The United States on Friday said it would add its most serious warning label to a gene therapy treatment for a debilitating muscle-wasting disease, and also limit its approved use.
The Food and Drug Administration said the decision to narrow the approved use of Elevidys—which is administered as an infusion to try and slow the progression of Duchenne muscular dystrophy—comes after two adolescent patients died of acute liver failure after receiving the treatment.
When they began the therapy, both of those patients were no longer able to walk. Prescription of it will now be limited to people who can walk and are older than four.
Duchenne muscular dystrophy is a rare, fatal disease that causes muscular deterioration, including the heart. Most patients with the devastating condition are boys with an average life expectancy of 28.
Over the summer Sarepta, the US-based biopharmaceutical giant behind the therapy, paused distribution of the treatment to non-ambulatory patients.
The FDA halted trials and announced a safety review in July—and asked Sarepta to stop all shipments of Elevidys. The company said it would continue providing the treatment to patients who could walk.
Also in July Europe’s Committee for Medicinal Products for Human Use recommended against marketing approval for Elevidys, saying data had failed to adequately demonstrate the treatment’s effect on movement abilities.
According to trade publications, debate over whether to approve the treatment had provoked tensions within the FDA itself, as parent and advocacy organizations desperately seek options to relieve suffering from the disease.
The new labeling will recommend precautions including weekly liver function monitoring for the first three months after treatment starts.
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