FDA Rebukes uniQure Over 'Distorted' Data in Gene Therapy Trial

Hagens Berman updates investigation into QURE stock plunge after FDA criticism of company's clinical study design

Apr. 4, 2026 at 3:19pm by Ben Kaplan

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National shareholder rights law firm Hagens Berman is updating its investigation into uniQure N.V. (NASDAQ: QURE) following a series of extraordinary rebukes by Food and Drug Administration (FDA) officials over the company's lead gene therapy candidate, AMT-130. The investigation follows the filing of a securities class action lawsuit seeking to represent investors who purchased or otherwise acquired uniQure ordinary shares between September 24, 2025, and October 31, 2025.

Why it matters

The FDA's public criticism of uniQure's clinical study design and data presentation raises serious concerns about the company's transparency and regulatory strategy, potentially impacting investor confidence and the future of its lead drug candidate.

The details

The FDA official reportedly called AMT-130 a 'failed therapy,' alleging that the company is 'performing a distorted or manipulated comparison in the mind of FDA' instead of running a correct clinical study. Key revelations include the FDA dismissing uniQure's ethical concerns over 'sham surgeries' and denying the company had prior agreement on using external historical data as a comparator. The official also disputed AMT-130's eligibility for streamlined rare-disease pathways.

  • On March 5 and 6, 2026, media outlets reported on the FDA's call with reporters criticizing uniQure.
  • The securities class action lawsuit was filed in September 2025, covering the period from September 24, 2025 to October 31, 2025.
  • Investors have until April 13, 2026 to ask the Court to appoint them as Lead Plaintiff in the pending securities class action.

The players

uniQure N.V.

A biopharmaceutical company developing gene therapies, including the lead candidate AMT-130.

Food and Drug Administration (FDA)

The U.S. government agency responsible for regulating and supervising the safety of food, drugs, and medical devices.

Hagens Berman

A global plaintiffs' rights complex litigation firm representing investors and whistleblowers in cases of corporate wrongdoing.

Matt Kapusta

CEO of uniQure, who described the FDA's sham surgery requirement as a 'drastic change' from previous guidance.

Reed Kathrein

The Hagens Berman partner leading the firm's investigation of the alleged claims in the pending litigation against uniQure.

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What they’re saying

“While uniQure CEO Matt Kapusta described the sham surgery requirement as a 'drastic change' from previous guidance, the FDA official pushed back, stating the agency 'never agreed to accept this distorted comparison' using natural history as a comparator.”

— Matt Kapusta, CEO, uniQure

What’s next

Investors in uniQure (QURE) have until April 13, 2026 to ask the Court to appoint them as Lead Plaintiff in the pending securities class action lawsuit.

The takeaway

The FDA's public rebuke of uniQure's clinical study design and data presentation raises serious questions about the company's transparency and regulatory strategy, potentially impacting investor confidence and the future of its lead drug candidate. This case highlights the importance of clear communication and alignment between pharmaceutical companies and the FDA during the drug development process.