FDA Accuses uniQure of Pushing "Distorted" and "Manipulated" Data for "Failed" AMT-130 Drug

Hagens Berman investigating uniQure following securities class action lawsuit over misleading clinical trial data

Published on Mar. 10, 2026

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The U.S. Food and Drug Administration and Department of Health and Human Services have publicly criticized uniQure N.V., accusing the company of providing "distorted" and "manipulated" data for its AMT-130 gene therapy drug candidate for Huntington's disease, which the FDA says "failed" to meet approval standards. This follows the filing of a securities class action lawsuit alleging uniQure misled investors about the status of its AMT-130 drug development and interactions with the FDA.

Why it matters

The FDA's unprecedented public rebuke of uniQure raises serious questions about the company's integrity and transparency in its dealings with regulators. This could have significant implications for uniQure's future drug development efforts and the company's standing with investors, who have already suffered steep losses following the revelations.

The details

According to reports, a senior FDA official said uniQure had "lied" and "mischaracterized" the agency's requests regarding how to compare AMT-130 to a placebo in clinical trials. The FDA official also stated that the data from AMT-130 testing were "flawed" and indicated the drug candidate "didn't meet the bar for agency approval." An HHS official went further, accusing uniQure of putting out "misleading statements" about the therapy.

  • On March 5, 2026, The Wall Street Journal published a report on the FDA and HHS criticism of uniQure.
  • The next day, STAT reported on the FDA's accusations that uniQure pushed "distorted" and "manipulated" clinical data for a "failed" therapy.
  • This follows a series of reports beginning November 3, 2025, that have driven uniQure's share price down nearly 84%.
  • A securities class action lawsuit was filed against uniQure, with a lead plaintiff deadline of April 13, 2026.

The players

uniQure N.V.

A biopharmaceutical company developing gene therapies, including the AMT-130 drug candidate for Huntington's disease that is at the center of the controversy.

Food and Drug Administration (FDA)

The U.S. federal agency responsible for regulating and supervising the safety of drugs and medical devices, which has publicly criticized uniQure's handling of the AMT-130 clinical trial data.

Department of Health and Human Services (HHS)

The U.S. federal executive department responsible for protecting the health of all Americans, whose officials have accused uniQure of lying and making misleading statements about the AMT-130 drug.

Hagens Berman

A national shareholder rights law firm that is investigating uniQure and has filed a securities class action lawsuit on behalf of investors.

Reed Kathrein

The Hagens Berman partner leading the firm's investigation into uniQure.

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What they’re saying

“The core of our investigation is the potential delta between what uniQure told investors about its interactions with the FDA and the details revealed in the recent Type A meeting minutes.”

— Reed Kathrein, Hagens Berman partner (Hagens Berman)

What’s next

The judge in the securities class action lawsuit will decide on April 13, 2026 whether to certify the class of uniQure investors.

The takeaway

The FDA's unprecedented public rebuke of uniQure's handling of the AMT-130 clinical trial data raises serious concerns about the company's integrity and transparency in its dealings with regulators. This could have significant implications for uniQure's future drug development efforts and its standing with investors, who have already suffered steep losses.