(Reuters) -UniQure, said on Monday the U.S. health regulator has deemed clinical data for its gene therapy for Huntington’s disease as inadequate, sending the Dutch company’s U.S.-listed shares plummeting over 68% in premarket trading.
The company’s shares had surged nearly 200% in September after reporting trial data that showed its gene therapy, AMT-130, slowed progression of the brain disorder by 75%.
“We are surprised by the (U.S. Food & Drug Administration’s) feedback, which is a drastic change from the guidance the FDA provided in November 2024,” said Matt Kapusta, UniQure’s CEO.
UniQure said it plans to urgently interact with the FDA to find a path forward for the timely accelerated approval of AMT-130.
(Reporting by Christy Santhosh in Bengaluru; Editing by Krishna Chandra Elur

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