uniQure Announces FDA Acceptance and Priority Review of the Biologics License Application for ifezuntirgene inilparvovec for the Treatment of Huntington’s Disease
Key Highlights
- ➤FDA accepts uniQure’s BLA for ifezuntirgene inilparvovec with Priority Review
- ➤PDUFA target action date set for May 1, 2027
- ➤Confirmatory trial plans approximately 200 patients, with screening by end-2026
- ➤Potential approval could make ifezuntirgene inilparvovec Huntington’s first disease-modifying treatment
Expert Statements
Matt Kapusta, Chief executive officer of uniQure
“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”
Matt Kapusta, Chief executive officer of uniQure
“Backed by compelling data from our Phase I/II studies, we believe ifezuntirgene inilparvovec has the potential to become the first disease modifying treatment for Huntington’s disease.”
Matt Kapusta, Chief executive officer of uniQure
“With the FDA’s priority review underway, we will work in conjunction with the Agency, clinicians, and the Huntington’s community to support the review process.”
Matt Kapusta, Chief executive officer of uniQure
“The confirmatory trial remains a key priority, with screening anticipated to begin by year end and the study is expected to be well underway by the time of potential approval.”
~ If approved, ifezuntirgene inilparvovec will be the world’s first disease-modifying treatment for Huntington’s disease ~
LEXINGTON, Mass. and AMSTERDAM, Nov. 02, 2026 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for ifezuntirgene inilparvovec, 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 Prescription Drug User Fee Act (PDUFA) target action date of May 1, 2027.
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