Miscellaneous musculoskeletal disorders:

Indications for: ZOLGENSMA

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 gradually over the next 28 days if LFTs are unremarkable.

Boxed Warning:

Acute serious liver injury. Acute liver failure.

ZOLGENSMA Warnings/Precautions:

Risk of acute serious liver injury and acute liver failure (esp. preexisting liver impairment or acute hepatic viral infection). ALT, AST, or total bilirubin levels (except due to neonatal jaundice) >2×ULN: not studied. Monitor liver function prior to infusion, weekly for the 1st month, then every other week for the 2nd/3rd months until unremarkable results. Monitor creatinine, CBC prior to infusion. Monitor platelets and 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. Monitor for thrombotic microangiopathy (TMA); evaluate for hemolytic anemia and renal dysfunction if thrombocytopenia develops. Increased risk of serious systemic immune response; delay infusion in those with concurrent infections until resolved. Perform baseline anti-AAV9 antibody testing prior to infusion; may retest if titers are >1:50. Consult a specialist if acute serious liver injury/liver failure is suspected, troponin elevations accompanied by signs/symptoms, or TMA occurs. 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; hepatotoxicity, thrombocytopenia, TMA, elevated troponin-I.

Generic Drug Availability:


How Supplied:

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