uniQure's Huntington's gene therapy BLA gets FDA priority review

uniQure said the FDA accepted its Biologics License Application for ifezuntirgene inilparvovec (AMT-130) in Huntington's disease, submitted under the Accelerated Approval pathway in November 2026.¹ The agency granted the application Priority Review, setting a PDUFA target action date of May 1, 2027.¹ The filing rests on 36-month data from 12 high-dose patients measured against a propensity score-matched external control group.¹

The company also reported additional follow-up data from its ongoing Phase I/II studies, cutoff June 30, 2026. Among 12 high-dose patients followed for 48 months, the composite Unified Huntington's Rating Scale, the primary endpoint for that analysis, showed a 44% slowing of disease progression versus the external control, not reaching statistical significance (p=0.144), while Total Functional Capacity, the endpoint planned for the confirmatory study, showed a 61% slowing with nominal p=0.008.¹ A separate 36-month analysis covering all 15 high-dose patients found an 80% slowing on the composite scale (nominal p=0.005) and a 67% slowing on Total Functional Capacity (nominal p=0.011).¹ Across 51 treated patients overall, the therapy was described as generally well tolerated with a manageable safety profile.¹

On the confirmatory study required for accelerated approval, uniQure said it reached alignment with the FDA on key design elements: a randomized trial against standard of care enrolling about 200 patients in a 2:1 ratio, with Total Functional Capacity measured at 36 months as the primary endpoint.¹ Patient screening is planned to begin in the U.S. by the end of 2026.¹ Separately, the U.K.'s MHRA validated a Marketing Authorization Application for the same therapy in the third quarter of 2026.¹

On finances, the company held $768.4 million in cash, cash equivalents and investment securities as of September 30, 2026, which it said should fund operating expenses into 2030.¹ Net loss for the quarter was $61.2 million, or $0.88 per share, compared with $80.5 million, or $1.38 per share, a year earlier.¹