Delandistrogene moxeparvovec

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. 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 limit of normal or total bilirubin > the upper limit of normal not due to Gilbert's syndrome) or active hepatic 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.

ELEVIDYS is the recombinant gene therapy product that is comprised of a non-replicating, recombinant, adeno-associated virus (AAV) serotype rh74 (AAVrh74) capsid and a single-stranded DNA expression cassette flanked by inverted terminal repeats (ITRs) derived from AAV2. The cassette contains: 1) an MHCK7 gene regulatory component comprising a creatine kinase 7 promoter and an α-myosin heavy chain enhancer, and 2) the DNA transgene encoding the engineered micro-dystrophin protein. Vector/Capsid: Clinical and nonclinical studies have demonstrated AAVrh74 serotype transduction in skeletal muscle cells. Additionally, in nonclinical studies, AAVrh74 serotype transduction has been demonstrated in cardiac and diaphragm muscle cells.

For single-dose intravenous infusion only. ELEVIDYS is for single-dose intravenous infusion only. Select patients for treatment with ELEVIDYS with anti-AAVrh74 total binding antibody titers <1:400, as measured by an FDA authorized test. Postpone in patients with active or recent (within 4 weeks) infections. Assess liver function, platelet counts and troponin-I before ELEVIDYS infusion. Recommended dosage: 10 to 70 kg: 1.33 × 10 14 vector genomes (vg) per kg of body weight, 70 kg or greater: 9.31 × 10 15 vg. One day prior to infusion, initiate a corticosteroid regimen for a minimum of 60 days. Recommend modifying corticosteroid dose for patients with liver function abnormalities. Administer as an intravenous infusion over 1-2 hours. Infuse at a rate of less than 10 mL/kg/hour.

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.

Boxed 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. Patients with preexisting liver impairment may be at higher risk. Prior to infusion, assess liver function by clinical examination and laboratory testing. Administer systemic corticosteroids before and after ELEVIDYS infusion. 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. 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.

Most common adverse reactions (incidence ≥5%) were vomiting and nausea, liver injury, pyrexia, thrombocytopenia, and troponin-I increased.

Prior to initiating the corticosteroid regimen required before ELEVIDYS administration, consider the patient's vaccination status. Patients should, if possible, be brought up to date with all immunizations in agreement with current immunization guidelines. Vaccinations should be completed at least 4 weeks prior to initiation of the corticosteroid regimen.

ELEVIDYS is shipped and delivered at ≤ −60ºC [−76ºF]. ELEVIDYS can be refrigerated for up to 14 days when stored at 2°C to 8°C (36º F to 46º F) in the upright position. Do not place back in the refrigerator once brought to room temperature. Do not shake.