Securities suits allege uniQure misrepresented FDA sign‑off for AMT‑130 pivotal study

Allegations over AMT‑130 pivotal design trigger cluster of suits

Main development: lawsuits say uniQure misled on FDA sign‑off of AMT‑130 study design

Three U.S. law firms are bringing nearly simultaneous securities class actions against uniQure N.V., alleging the Amsterdam‑based gene therapy developer misrepresented regulatory clearance of its pivotal study for AMT‑130, its lead Huntington’s disease candidate. The complaints, filed on behalf of investors who bought ordinary shares between Sept. 24, 2025 and Oct. 31, 2025, contend that uniQure falsely represented that the Pivotal Study design — notably its planned comparison to the ENROLL‑HD external historical data set — had full U.S. Food and Drug Administration approval. Plaintiffs assert those statements leave uniQure’s public comments about business prospects and regulatory timelines without a reasonable basis.

The suits further allege company executives downplay the likelihood that AMT‑130 will require additional studies or a delay to a Biologics License Application (BLA) submission despite allegedly strong Pivotal Study results. That contention strikes at a central question for gene therapy developers: how regulators will treat trials that incorporate external historical control datasets rather than randomized concurrent controls. Use of ENROLL‑HD — a large observational registry for Huntington’s disease — is under increased scrutiny across the biotech sector, and the outcome of these cases could influence how sponsors design pivotal programs and interact with regulators.

One of the complaints, Scocco v. uniQure N.V., et al., is docketed in the Southern District of New York (Case No. 1:26‑cv‑01124). Plaintiffs seek to hold uniQure and certain officers accountable for alleged misrepresentations and to recover investor losses once alleged corrective information emerges; the class is not yet certified. The litigation highlights both the legal risks tied to regulatory strategy in rare‑disease gene therapy and broader industry challenges in securing FDA agreement on alternatives to randomized control groups.

Other procedural and firm details

The Rosen Law Firm, Kessler Topaz Meltzer & Check and The Schall Law Firm all announce representations and urge potentially affected investors to preserve records and act promptly. Investors who wish to move to serve as lead plaintiff must do so by April 13, 2026; Kessler Topaz files provide a contact for counsel and a case webpage.

Rosen Law promotes its past settlement record, while Kessler Topaz and Schall Law emphasize free consultations and no obligation to inquire. Each firm provides telephone, email and online contact routes for prospective class members to assess eligibility and next steps.