- uniQure N.V. announced the FDA accepted for review the BLA for ifezuntirgene inilparvovec, an investigational gene therapy for Huntington's disease submitted under the Accelerated Approval pathway, and granted it Priority Review.
- The PDUFA target action date is May 1, 2027.
- In its acceptance letter, the FDA stated it is tentatively planning to discuss the application at an Advisory Committee meeting; uniQure will be notified once the date and timelines are finalized.
- The BLA is supported by 36-month data from the Phase I/II study compared with a propensity score-matched external control from the Enroll-HD natural history database, previously announced September 24, 2025.
- The agreed confirmatory study is a randomized, standard-of-care trial of approximately 200 patients at a 2:1 ratio, with Total Functional Capacity at 36 months as the primary endpoint; U.S. patient screening is planned to begin by the end of 2026.
Quotes
“The FDA’s acceptance of our BLA for review marks an important milestone for the Huntington’s disease community, which has waited generations for a treatment that changes the course of this devastating disease”