The US Food and Drug Administration (FDA) has accepted for review uniQure’s Biologics License Application (BLA) for ifezuntirgene inilparvovec, a gene therapy for Huntington’s disease, with a Prescription Drug User Fee Act target date of 1 May 2027 under Priority Review. The application was submitted under the Accelerated Approval pathway.
Backing the filing are 36-month results from a Phase I/II study, which were compared with an external control cohort taken from the Enroll-HD natural history database.
UniQure calls acceptance a milestone
“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,” said Matt Kapusta, CEO of uniQure.
Kapusta said the company believes ifezuntirgene inilparvovec has the potential to become the first disease-modifying treatment for Huntington’s disease. He added that the confirmatory trial remains a key priority, with screening anticipated to begin by year end and the study expected to be well underway by the time of potential approval.
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Regulatory designations already in hand
The FDA has already granted ifezuntirgene inilparvovec Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations, making it the first Huntington’s disease therapy to have received the first two of these.
FDA also issues potency guidance
In the same week, the FDA issued final potency guidance for cell and gene therapy manufacturers, outlining a comprehensive, science- and risk-based plan to ensure products meet potency standards. The guidance covers how manufacturers can verify product effectiveness from initial approval through post-market monitoring, and includes special considerations for products with short shelf lives, viral vector gene therapies and patient-specific treatments.
“Patients who receive cellular and gene therapies are often battling serious or life-threatening conditions, such as cancers and rare diseases, and they deserve the assurance that the product administered to them will perform as intended,” said Karim Mikhail, Director of the Center for Biologics Evaluation and Research.
The BLA acceptance arrives with the Priority Review clock set to run through spring 2027, keeping the therapy on an accelerated regulatory timetable.
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#BREAKING: The FDA has accepted @uniQure_NV's BLA for ifezuntirgene inilparvovec (AMT-130), an investigational gene therapy for Huntington disease, and granted it Priority Review. The agency set a target action date of May 1, 2027, under the PDUFA, and said it is tentatively…
A Huntington's gene therapy just got an FDA date 📅$QURE: FDA accepted the BLA for AMT-130 under accelerated approval, with Priority Review. Decision day: May 1, 2027. The case: 75% slower decline at 3 years in 12 high-dose patients, vs matched outside records. The catch: the… pic.twitter.com/5IvDxMBS15
🧬 FDA accepts $QURE’s BLA for AMT-130, its Huntington’s gene therapy, with Priority Review. PDUFA date: May 1, 2027. If approved, it would be the first disease-modifying treatment for Huntington’s. Full story 👇https://t.co/56JBzRswt0 pic.twitter.com/4U4aRdNmOj
