Elevidys, a gene therapy for Duchenne Muscular Dystrophy (DMD) from Sarepta Therapeutics, is now available to ambulatory patients, and the U.S. Food and Drug Administration is suggesting that the voluntary hold be lifted. The FDA’s inquiry found that the gene therapy product had nothing to do with the 8-year-old boy’s death.
The story of Sarepta’s gene therapy, which started earlier this year after serious safety concerns and fatalities surfaced, has taken a new turn with the regulatory whiplash. The FDA intervened and requested that Sarepta halt all U.S. deliveries of the treatment for ambulatory patients after a third patient—a 51-year-old man who had been taking part in a Phase I study for the drug in limb-girdle muscular dystrophy, which also uses an AAV vector—died. The FDA then launched a probe after another death was reported on Friday.
However, in a succinct statement released Monday afternoon, the FDA suggested lifting the voluntary hold, allowing ambulatory individuals to keep taking Elevidys. Sarepta and Brazilian authorities concurred with the agency that the eight-year-old Brazilian patient’s most recent death was probably caused by a severe bout of influenza rather than the gene therapy product itself. an infection that is made worse by immunosuppression. – Read More: FDA Investigating Death of 8-Year-Old Brazilian Boy Who Received Elevidys –
A voluntary hold is still in effect for non-ambulatory patients, for whom the FDA has not yet issued any recommendations following two deaths connected to Elevidys.
