Delandistrogene Moxeparvovec-rokl

1 INDICATIONS AND USAGE ELEVIDYS is 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 [see Clinical Pharmacology ( 12.2 ), Clinical Studies ( 14 )] . 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.

6 ADVERSE REACTIONS Most common adverse reactions (incidence ≥5%) were vomiting and nausea, liver injury, pyrexia, thrombocytopenia, and troponin-I increased. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Sarepta Therapeutics, Inc., at 1-888-SAREPTA (1-888-727-3782) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.

8.1 Pregnancy Risk Summary ELEVIDYS is not intended for use in pregnant women. In the U.S. general population, the estimated background risks of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively.

WARNING: ACUTE SERIOUS LIVER INJURY AND ACUTE LIVER FAILURE Acute serious liver injury, including life-threatening and fatal acute liver failure, has occurred with ELEVIDYS [see Warnings and Precautions ( 5.1 )]. Patients with preexisting liver impairment may be at higher risk [see Warnings and Precautions ( 5.1 )]. Prior to infusion, assess liver function by clinical examination and laboratory testing. Administer systemic corticosteroids before and after ELEVIDYS infusion. 5 WARNINGS AND PRECAUTIONS Serious Infections: Serious infections with fatal outcomes may occur due to concomitant administration of corticosteroids, additional immunosuppressants, and ELEVIDYS. Monitor patients for signs and symptoms of infection; treat appropriately. ( 5.2 ) Myocarditis: Acute, serious, life-threatening myocarditis and troponin-I elevations have been observed. Monitor troponin-I before ELEVIDYS infusion, and weekly for the first month after ELEVIDYS infusion. ( 5.3 ) Infusion-related Reactions: Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred. Monitor during administration and for at least 3 hours after end of infusion. If symptoms occur, slow or stop the infusion and give appropriate treatment. 4 CONTRAINDICATIONS ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9, including a deletion of any portion or the entirety of these exons, in the DMD gene [see Warnings and Precautions ( 5.5 )] . ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9, including a deletion of any portion or the entirety of these exons, in the DMD gene.