Hagens Berman updates uniQure probe after FDA criticisms as lead plaintiff deadline nears

Hagens Berman said it is updating its investigation into uniQure N.V. (NASDAQ: QURE) following what it described as “extraordinary rebukes” from Food and Drug Administration (FDA) officials. The update comes as a securities class action lawsuit has been filed seeking to represent investors who purchased or otherwise acquired uniQure ordinary shares between September 24, 2025, and October 31, 2025 (the “Class Period”). The firm also reminded investors of an April 13, 2026 deadline to seek appointment as Lead Plaintiff in the pending case.

FDA criticism highlighted in March 2026 media reports

On March 5 and 6, 2026, media outlets including The Wall Street Journal, CNBC and CNN reported on a call with reporters in which an FDA official criticized uniQure. The official reportedly referred to uniQure’s lead gene therapy candidate, AMT-130, as a “failed therapy,” and alleged the company was “performing a distorted or manipulated comparison in the mind of FDA” rather than conducting a correct clinical study.

Key points cited by the FDA official

• Sham surgery mischaracterization: The FDA official dismissed uniQure’s ethical concerns about sham surgeries, alleging the company mischaracterized the agency’s request. The official reportedly said the FDA did not ask for “drill holes in skulls,” but instead required “one to three nicks in the scalp” under minimal anesthesia.
• Dispute over prior agreement: While uniQure CEO Matt Kapusta described the sham surgery requirement as a “drastic change” from previous guidance, the FDA official reportedly said the agency “never agreed to accept this distorted comparison” using natural history as a comparator.
• Rare-disease pathway eligibility: The FDA official reportedly disputed AMT-130’s eligibility for streamlined rare-disease pathways, stating it is not an individualized treatment.

The firm said the reports followed the filing of the securities class action.

Claims in the securities class action

Hagens Berman partner Reed Kathrein said the pending securities class action alleges a “consistent pattern” in which uniQure misrepresented its interactions with the FDA and used a pivotal study design that it knew the agency had not approved.

Summary of allegations cited in the complaint

The lawsuit, Scocco v. uniQure N.V., et al. (S.D.N.Y.), alleges that during the Class Period (Sept. 24, 2025 – Oct. 31, 2025), defendants failed to disclose the following:

• No regulatory consensus: That the FDA had not approved the use of the ENROLL-HD external historical data set as a primary control for AMT-130.
• Hidden requirements: That uniQure downplayed the necessity of a sham-controlled surgery arm for Phase III—a requirement the FDA now claims was never waived.
• Timeline deception: That defendants misled investors regarding the timing of a Biologics License Application (BLA), which was described as “unclear” once the lack of FDA agreement was revealed on November 3, 2025, after which the stock fell 49%.

Lead Plaintiff deadline: April 13, 2026

Hagens Berman said investors who purchased uniQure ordinary shares during the Class Period and suffered losses have until April 13, 2026 to ask the Court to appoint them as Lead Plaintiff.

The firm encouraged investors to review the allegations on its uniQure case page and said it is also providing a video summary of the allegations.

Whistleblower information

The firm also noted that individuals with non-public information regarding uniQure should consider their options to help in the investigation or take advantage of the SEC Whistleblower program. It said whistleblowers who provide original information may receive rewards totaling up to 30 percent of any successful recovery made by the SEC.

Company and firm background

Hagens Berman described itself as a global plaintiffs’ rights complex litigation firm focused on corporate accountability. The firm said its team has secured more than $2.9 billion in this area of law.