TM Owner Novartis
Zolgensma 2.0e13vg/ml (dosed 1.1e14vg/kg) Suspension for IV infusion (kit)
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Indications & Clinical Uses
- Zolgensma (onasemnogene abeparvovec-xioi) is a one-time adeno-associated virus vector-based gene therapy for spinal muscular atrophy (SMA).
- In the US it is approved for children less than 2 years of age with SMA who have bi-allelic mutations in the SMN1 gene.
- It is supplied as a customised kit of 2 to 14 vials of suspension at a nominal 2.0 x 10^13 vector genomes (vg) per mL, and the dose is calculated by body weight (1.1 x 10^14 vg per kg).
- It is given once as an intravenous infusion over about 60 minutes by a specialist healthcare team; it is never given at home.
- Zolgensma is the intravenous product; a separate onasemnogene abeparvovec-brve product (Itvisma) is listed on DailyMed and must not be confused with it.
- This information comes from the US label held by Novartis Gene Therapies, Inc.
Side Effects
- BOXED WARNING: cases of acute liver failure with fatal outcomes have been reported, and acute serious liver injury and raised liver enzymes (aminotransferases) can also occur after Zolgensma.
- Children with existing liver problems may be at higher risk.
- Liver function is checked before infusion, all children receive a systemic corticosteroid before and after infusion, and liver monitoring continues for at least 3 months.
- The most common side effects are raised aminotransferases (about 27%) and vomiting (about 7%).
- Other serious effects include thrombotic microangiopathy (blood-clotting problems with a fall in platelets), low platelet counts, raised troponin-I (a heart marker), infusion reactions including allergy, a heightened immune response to infection, and a theoretical risk of tumours.
- Caregivers should contact the care team immediately if the skin or whites of the eyes look yellow, the child is less alert, or there is fever, unusual bruising or bleeding, reduced urine output or seizures.
Precautions & Warnings
- Before treatment, liver function, platelet count and anti-AAV9 antibody level (which must be 1:50 or lower) are checked, and any illness or infection should be reported because infections before or after infusion can cause more serious complications.
- The US label lists no contraindications.
- To reduce liver injury the child takes oral prednisolone-equivalent corticosteroid at 1 mg/kg/day starting one day before infusion for 30 days, followed by a gradual 28-day taper if liver tests are normal.
- Liver tests and platelet counts are done weekly for the first month and then less often, with liver monitoring for at least 3 months in total; a cardiac evaluation is recommended.
- There is a theoretical tumour risk from vector DNA integration.
- The vaccination schedule may need adjusting during corticosteroid treatment.
- Zolgensma is shipped frozen at or below -60°C, refrigerated at 2°C to 8°C on receipt, used within 14 days and never refrozen; the hospital pharmacy handles it.
Avoid Interactions
- Increase effect or toxicity: the key issue is the corticosteroid required with Zolgensma, which suppresses the immune system.
- Live vaccines such as measles, mumps and rubella (MMR) and varicella are contraindicated while a child is on a substantially immunosuppressive steroid dose (20 mg or 2 mg/kg prednisone equivalent daily for 2 weeks or more), and the vaccination schedule should be adjusted; RSV prophylaxis is recommended.
- Reduce efficacy: high anti-AAV9 antibody levels (above 1:50) are a reason the child is not treated.
- The label does not list other specific drug interactions.
- Pharmacy points: prepared doses must be used within 8 hours of being drawn into the syringe and must not be given as a push or bolus.
- Tell the care team about all medicines and supplements the child receives.
References
Frequently Asked Questions
Zolgensma (onasemnogene abeparvovec-xioi) is a one-time gene therapy for children less than 2 years old with spinal muscular atrophy (SMA) who have bi-allelic mutations in the SMN1 gene. In the US it is given as a single intravenous infusion by a specialist team in hospital.
Zolgensma is given once, as an intravenous infusion over about 60 minutes by a healthcare team, and never as a push or bolus. The dose depends on body weight (1.1 x 10^14 vg/kg). Your child also takes a corticosteroid, starting one day before infusion and continuing for about 30 days, then tapered.
The most common Zolgensma side effects are raised liver enzymes and vomiting. Serious risks include acute liver injury or failure, low platelets and blood-clotting problems (thrombotic microangiopathy). Call the care team at once if your child's skin or eyes look yellow, or if there is fever, unusual bruising or bleeding, less urine or seizures.
Avoid live vaccines such as MMR and varicella while your child takes high-dose corticosteroids with Zolgensma (onasemnogene abeparvovec), and ask the team to adjust the vaccination schedule. Report any infection or illness before or after infusion, because infections can cause more serious complications. Tell the team about all medicines your child takes.
After Zolgensma, liver function is checked weekly for the first month and monitored for at least 3 months in total, through the steroid taper. Platelet counts are checked weekly for the first month, then every two weeks until they return to baseline. Liver tests and anti-AAV9 antibody levels are checked before infusion.
Yes. Zolgensma carries a boxed warning for acute serious liver injury and acute liver failure, including fatal cases. Children with existing liver problems may be at higher risk. Liver function is tested before infusion, corticosteroids are given before and after, and liver monitoring continues for at least 3 months.
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