uniQure gets priority review for Huntington’s gene therapy

The FDA has accepted uniQure’s BLA for ifezuntirgene inilparvovec (AMT-130), an investigational gene therapy for the treatment of Huntington’s disease submitted under the accelerated approval pathway.

The application has been granted priority review with a PDUFA date of May 1, 2027. In its acceptance letter, the FDA stated that they are tentatively planning to discuss the application at an advisory committee meeting. 

AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene, leveraging the company’s proprietary miQURE silencing technology. The therapeutic goal is to inhibit the production of the mutant protein. If approved, it will be the first genetic treatment for Huntington’s disease.

The BLA, which was submitted back in September, is supported by the previously announced 36-month data analysis from the phase 1/2 clinical study of AMT-130 compared to a propensity score-matched external control derived from the Enroll-HD natural history database. While the study showed a 44% slowing of Huntington’s disease progression compared with the external control, the result did not reach statistical significance.

uniQure says it has aligned with the FDA on key elements of a confirmatory study design. 

The BLA submission came after much back and forth between uniQure and the agency. The trouble began at a pre-BLA meeting in October 2025, when the agency said it did not consider data from the phase 1/2 studies of AMT-130 sufficient to support a BLA. In February 2026, FDA Commissioner Marty Makary appeared to reference and criticize the therapy during an interview with CNBC. Final Type A meeting minutes released in March confirmed the FDA’s position and strongly recommended a prospective, randomized, double-blind, sham surgery-controlled study. Such a trial would be particularly challenging for AMT-130, which is administered once directly into the brain through a complex neurosurgical procedure. The FDA reversed course in June, following the March resignation of CBER director Vinay Prasad and Makary’s May resignation, agreeing that three-year data from the phase 1/2 study could serve as the primary basis for a BLA.