Children's Hospital LA halts use of Sarepta's gene therapy for all patients

India Today

6 days ago

Children's Hospital Los Angeles has paused the use of Sarepta Therapeutics' gene therapy Elevidys for all patients with muscular dystrophy, following recent US Food and Drug Administration (FDA) actions that raised safety concerns.In a statement Monday, Children's Hospital LA said the pause has been communicated to affected patient families while it awaits any further determination by the FDA. The hospital is amongst more than 70 nationwide authorised to administer Elevidys. Five other major children's hospitals asked to comment on whether they planned to continue use of the gene therapy did not immediately respond, Reuters reported.advertisementThe decision, effective July 18, comes after the FDA asked Sarepta last week to voluntarily halt shipments of the treatment. The request followed the death of a muscular dystrophy patient who received a different, experimental gene therapy. Sarepta has so far refused to comply, stating it will "continue making the treatment available to ambulatory patients."
Elevidys was granted full FDA approval last year for children with Duchenne Muscular Dystrophy who can still walk, and received conditional approval for non-ambulatory patients. However, Sarepta suspended use in non-ambulatory cases in June, after two teenagers died of liver failure following treatment.The FDA's recent request reignited scrutiny of the therapy's safety profile. Shares of Sarepta, down about 90% year-to-date, fell 5% on Monday to close at $13.32 on Nasdaq.- EndsWith inputs from Reuters