• Miscellaneous musculoskeletal disorders

Zolgensma Generic Name & Formulations

General Description

Onasemnogene abeparvovec-xioi 2.0 × 1013 vector genomes (vg); per mL; susp for IV infusion; preservative-free.

Pharmacological Class

Adeno-associated virus vector-based gene therapy.

How Supplied

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

Generic Availability


Zolgensma Indications


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.

Zolgensma Dosage and Administration


≥2yrs: not applicable.


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.

Zolgensma Contraindications

Not Applicable

Zolgensma Boxed Warnings

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 Pharmacokinetics

See Literature

Zolgensma Interactions


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

Zolgensma Adverse Reactions

Adverse Reactions

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

Zolgensma Clinical Trials

See Literature

Zolgensma Note

Not Applicable

Zolgensma Patient Counseling

See Literature