ELEVIDYS

STN: 125781 Proper Name: delandistrogene moxeparvovec-rokl Tradename: ELEVIDYS Manufacturer: Sarepta Therapeutics, Inc. Indication: ELEVIDYS is an adeno-associated virus vector-based gene therapy indicated for the treatment of patients 4 years of age and older with Duchenne muscular dystrophy (DMD) who are ambulatory and have a confirmed mutation in the DMD gene. Limitations of Use: ELEVIDYS is not recommended in patients with: Preexisting liver impairment (defined as gamma-glutamyl transferase [GGT] > 2 x upper limitof normal or total bilirubin > the upper limit of normal not due to Gilbert’s syndrome) or activehepatic viral infection due to the high risk of acute serious liver injury and acute liver failure. Recent vaccination (within 4 weeks of treatment) due to immunogenicity and potential safety concerns. Active or recent (within 4 weeks) infections due to safety concerns. Product Information Package Insert - ELEVIDYS Medication Guide - ELEVIDYS Demographic Subgroup Information – delandistrogene moxeparvovec-rokl (ELEVIDYS) Refer to Section 1.1 of the Clinical Review Memo for information about participation in the clinical trials and any analysis of demographic subgroup outcomes that is notable. Latest Safety Information FDA Takes Action on New Boxed Warning for Acute Serious Liver Injury and Acute Liver Failure Following Treatment with Elevidys and Revised Indication that is Limited to Ambulatory Duchenne Muscular Dystrophy Patients November 14, 2025 FDA Investigating Deaths Due to Acute Liver Failure in Non-ambulatory Duchenne Muscular Dystrophy Patients Following ELEVIDYS June 24, 2025 Supporting Documents November 14, 2025 Approval Letter - ELEVIDYS August 29, 2024 Approval Letter - ELEVIDYS June 20, 2024 Approval Letter - ELEVIDYS Center Director Decisional Memo - ELEVIDYS Office of Clinical Evaluation Director Memo - ELEVIDYS June 18, 2024 Integrated Clinical and Clinical Pharmacology Review Memo - ELEVIDYS Statistical Review - ELEVIDYS June 11, 2024 CMC Review Memo - ELEVIDYS January 10, 2024 Approval Letter - ELEVIDYS June 21, 2023 Summary Basis for Regulatory Action - ELEVIDYS Center Director Decisional Memo - ELEVIDYS Approval History, Letters, Reviews, and Related Documents - ELEVIDYS