Onasemnogene abeparvovec (Zolgensma)

Generic name: onasemnogene abeparvovec (Zolgensma) [ ON-a-SEM-noe-jeen-A-be-PAR-voe-vek ]
Brand names: Zolgensma (2.6-3.0 kg), Zolgensma (3.1-3.5 kg), Zolgensma (3.6-4.0 kg), Zolgensma (4.1-4.5 kg), Zolgensma (4.6-5.0 kg), ... show all 22 brands Zolgensma (5.1-5.5 kg), Zolgensma (5.6-6.0 kg), Zolgensma (6.1-6.5 kg), Zolgensma (6.6-7.0 kg), Zolgensma (7.1-7.5 kg), Zolgensma (7.6-8.0 kg), Zolgensma (8.1-8.5 kg), Zolgensma (8.6-9.0 kg), Zolgensma (9.1-9.5 kg), Zolgensma (9.6-10.0 kg), Zolgensma (10.1-10.5 kg), Zolgensma (10.6-11.0 kg), Zolgensma (11.1-11.5 kg), Zolgensma (11.6-12.0 kg), Zolgensma (12.1-12.5 kg), Zolgensma (12.6-13.0 kg), Zolgensma (13.1-13.5 kg)
Dosage form: intravenous kit (20 trillion vg/mL)
Drug class:Miscellaneous uncategorized agents

Medically reviewed by Drugs.com on Mar 3, 2022. Written by Cerner Multum.

What is onasemnogene abeparvovec?

Onasemnogene abeparvovec is used to treat spinal muscular atrophy in children younger than 2 years old who have a specific gene mutation that affects nerve signals to the muscles.

onasemnogene abeparvovec may not be effective in a child who is completely paralyzed or is dependent on a ventilator for breathing.

Onasemnogene abeparvovec may also be used for purposes not listed in this medication guide.

Warnings

This medicine can cause serious liver damage, especially in children who already have liver problems. Your doctor will perform blood tests to make sure your child does not have conditions that would make it unsafe to receive onasemnogene abeparvovec.

Before taking this medicine

onasemnogene abeparvovec can cause serious liver damage. Tell your doctor if your child has ever had liver problems.

It may not be safe to breastfeed your child while he or she is receiving this medicine. Ask your doctor about any risk.

How is onasemnogene abeparvovec given?

Your doctor will perform blood tests to make sure your child does not have conditions that would make it unsafe to receive onasemnogene abeparvovec.

Onasemnogene abeparvovec is given as an infusion into a vein, usually only one time. A healthcare provider will give your child this injection.

This medicine must be given slowly, and the infusion can take about 1 hour to complete.

Your child may be given steroid medication to help prevent serious side effects of onasemnogene abeparvovec. Keep giving your child the steroid for as long as your doctor has prescribed. The doctor may gradually reduce the dose as your child continues taking the steroid.

Your child will need frequent blood tests to check his or her liver function.

Your child may need to be vaccinated against respiratory syncyti