Sarepta Therapeutics announced a voluntarily pause on all shipments of the Duchenne muscular dystrophy (DMD) gene therapy Elevidys (delandistrogene moxeparvovec) effective July 22, announced a recently published press release.
Elevidys is designed to address the underlying cause of DMD by enabling the production of microdystrophin in skeletal muscle. Despite promising results from clinical trials such as the ENDEAVOR study — which showed that patients receiving Elevidys experienced significant functional improvements — recent reports of two cases of fatal acute liver failure in teenage patients with DMD treated with Elevidys have raised serious safety concerns.
The exact mechanisms leading to fulminant liver failure in these patients remain unclear.
Both deaths occurred in individuals who were unable to walk without assistance (nonambulatory patients), prompting Sarepta Therapeutics to temporarily halt shipments to nonambulatory patients and pause the Phase 3 ENVISION clinical trial, while continuing shipments to ambulatory patients.
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Sarepta later agreed to the FDA’s request to add a black box warning for acute liver injury and acute liver failure to the Elevidys label.
In a new development, Sarepta has paused shipments to all patients in order to complete the appropriate safety labeling process.
“As a patient-centric organization, the decision to voluntarily and temporarily pause shipments of ELEVIDYS was a painful one, as individuals with Duchenne are losing muscle daily and are in need of disease-modifying options,” said Doug Ingram, chief executive officer of Sarepta. “It is important for the patients we serve that Sarepta maintains a productive and positive working relationship with the FDA, and it became clear that maintaining that relationship required this temporary suspension while we address any questions the FDA may have and complete the ELEVIDYS label supplement process.”
Other complications observed in patients receiving Elevidys include myocarditis (inflammation of the heart muscle, which can lead to heart failure), myositis (skeletal muscle inflammation) and infusion-related reactions such as anaphylaxis.
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