Zolgensma (onasemnogene abeparvovec or onasemnogene abeparvovec-xioi) is a medication that's used to treat a certain type of spinal muscular atropy in children under 2 years old. Zolgensma is given as a one-time infusion into the vein, but your child has to be monitored for at least 3 months after the treatment due to the risk of liver and clotting problems.
Spinal muscular atropy (SMA) is rare, genetic condition that affects your nerves and muscles. People with SMA are either missing a gene called survivor motor neuron 1 (SMN1) or have a SMN1 gene that's not working properly. The SMN1 gene is needed to make a protein that helps your nerves "talk" with your muscles. When it's missing, you have a hard time moving your muscles.
Zolgensma is a type of gene therapy. It's made up a working copies of the SMN1 gene inside of vectors. You can think of the vectors as delivery trucks and the SMN1 genes as package that the trucks are delivering. The vectors (delivery trucks) "deliver" working SMN1 genes to the person with SMA. This new SMN1 genes help your body make the proteins it needs to make your muscles move.
| Common Brands | Zolgensma |
|---|---|
| Drug Class | Gene therapy |
| Controlled Substance Classification | Not a controlled medication |
| Generic Status | No lower-cost generic available |
| Availability | Prescription only |
The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.
Contact your healthcare provider immediately if you experience any of the following.
Side effects that you should report to your care team as soon as possible:
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
Zolgensma can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Zolgensma can cause sudden liver problems. To lower the risk, your child will be given corticosteroids before and after their infusion. Their provider will also order blood work to check their liver health before and for at least 3 months after their Zolgensma infusion. Speak with your provider before starting Zolgensma to see if this medication is right for your child.
Zolgensma can cause low platelet levels and raise the risk of a condition called thrombocytopenia. This generally occurs within the first two weeks of Zolgensma infusion. Your provider will monitor your child's platelet levels before their infusion and regularly for at least 3 months until their platelet levels are normal.
Zolgensma can raise the risk of a clotting problem called thrombotic microangiopathy (TMA). TMA is a problem where the body forms small blood clots in the capillaries and small arteries, and has been seen in some people one week after their Zolgensma infusion. Let your provider know if your child has signs and symptoms of TMA, such as high blood pressure, bruising, seizures, or urinating less than usual.
Zolgensma might raise the levels of a protein in the blood called cardiac troponin-I. This can be sign of heart problems. Because of this risk, your child's provider will monitor their cardiac troponin-I levels before and for at least 3 months after their Zolgensma infusion, until their levels are normal. Let your provider know if your child experiences any changes in heart rate, bluish or grayish color of the skin due to the lack of oxygen, a fast respiratory rate, or difficulty breathing.
Your child's healthcare provider will determine the right dose of Zolgensma for your child based on their weight.
The provider will infuse their dose of Zolgensma into the veins over 1 hour.
Prescription savings
Stop paying too much for your prescriptions. Compare prices, get pharmacy coupons, and save up to 80%.
Find expert answers to your health questions so you can make the best decisions for yourself and your family.Health conditions
Medications & treatment
Access & affordability
Our monthly healthcare membership can help you save thousands on your prescription costs.Resources
Zolgensma shouldn't be given to your child if they currently have an infection. Wait until they've recovered from the infection before they receive this medication. Giving Zolgensma during an infection (e.g., cold, flu, stomach infections, ear infections, lung infections) can lead to serious problems. Let your provider know right away if your child experiences any signs of a possible infection, such as coughing, wheezing, sneezing, runny nose, sore throat, or fever.
Zolgensma should only be given to babies who've reached full-term gestational age. Babies born premature must wait until after they've reached full-term gestational age to be treated with this medication. This is because your child will receive corticosteroids as a part of Zolgensma treatment to help with certain side effects. But giving corticosteroids to babies born premature can affect their nerve or brain development. Speak to your provider to see if Zolgensma treatment is safe for your baby.
Your provider will give your child corticosteroids starting one day before their Zolgensma infusion and continue the treatment for 30 days. The purpose of corticosteroids is to help lower your child's risk of liver damage from Zolgensma. If labs show that your child's liver is working normally at the end of the 30 days, your child's provider will stop the corticosteroids by slowly lower the dose over the next month. Don't suddenly stop your child's corticosteroids because doing so can lead to side effects such as weakness, nausea, and vomitting.
Your provider will monitor your child for at least 3 months after their Zolgensma infusion. Your child will need to get blood work once a week for the first month, then every other week for the second and third months. This is to make sure their liver, certain blood cell levels, and heart are healthy.
Speak with your provider regarding the vaccination schedule before your child starts their treatment with Zolgensma. Certain vaccines, such as measles, mumps, and rubella (MMR) and varicella (chickenpox), are not allowed to be given while your child is taking corticosteroids. Corticosteroids can lower your child's immune system and affect their response to those vaccines. Your provider can discuss with you which vaccines your child needs to avoid during their treatment.
