The first gene therapy for a deadly form of muscular dystrophy received preliminary U.S. approval despite concerns from some government scientists.
FILE - A sign for the Food and Drug Administration is displayed outside their offices in Silver Spring, Md., on Dec. 10, 2020. The first gene therapy for Duchenne muscular dystrophy received preliminary U.S. approval on Thursday, June 22, 2023, despite concerns from some government scientists about the treatment's ability to help boys with the inherited disease. The first gene therapy for a deadly form of muscular dystrophy received preliminary U.S.
The FDA OK'd the treatment only for children ages 4 and 5, based on study results showing the therapy helped produce a protein needed for muscle growth, which is missing in boys with the condition. The gene therapy had been studied in children up to age 7. The FDA said the increase in protein seen with the therapy, Elevidys, is “reasonably likely to predict” a benefit in patients 4 to 5 years old, who don’t have other preexisting complications.
Still, the FDA's outside experts voted narrowly in favor of making the gene therapy available on a preliminary basis, noting the deadly nature of Duchenne and the risk of delaying a potentially beneficial treatment. The vote was non-binding, but the FDA often uses such recommendations to bolster its decisions.
The shortcut approach has come under increasing scrutiny from academic researchers, government watchdogs, and congressional investigators. But the FDA has also faced pressure from patient groups to use that route more aggressively for debilitating diseases, approving a string of recent treatments for Alzheimer’s, Lou Gehrig’s disease and other conditions with few treatment options.
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