Sarepta Therapeutics announced in a recent press release that the prescribing information for delandistrogene moxeparvovec (Elevidys) had been updated to emphasize stronger safety protections for patients with Duchenne muscular dystrophy (DMD). The new labelling reinforces careful liver monitoring, infection precautions and limits on who can receive the therapy.
For families, the changes mean more structured follow-up and clearer instructions but also a pause in treatment availability for nonambulatory individuals until additional research is completed.
Sarepta said the FDA-approved update adds a boxed warning about acute serious liver injury and acute liver failure, conditions that have occurred in real-world use and can be life-threatening. Because of these risks, delandistrogene moxeparvovec is no longer indicated for nonambulatory patients.
Sarepta plans to begin a study using an enhanced sirolimus immunosuppressive regimen to address liver complications with the goal of reopening access to those patients if the FDA agrees.
Read more about treatment and care for DMD
“We want to thank the FDA for their thorough and collaborative review. Completion of the safety labeling change for Elevidys will ensure that families and healthcare professionals have clear information, supported by a Medication Guide, to help understand these updates and guide treatment decisions,” said Louise Rodino-Klapac, president of research & development and technical operations at Sarepta.
The revised label also expands instructions for doctors. It includes updated steroid schedules before and after infusion, plus recommendations for weekly liver checks for three months. Patients must remain within reach of appropriate medical care for at least two months after receiving the infusion. The updated warnings explain that people with existing liver problems or active infections face higher risk and should delay treatment.
A new precaution describes increased susceptibility to serious infections because the therapy requires corticosteroids, and sometimes other immunosuppressants. Serious respiratory infections, including some fatal cases, have been reported. Families are urged to ensure immunizations are current before treatment and to avoid infusion during any active illness. The label also reiterates the contraindication for patients with deletions in exon 8 and/or exon 9 of the DMD gene.
Additional cautions outline risks of myocarditis, infusion reactions and immune-mediated myositis, some of which have been severe or fatal. The therapy is not recommended for patients with high levels of anti-AAVrh74 antibodies because these may interfere with how it works. Common side effects include vomiting, nausea, fever, thrombocytopenia, liver injury and elevated troponin-I.
For patients and caregivers, these updates offer more detailed guidance. While the added warnings may feel unsettling, they aim to improve safety for the more than 1,100 people already treated worldwide and those who may receive delandistrogene moxeparvovec in the future.
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