FDA Pushes uniQure Huntington Therapy Forward, Analyst Brushes Off Softening Signals

The U.S. Food and Drug Administration has accepted uniQure NV’s (NASDAQ:QURE) Biologics License Application (BLA) for ifezuntirgene inilparvovec, an investigational gene therapy for Huntington’s disease submitted under the Accelerated Approval pathway.

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The application has been granted Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of May 1, 2027.

In its acceptance letter, the FDA stated it is tentatively planning to discuss this application at an Advisory Committee meeting.  The company will be notified once the date and timelines have been finalized.

Clinical Evidence and Confirmatory Study Design

The BLA is supported by the previously announced 36-month data analysis from the Phase 1/2 study of ifezuntirgene inilparvovec, compared to a propensity score-matched external control derived from the Enroll-HD natural history database, as previously announced on Sept. 24, 2025.

The gene therapy company and FDA have aligned on key elements of the confirmatory study design.

The study is designed as a randomized, standard-of-care trial enrolling approximately 200 patients in a 2:1 ratio of ifezuntirgene inilparvovec to standard of care, with Total Functional Capacity (TFC) as the primary endpoint at 36 months.

The company plans to initiate patient screening in the U.S. by the end of 2026.

Analyst Assessment and Long-Term Efficacy Outlook

William Blair sees the acceptance of the BLA as an important milestone for ifezuntirgene inilparvovec as it further removes regulatory risk.

Analyst Lachlan Hanbury-Brown wrote that the additional 48-month results from the ongoing Phase 1/2 study of AMT-130 in Huntington’s disease continue to show a positive clinical benefit, especially around the more important regulatory endpoint of TFC, which increased separation from the external control group from 3 to 4 years following treatment.

While efficacy in the high-dose group as measured by the cUHDRS softened from year three to year four and thus could point to a weakening of treatment effect, analyst Hanbury-Brown sees concerns from this endpoint as overdone given several caveats in the analysis, and the fact that it is not the primary endpoint of the confirmatory study or the basis for the current BLA.

William Blair reiterates an Outperform rating for uniQure.

QURE Price Action: uniQure shares were down 2.98% at $24.09 at the time of publication on Monday, according to Benzinga Pro data.

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