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

Pending Securities Class Action Filed Against Biotech Firm Amid Regulatory Rebuke

Mar. 12, 2026 at 8:06pm

Got story updates? Submit your updates here. ›

The U.S. Food and Drug Administration and Department of Health and Human Services have publicly criticized uniQure N.V., accusing the biotech firm of providing "distorted" and "manipulated" data for its AMT-130 gene therapy drug candidate for Huntington's disease, which federal officials say "failed" to meet approval standards. This comes amid a pending securities class action lawsuit filed on behalf of investors who purchased uniQure shares between September 24, 2025 and October 31, 2025.

Why it matters

The FDA's unprecedented public rebuke of uniQure raises serious questions about the company's transparency and integrity in its dealings with regulators, as well as the viability of its lead drug candidate. This could have major implications for uniQure's future, including potential regulatory action, legal liability, and investor confidence.

The details

According to reports, a senior FDA official stated that the data from uniQure's testing of AMT-130 was "flawed" and indicated the drug candidate "didn't meet the bar for agency approval." The official also accused a uniQure executive of "mischaracterizing" the FDA's requests regarding how to compare the therapy to a placebo. An HHS official went further, stating that the company had "lied" and put out "misleading statements." This comes after a series of reports since November 2025 that have driven uniQure's share price down nearly 84%.

  • On March 5, 2026, The Wall Street Journal published a report on the FDA and HHS officials' criticism of uniQure.
  • The next day, STAT reported on the FDA's accusations that uniQure pushed "distorted" and "manipulated" data for its "failed" AMT-130 therapy.
  • The reports follow the filing of a securities class action lawsuit against uniQure on behalf of investors who purchased shares between September 24, 2025 and October 31, 2025.

The players

uniQure N.V.

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

U.S. Food and Drug Administration (FDA)

The federal agency responsible for regulating and supervising the safety of drugs and medical devices, which has publicly criticized uniQure's conduct regarding the AMT-130 drug.

U.S. Department of Health and Human Services (HHS)

The federal executive department responsible for protecting the health and well-being of Americans, whose officials have also accused uniQure of lying and making misleading statements.

Hagens Berman

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

Reed Kathrein

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

Got photos? Submit your photos here. ›

What’s next

The judge in the pending securities class action lawsuit will decide on April 13, 2026 whether to certify the class of investors seeking to represent all those who purchased uniQure shares during the relevant period.

The takeaway

The FDA's unprecedented public rebuke of uniQure's conduct regarding its AMT-130 drug candidate has severely damaged the company's credibility and raised serious questions about its transparency and integrity in dealing with regulators. This could have far-reaching consequences for uniQure, including potential regulatory action, legal liability, and a lasting impact on investor confidence in the company.