Study‑Design Dispute Sparks Multiple Securities Suits Over uniQure’s AMT‑130

Regulatory Study Design Dispute Drives Multiple Securities Suits Against uniQure

Three U.S. plaintiffs’ firms announce class actions alleging uniQure misled investors about the regulatory status of its Pivotal Study for AMT‑130, the company’s lead Huntington’s disease gene therapy candidate. The complaints, filed on behalf of purchasers of uniQure ordinary shares between Sept. 24 and Oct. 31, 2025, assert that uniQure represented the trial design — including planned comparisons to the ENROLL‑HD external historical data set — as fully approved by the U.S. Food and Drug Administration when, the suits allege, it was not. Plaintiffs further claim company statements downplay the likelihood that uniQure would need to delay a Biologics License Application (BLA) or perform additional studies to support a submission.

The litigation centers on the company’s interactions with regulators and the design choices for a pivotal trial, a critical junction for gene therapy developers seeking licensure. Plaintiffs contend that uniQure’s public comments about the study and its allegedly “highly successful” results lacked a reasonable basis because the FDA had not fully signed off on the external comparator approach. The lawsuits argue that this regulatory uncertainty made delays to a BLA filing more likely than the company disclosed, potentially altering the programme’s development timetable and regulatory strategy.

uniQure operates in a sector where study design, endpoints and comparator selection can materially affect approval prospects for novel gene therapies. The suits underscore heightened legal and commercial scrutiny of how biotech companies describe regulatory interactions and trial methodologies when pursuing accelerated or landmark pathways. Observers say the outcome of these cases could influence how sponsors document and disclose the extent of FDA agreement on using external controls in pivotal trials for rare neurodegenerative conditions.

Lead Plaintiff Deadline and Firm Notices

Each firm — Rosen Law, Kessler Topaz Meltzer & Check and The Schall Law Firm — urges affected investors to preserve records and consider acting by an April 13, 2026 deadline to move for lead plaintiff status. The firms provide contact details for free consultations and note there is no cost to speak with counsel; Rosen Law also highlights its prior securities settlements record.

Clinical and Regulatory Context

AMT‑130 uses an adeno‑associated viral vector to deliver a microRNA targeting huntingtin expression, and ENROLL‑HD is a widely referenced external observational dataset for Huntington’s disease natural history. The dispute highlights ongoing regulatory debate over the acceptability and validation of external controls in pivotal trials for rare diseases and the operational risks sponsors face when relying on such comparators.