Miscellaneous musculoskeletal disorders:
Indications for ZOLGENSMA:
Treatment of pediatrics <2yrs of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.
Limitations of Use:
Safety and efficacy of repeat administration has not been evaluated. Use in patients with advanced SMA (eg, complete paralysis of limbs, permanent ventilator dependence) has not been evaluated.
Adult Dosage:
≥2yrs: not applicable.
Children Dosage:
Give as a slow IV infusion over 60mins. <2yrs: 1.1×1014 vg/kg (see full labeling). Starting one day prior to Zolgensma infusion: give systemic corticosteroids equivalent to oral prednisolone 1mg/kg/day for 30 days, then taper dose for the next 28 days if LFTs are unremarkable.
Boxed Warning:
Acute serious liver injury.
ZOLGENSMA Warnings/Precautions:
Risk of acute serious liver injury. Monitor liver function prior to infusion, weekly for the 1st month, then every other week for the 2nd/3rd months until unremarkable results. Monitor platelets, troponin-I prior to infusion, weekly for the 1st month, then every other week (platelets) and monthly (troponin-I) for the 2nd/3rd months until levels return to baseline. Perform baseline anti-AAV9 antibody testing prior to infusion; may retest if titers are >1:50. Premature neonates: delay infusion until full-term gestational age is reached.
ZOLGENSMA Classification:
Adeno-associated virus vector-based gene therapy.
ZOLGENSMA Interactions:
Adjust vaccination schedule to accommodate concomitant corticosteroid use before and after infusion.
Adverse Reactions:
Elevated aminotransferases, vomiting; thrombocytopenia, elevated troponin-I.
Generic Drug Availability:
NO
How Supplied:
Customized kit—1 (2–9 vials + alcohol wipes)
