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.
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)
