The US Food and Drug Administration has updated a warning about the safety of Elevidys (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular dystrophy (DMD), and restricted its use. This follows reports earlier this year of fatal acute liver failure in certain non-ambulatory, male, paediatric patients taking the drug. Following the incidents, the developer, Sarepta Therapeutics Inc, voluntarily paused distribution of Elevidys to this patient group. This has now been formalised in a notice issued on 14 November.
