FDA Clashes with uniQure Over Gene Therapy Study Design

Shareholder lawsuit alleges company misled investors about regulatory approval for clinical trial

Apr. 5, 2026 at 5:23pm by Ben Kaplan

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A national shareholder rights law firm is investigating uniQure N.V. (NASDAQ: QURE) following a series of rebukes from the FDA over the company's lead gene therapy candidate, AMT-130. This comes after the filing of a securities class action lawsuit alleging uniQure misrepresented its interactions with the FDA and used a pivotal study design that the agency had not approved.

Why it matters

The FDA's criticism of uniQure's study design and the company's alleged misrepresentations to investors raise concerns about the regulatory pathway for the gene therapy and the potential impact on the company's stock price and future prospects.

The details

The FDA official reportedly called AMT-130 a "failed therapy" and accused uniQure of "performing a distorted or manipulated comparison" instead of running a correct clinical study. The official disputed the company's claims about the agency's requirements for a sham surgery arm in the Phase III trial, stating the FDA "never agreed to accept this distorted comparison" using natural history as a control. The official also said AMT-130 is not eligible for streamlined rare-disease pathways because it is not an individualized treatment.

  • On March 5 and 6, 2026, media outlets reported on the FDA's criticism of uniQure.
  • 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 lead candidate AMT-130.

U.S. Food and Drug Administration (FDA)

The federal agency responsible for regulating and supervising the safety of food, drugs, and medical devices, including gene therapies.

Hagens Berman

A national shareholder rights law firm investigating the allegations against uniQure and leading the securities class action lawsuit.

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 investigation into the alleged claims against uniQure.

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What they’re saying

“The FDA official called uniQure's lead gene therapy candidate, AMT-130, a "failed therapy" and alleged that the company is "performing a distorted or manipulated comparison in the mind of FDA" instead of running a correct clinical study.”

— FDA Official

“The FDA official dismissed uniQure's ethical concerns regarding sham surgeries, accusing the company of mischaracterizing the agency's request. The official clarified that the FDA did not ask to "drill holes in skulls," but rather required "one to three nicks in the scalp" under minimal anesthesia.”

— FDA Official

“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.”

— FDA Official

What’s next

The judge in the securities class action lawsuit will decide on the lead plaintiff by the April 13, 2026 deadline.

The takeaway

The clash between uniQure and the FDA over the design of the pivotal clinical trial for AMT-130 highlights the challenges gene therapy companies face in navigating the regulatory approval process. The allegations of misrepresentation to investors also raise concerns about the company's transparency and communication with the public.