppression and in patients with lower striatal volumes.
