FDA approves new safety warning and revised indication for Sarepta’s Elevidys

The U.S. Food and Drug Administration announced it is taking action to approve new labeling submitted by the company that includes the addition of a Boxed Warning, the agency’s most prominent safety warning, to Elevidys, and that the indication section of the labeling limits the therapy’s indication to ambulatory patients four years of age and older with Duchenne muscular dystrophy. These actions follow reports of fatal acute liver failure in non-ambulatory patients treated with the product. After a comprehensive evaluation of the available safety data, FDA has now approved substantial labeling revisions for Elevidys, including: Addition of a Boxed Warning describing the risk of serious liver injury and acute liver failure, including fatal outcomes; Limiting the indication to ambulatory patients with DMD who are 4 years of age and older with a confirmed mutation in the DMD gene; Removal of the indication for non-ambulatory patients with DMD; Addition of a Limitations of Use statement to guide clinical decision-making; Updates to the Warnings and Precautions, Dosage and Administration, Adverse Reactions, Use in Specific Populations, Clinical Studies, and Patient Counseling Information sections; and Inclusion of a new Medication Guide for patients and caregivers. The FDA is requiring the manufacturer, Sarepta (SRPT) Therapeutics, to conduct a postmarketing observational study to further assess the risk of serious liver injury. The study will enroll approximately 200 patients with DMD and follow them for at least 12 months after administration of Elevidys, with periodic liver function assessments.