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:

≥2yrs: not applicable.

Children:

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.

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.

Pharmacologic Class:

Adeno-associated virus vector-based gene therapy.

Interactions:

Adjust vaccination schedule to accommodate concomitant corticosteroid use before and after infusion.

Adverse Reactions:

Elevated aminotransferases, vomiting; thrombocytopenia, elevated troponin-I.

Generic Availability:

NO

How Supplied:

Customized kit—1 (2–9 vials + alcohol wipes)

Pricing for ZOLGENSMA

1 (5.5ml) and 3 (8.3ml) vials kit (Qty: 1)
Appx. price $2101210
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