FDA tells uniQure early trial data insufficient for brain disorder therapy filing
FDA tells uniQure early trial data insufficient for brain disorder therapy filing
ReutersMon, March 2, 2026 at 12:37 PM UTC
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FILE PHOTO: Signage is seen outside of the Food and Drug Administration headquarters in White Oak, Maryland, U.S., August 29, 2020. REUTERS/Andrew Kell//File Photo
March 2 (Reuters) - UniQure said on Monday the U.S. health regulator has informed the company that data from an early- to mid-stage trial of its gene therapy for a brain disorder would not support a marketing application for the treatment.
The company's shares slumped nearly 44% in premarket trading.
UniQure said it received final minutes from a U.S. Food and Drug Administration meeting held on January 30 to discuss the gene therapy for Huntington's disease. The regulator concluded that the results from the studies — when compared to an external control — cannot serve as primary evidence of effectiveness required for approval.
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Huntington's disease is a fatal, inherited neurodegenerative disorder causing progressive breakdown of nerve cells in the brain.
The FDA recommended uniQure to run a new study that randomly assigns patients to get either the gene therapy or a sham surgery.
(Reporting by Puyaan Singh in Bengaluru; Editing by Shilpi Majumdar)
Source: “AOL Breaking”
