The U.S. Food and Drug Administration (FDA) has updated the prescribing information for Elevidys (delandistrogene moxeparvovec) for Duchenne muscular dystrophy (DMD).
DMD is a rare genetic, neuromuscular condition characterized by progressive muscle wasting. Symptoms of DMD include progressive weakness and atrophy of both skeletal and heart muscle. Early signs may include delayed ability to sit, stand, or walk and difficulties learning to speak. DMD is caused by genetic changes in the DMD gene.
Delandistrogene moxeparvovec is adeno-associated virus vector-based gene therapy indicated for the treatment of patients ages 4 years and older with DMD.
The new label now includes a boxed warning for the risk of acute serious liver injury and acute liver failure. The non-ambulatory indication has been removed from the Indication and Usage section of the prescribing information. The manufacturers of delandistrogene moxeparvovec, Sarepta, expects to initiate a study of enhanced sirolimus immunosuppressive regimen to address the risk of acute serious liver injury and acute liver failure so that dosing may resume for non-ambulatory patients.
Expanded guidance also includes a modified pre- and post-infusion oral corticosteroids regimen, and enhanced monitoring recommendations on a weekly basis for three months post-infusion. A new Warnings and Precaution regarding increased susceptibility to serious infections due to immunosuppression.
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To learn more about DMD and other rare musculoskeletal conditions, visit https://checkrare.com/diseases/musculoskeletal-diseases/
