The company’s one-time IV treatment delivers a replacement gene for the one that is mutated in boys with the condition.
The Food and Drug Administration approval provides a new option for some patients with Duchenne muscular dystrophy, a rare muscle-wasting disease that causes weakness, loss of mobility and early death. It almost always affects males.
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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