FDA's Rigid Approach Adds Uncertainty To uniQure's Accelerated Pathway For Huntington's Gene Therapy

Author: Vandana Singh | November 04, 2025 02:25pm

The U.S. Food and Drug Administration (FDA) on Monday provided feedback to uniQure N.V. (NASDAQ:QURE) regarding its investigational gene therapy for Huntington's disease.

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uniQure believes that the FDA currently disagrees that the data from the Phase 1/2 studies of AMT-130, in comparison to an external control, as per the prespecified protocols and statistical analysis plans shared with the FDA in advance of the analyses, may be adequate to provide the primary evidence in support of a BLA submission.

Also Read: FDA Outlines Process To Speed Up Rare Disease Therapy Approvals

Analyst Take

William Blair highlighted that the key difference between this meeting and the company's prior meetings with the FDA is that Nicole Verdun was not present. Vinay Prasad was also not in the meeting.

The analyst downgraded uniQure from Outperform to Market Perform.

Investors should note that Verdun, the director of the FDA's Center for Biologics Evaluation and Research, was placed on administrative leave and escorted out of the agency.

Analyst Sami Corwin noted that the FDA raised no issues with the previously agreed pathway when the company submitted its final Statistical Analysis Plan after Dr. Verdun's exit.

Corwin added that while management remains focused on securing an expedited path for AMT-130, the timing of its BLA submission is now uncertain.

The FDA's commentary on AMT-130 suggests that the agency is being more stringent in regulating gene and cell therapy programs for rare diseases.

The update could put negative pressure on other gene/cell therapy companies that previously aligned on single-arm trials, including Neurogene Inc. (NASDAQ:NGNE), Cabaletta Bio Inc. (NASDAQ:CABA), Kyverna Therapeutics Inc. (NASDAQ:KYTX), and Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT).

However, since the agency's concern centers on the SAP not being submitted before the trial began, William Blair sees minimal read-through risk for the firms under its coverage.

QURE Price Action: uniQure shares were down 10.89% at $30.55 at the time of publication on Tuesday, according to Benzinga Pro data.

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