Sarepta confirms FDA approval of updated Elevidys prescribing information

Sarepta (SRPT) Therapeutics announced an update to the prescribing information for Elevidys, which it identifies as “the only approved gene therapy for Duchenne muscular dystrophy.” As previously disclosed, the Elevidys label now includes several key updates, including: 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 Company expects to quickly commence a study of an enhanced sirolimus immunosuppressive regimen to address the risk of ALI and ALF so that, with the concurrence of the FDA, dosing may resume for non-ambulatory patients; Expanded guidance for prescribers, including a modified pre- and post-infusion oral corticosteroids regimen, and enhanced monitoring recommendations on a weekly basis for 3 months post-infusion; and a new Warnings & Precaution regarding increased susceptibility to serious infections due to immunosuppression. “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, Ph.D., president of research & development and technical operations.