uniQure Faces FDA Setback, Lawsuits Over AMT-130 as Fabry Program Advances

uniQure received final meeting minutes from the U.S. Food and Drug Administration on a pre Biologics License Application meeting for AMT-130, in which the FDA stated that current Phase I/II data are unlikely to provide the primary evidence needed to support a Biologics License Application. The company plans to seek a follow up meeting in the first quarter of 2026.

A securities fraud class action lawsuit has been filed against uniQure N.V. in the U.S. District Court for the Southern District of New York, covering investors who bought ordinary shares between September 24, 2025 and October 31, 2025. The lawsuit focuses on disclosures related to AMT-130 data and expectations for an accelerated Biologics License Application filing for Huntington's disease. Multiple law firms launched securities class actions alleging the company misrepresented FDA approval status and pivotal study timelines.

In recent weeks, uniQure has faced heightened scrutiny after the FDA Commissioner raised concerns about the safety and perceived lack of benefit of its AMT-130 Huntington's disease gene therapy. The stock has logged a 10.2% decline over the past 30 days and is down 33.3% in recent weeks.

Updated preliminary safety and exploratory efficacy data from 11 patients in the Phase I/IIa AMT-191 trial for Fabry disease showed elevated a galactosidase A activity across all three dose cohorts and a manageable safety profile. One Grade 3 liver enzyme elevation resolved after corticosteroid treatment. The company paused new dosing to review safety findings.

Details of the AMT-191 program were presented at the 22nd Annual WORLDSymposium, including a poster and a planned oral presentation. Additional information is available through the clinical trial listing NCT06270316, and AMT-191 has Orphan Drug and Fast Track designations from the U.S. Food and Drug Administration.

At US$20.50 versus a consensus target of about US$53.54, the share price sits roughly 62% below analyst expectations. Analysts have trimmed their fair value estimate for uniQure to about $53.54 from roughly $54.42. This reflects slightly higher discount rate assumptions and modest tweaks to growth and margin expectations after recent neutral Street research coverage.

The discount rate was adjusted marginally higher to 7.41% from 7.41%, reflecting a very small change in risk assumptions. Revenue growth was updated to 159.79% from 159.34%, indicating a very small adjustment in long term growth expectations. Net profit margin was revised modestly lower to 15.70% from 16.05%, implying a slightly leaner long run profitability assumption.

uniQure develops treatments for patients suffering from rare and other devastating diseases in the United States. The company is also expanding its clinical pipeline with new studies in refractory temporal lobe epilepsy and Fabry disease.