uniQure gains FDA priority review for Huntington's gene therapy
The agency set a May 1, 2027 action target for AMT-130 and is tentatively planning an advisory committee meeting.
Adrian Kessler
A May 1, 2027 FDA decision target is now set as uniQure N.V. NASDAQQURE announced on Monday that its AMT-130 Huntington's disease gene therapy application has secured priority review. The company seeks accelerated approval based on 36-month data compared with an external control.
The FDA accepted the biologics licence application for ifezuntirgene inilparvovec, also called AMT-130, according to uniQure's Nov. 2 release. The agency is tentatively planning an advisory committee meeting, but the company said the date and timelines have yet to be finalized. The application and FDA acceptance letter were not independently reviewed.
uniQure is a specialist gene therapy developer based in Lexington, Massachusetts, and Amsterdam, with a pipeline targeting severe neurological and inherited diseases. Acceptance starts substantive review; it does not authorize sales or establish that the evidence supports approval.
Before the announcement, shares closed at US$24.83 on Nasdaq on Oct. 30, down 1.86 per cent, according to Google Finance's NASDAQQURE quote at 4 p.m. EDT. That close precedes the new regulatory milestone and provides no measure of Monday's reaction.
Priority Review Leaves The Evidence Standard Intact
The FDA's priority-review guidance sets a six-month action goal, compared with ten months for standard review. The designation does not change the scientific or medical standard for approval, or the quality of evidence required.
Read more: Palvella Therapeutics gains FDA priority review with February decision target
Huntington's disease causes progressive motor, cognitive and behavioural deterioration. In its Nov. 2 announcement, uniQure said no approved therapy delays onset or slows progression, making disease modification the proposed treatment's central commercial distinction.
The Huntington's development field includes Wave Life Sciences Ltd. NASDAQWVE, whose SELECT-HD announcement describes allele-selective mutant huntingtin lowering with WVE-003. AMT-130 instead uses a gene-silencing construct administered once through MRI-guided neurosurgery directly into the striatum.
uniQure says the application relies on the earlier three-year analysis against a propensity score-matched external control from the Enroll-HD natural-history database. Its Nov. 2 release reports that the broader early-stage programme has dosed 51 patients, with low-dose, high-dose and sham-procedure groups in the original U.S. study.
Later Results And A Confirmatory Trial Remain In View
A Sept. 29 update expanded the three-year high-dose analysis to 15 patients. Their composite Unified Huntington's Disease Rating Scale score declined by 0.28 points, compared with 1.39 points in the updated external control; the nominal p value was 0.005.
At four years, the high-dose analysis included 12 patients. The composite scale comparison was not statistically significant, with p=0.144, although Total Functional Capacity showed a nominal p value of 0.008.
uniQure said the September update followed submission and was not part of the original application. It attributed some weakening of the four-year comparison to missing data and survivor bias in the external control.
Five high-dose participants experienced treatment-related serious central nervous system inflammation events, all of which resolved, according to that update. A suicide in a low-dose patient was assessed as unrelated to treatment.
The Nov. 2 release says FDA and uniQure have aligned on key elements of a randomized confirmatory trial enrolling approximately 200 patients. It will allocate treatment against standard care in a two-to-one ratio, with Total Functional Capacity measured at three years as the primary endpoint.
"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," uniQure chief executive Matt Kapusta said in the release.
In its July 29 financial update, uniQure reported US$810.3 million in cash, equivalents and current investment securities at June 30. Management projected that those resources would fund operating expenses into 2030.
Patient screening is planned by Dec. 31, 2026, ahead of the May 1, 2027 FDA action target, when the agency is due to act on the accelerated-approval application.
