FDA hits Sarepta with liver warning labelling for its DMD drug Elevidys
As well as the new safety warnings for the Duchenne muscular dystrophy gene therapy, the biopharma company also faced a new, more restrictive licence for the treatment.
The US Food and Drug Administration (FDA) has mandated a raft of new labelling changes for the only approved gene therapy for Duchenne muscular dystrophy (DMD).
The addition of its most prominent safety warning means that packaging for Sarepta Therapeutics’ Elevidys (delandistrogene moxeparvovec-rokl) will now feature a ‘Boxed Warning’ on risks of acute serious liver injury (ALI) and acute liver failure (ALF).
The FDA’s other labelling revisions for Elevidys include expanded guidance for prescribers, including a modified pre- and post-infusion oral corticosteroids regimen, and enhanced monitoring recommendations on a weekly basis for three months post-infusion.
Moreover, there are additional warnings for patients of the potential for Elevidys’ to increase their susceptibility to serious infections due to immunosuppression.
Additionally, the FDA tightened the gene therapy’s indication, limiting it to ambulatory DMD patients aged four years old and over.
These actions follow reports in June 2025 of three cases of fatal acute liver failure in non-ambulatory patients treated with Elevidys. In response, Sarepta voluntarily paused distribution of Elevidys for this indication.
For dosing to resume in non-ambulatory DMD patients, FDA has mandated that Sarepta conduct a postmarketing observational study of an enhanced sirolimus immunosuppressive regimen to address the risk of ALI and ALF.
Completion of the safety labelling change for Elevidys will ensure that families and healthcare professionals have clear information… to help understand these updates and guide treatment decisions”
Louise Rodino-Klapac, PhD, Sarepta’s President of Research & Development and Technical Operations, said: “We want to thank the FDA for their thorough and collaborative review. Completion of the safety labelling 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.”
Questions from the FDA over Elevidys’ safety placed Sarepta at the centre of the regulator’s own staffing turmoil earlier this year.
The day the FDA authorised resuming of Elevidys shipments in some patients, its Center for Biologics Evaluation and Research (CBER) head Dr Vinay Prasad resigned, after less than three months in the role. He subsequently returned two weeks later.
The US regulator faced further personnel issues this month when it was forced to replace George Tidmarsh as Center for Drug Evaluation and Research (CDER) Director following his resignation after concerns arose over his personal conduct. His position was taken by Dr Richard Pazdur.
