FDA Criticizes uniQure's Gene Therapy, Lawsuit Filed

Hagens Berman Updates Probe After FDA Rebuke, April 13 Deadline Nears

Apr. 7, 2026 at 4:33pm by Ben Kaplan

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A photorealistic studio still-life image featuring a shattered glass vial or beaker set against a clean, monochromatic background, symbolizing the failure of uniQure's gene therapy candidate and the regulatory challenges faced by the company.The FDA's rebuke of uniQure's gene therapy candidate raises doubts about the company's regulatory strategy and transparency with investors.San Francisco Today

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

Why it matters

The FDA's criticism of uniQure's clinical trial design and data comparisons raises significant questions about the company's interactions with regulators and the viability of its lead drug candidate. The pending lawsuit alleges uniQure misled investors about its progress with the FDA, which could impact the company's future and investor confidence in the biotech sector.

The details

The FDA official called AMT-130 a "failed therapy" and accused uniQure of performing a "distorted or manipulated comparison" instead of running a proper clinical study. The agency disputed uniQure's ethical concerns over "sham surgeries," stating it only required "one to three nicks in the scalp" under minimal anesthesia. The official also said AMT-130 was ineligible for streamlined rare-disease approval pathways.

  • On March 5 and 6, 2026, media outlets reported on the FDA's rebuke of uniQure.
  • The securities class action lawsuit was filed in the Southern District of New York.

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

The CEO of uniQure.

Reed Kathrein

The Hagens Berman partner leading the investigation into the alleged claims 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 criticism of uniQure's clinical trial design and data comparisons raises significant questions about the company's interactions with regulators and the viability of its lead drug candidate. The pending lawsuit alleges uniQure misled investors, which could impact the company's future and investor confidence in the biotech sector.