HIGHLIGHTS OF PRESCRIBING INFORMATION
These highlights do not include all the information needed to use
ZOLGENSMA safely and effectively. See full prescribing informationfor ZOLGENSMA.
ZOLGENSMA(onasemnogene abeparvovec-xioi)Suspension for intravenous infusion
Initial U.S. Approval: 2019
WARNING: ACUTE SERIOUS LIVER INJURY
See full prescribing information for complete boxed warning.
• Acute serious liver injury and elevated aminotransferasescan occur with ZOLGENSMA. (5.1)
• Patients with pre-existing liver impairment may be athigher risk. (8.6)
• Prior to infusion, assess liver function of all patients byclinical examination and laboratory testing (e.g.,hepaticaminotransferases [aspartate aminotransferase (AST) andalanine aminotransferase (ALT)], total bilirubin, andprothrombin time). Administer systemic corticosteroid toall patients before and after ZOLGENSMA infusion.
Continue to monitor liver function for at least 3 monthsafter infusion (2.1) (2.3).
INDICATIONS AND USAGE
ZOLGENSMA (onasemnogene abeparvovec-xioi) is an adeno-associatedvirus vector-based gene therapy indicated for the treatment of pediatricpatients less than 2 years of age with spinal muscular atrophy (SMA) withbi-allelic mutations in the survival motor neuron 1 (SMN1) gene. (1)
Limitation of Use:
• The safety and effectiveness of repeat administration of ZOLGENSMA have not been eva luated. (1, 6.2)
• The use of ZOLGENSMA in patients with advanced SMA (e.g.,complete paralysis of limbs, permanent ventilator dependence) hasnot been eva luated. (1, 14)
DOSAGE AND ADMINISTRATION
ZOLGENSMA is for single-dose intravenous infusion only (2).
• The recommended dosage of ZOLGENSMA is 1.1 × 1014 vectorgenomes (vg) per kg of body weight.
• Administer ZOLGENSMA as an intravenous infusion over 60minutes. (2.1, 2.3)
• Starting one day prior to ZOLGENSMA infusion, administersystemic corticosteroids equivalent to oral prednisolone at 1 mg/kg of body weight per day for a total of 30 days. At the end of the 30-day period of systemic corticosteroid treatment, check liverfunction by clinical examination and by laboratory testing. Forpatients with unremarkable findings, taper the corticosteroid doseover the next 28 days. If liver function abnormalities persist,continue systemic corticosteroids (equivalent to oral prednisoloneat 1 mg/kg/day) until findings become unremarkable, and thentaper the corticosteroid dose over the next 28 days. Consultexpert(s) if patients do not respond adequately to the equivalent of1 mg/kg/day oral prednisolone. (2.1)
DOSAGE FORMS AND STRENGTHS
ZOLGENSMA is a suspension for intravenous infusion, supplied assingle-use vials.
ZOLGENSMA is provided in a kit containing 2 to 9 vials, as a combination of2 vial fill volumes (either 5.5 mL or 8.3 mL). All vials have a nominalconcentration of 2.0 × 1013 vector genomes (vg) per mL Each vial ofZOLGENSMA contains an extractable volume of not less than either 5.5 mLor 8.3 mL.
CONTRAINDICATIONS
None
WARNINGS AND PRECAUTIONS
• Thrombocytopenia: Monitor platelet counts before ZOLGENSMAinfusion, and weekly for the first month and then every other weekfor the second and third month until platelet counts return tobaseline. (2.3, 5.2)
• Elevated Troponin-I: Monitor troponin-I before ZOLGENSMAinfusion, and weekly for the first month and then monthl