FDA Says uniQure’s Huntington’s Gene Therapy Fell Short, Urges New Placebo-Controlled Study

A senior U.S. Food and Drug Administration (FDA) official said uniQure N.V.'s experimental gene therapy for Huntington's disease did not provide sufficient evidence that it slows disease progression and described the program as a failed product.

The FDA defended its request that uniQure run a new placebo-controlled study to demonstrate clinical benefit, saying the company must prove the therapy actually helps people with Huntington's disease.

Agency officials publicly criticized uniQure's trial design and, in comments that are uncommon for regulators, accused the Amsterdam-based company of misleading behavior in its interactions with federal health authorities.

The FDA's remarks come amid heightened scrutiny from lawmakers over recent rejections of rare-disease therapies and underscore that the agency is requiring additional, controlled data before it will consider approval of uniQure's treatment.

The FDA says the existing trial design did not provide sufficient evidence that uniQure's gene therapy slows Huntington's disease, so a new placebo-controlled study is needed to demonstrate clear clinical benefit.

The FDA official used that phrase to describe the current state of uniQure's program, meaning the data submitted so far did not show the treatment works well enough to support approval.

The FDA's request for more data delays approval and wider access until uniQure completes the requested study and demonstrates benefit; it does not directly address access through experimental or compassionate-use pathways.

Yes. Federal health officials publicly criticized uniQure and accused the company of misleading behavior in its communications with regulators, according to the agencies' comments.

Reuters

UniQure's , trial design for its gene therapy for Huntington's disease did not offer sufficient proof that the drug slowed disease progression, a top U.S. Food and Drug Administration official said on Wednesday, while defending the agency's call for a new ‌placebo-controlled study.

Benzinga

An official from the U.S. Food and Drug Administration (FDA) defended the agency's request for a new placebo-controlled study for uniQure N.V.'s (NASDAQ: QURE) experimental gene therapy for Huntington's disease.

Reuters

A senior FDA ‌official called Uniqure's experimental Huntington's disease ⁠treatment a "failed product" in a conference call with media members ‌on ⁠Thursday, casting further doubt on ⁠the prospects of its ⁠experimental gene ⁠therapy.

CNBC

UniQure needs to run another study to prove its gene therapy "actually helps people with Huntington's disease," a senior FDA official said. The official said UniQure is the "latest company to make a failed therapy for Huntington's patients.

WSJ

Federal health officials, facing criticism from lawmakers for recent rejections of rare-disease drugs, attacked an Amsterdam-based biotech company seeking approval of a Huntington's disease treatment and accused it of lying. The public criticism of Uniqure by officials at the Food and Drug Administr...

Zacks Investment Research

uniQure stock plunges 32% in a week after the FDA says AMT-130 phase I/II Huntington's disease data are not sufficient as primary evidence for a potential marketing application.

PRNewsWire

SAN FRANCISCO, March 10, 2026 /PRNewswire/ -- National shareholder rights law firm Hagens Berman is updating its investigation into uniQure N.V. (NASDAQ: QURE) following an unprecedented public rebuke of the company by federal health officials.

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