Hagens Berman Expands uniQure Probe After FDA Calls Study Design Distorted

The central dispute over AMT-130, uniQure's Huntington's disease gene therapy, is no longer confined to a regulatory disagreement. An FDA official's public declaration on March 5 and 6, 2026, that the Netherlands-based company was "performing a distorted or manipulated comparison in the mind of FDA" rather than running a correct clinical study has now drawn a securities class action, an expanded shareholder probe, and an eight-day deadline that will begin shaping the legal dimension of uniQure's path to market.

Hagens Berman, a shareholder rights firm, updated its investor notice on April 5 citing what it called extraordinary public FDA criticism, and is seeking lead plaintiffs in a securities class action covering the class period from September 24 through October 31, 2025, with a court-set deadline of April 13, 2026.

The regulatory dispute centers on what uniQure told investors about the company's study design and what the FDA says it actually agreed to. The FDA rejected uniQure's use of the ENROLL-HD external historical dataset as the primary control for AMT-130, and formally recommended that uniQure conduct a prospective, randomized, double-blind, sham surgery-controlled study instead. The distinction is consequential: a sham-surgery-controlled Phase III trial would require recruiting patients willing to undergo a placebo neurosurgical procedure, substantially extending timelines and increasing capital demands compared to the historical-control approach uniQure had communicated to investors as its regulatory framework.

An FDA official also described AMT-130 as a "failed therapy" during the March media call, language that went considerably beyond routine agency pushback on study design. The agency separately stated that uniQure's approach was "not eligible for streamlined, individualized rare-disease pathways," effectively closing the door on the faster regulatory route the company had publicly indicated it was pursuing.

uniQure executives have countered that the FDA's characterizations were taken out of context and that the company believes its study design and regulatory strategy remain defensible. The friction between those competing accounts is precisely what Hagens Berman argues constitutes securities exposure. The firm's probe asserts a consistent pattern: that uniQure misrepresented its interactions with the FDA and used a pivotal study design that it knew the agency had not approved.

For investors assessing the legal risk, the securities class action playbook here is familiar. Biotech stocks are acutely sensitive to regulatory pathway risk, and a law firm's expanded investigation after an FDA rebuke is a predictable structural response, not evidence of liability on its own. What distinguishes this case is that the negative commentary came not from a standard rejection letter but from an FDA official speaking directly to journalists at major outlets in early March, making the agency's position unusually public and creating a clearer basis for arguing that new material information entered the market.

The more durable question for uniQure is operational: a pivot to a sham-surgery-controlled randomized trial would reconfigure the company's capital requirements, enrollment timelines, and partnership negotiations. If forced to redesign or re-run pivotal study components, timeline and cost implications could be material and affect partner negotiations, capital needs, and stock valuation. The April 13 lead plaintiff deadline will resolve one near-term uncertainty; the study design question has no such clean horizon.