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Zolgensma

onasemnogene abeparvovec-xioi

Used for Musculoskeletal Conditions

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.

Reviewed by:

Yen Nguyen, PharmD

Allison Gourley, PharmD

Last reviewed on June 13, 2022

Basics Side effects Pros & cons Pharmacist tips FAQs Warnings Dosage Alternatives Latest news Prices Medicare

What is Zolgensma?

What is Zolgensma used for?

Certain type of spinal muscular atropy (SMA) in children under 2 years old

How Zolgensma works

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.

Drug Facts

Common BrandsZolgensma

Drug ClassGene therapy

Controlled Substance ClassificationNot a controlled medication

Generic StatusNo lower-cost generic available

AvailabilityPrescription only

What are the side effects of Zolgensma?

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.

Common Side Effects

Liver problems (high liver protein levels; 27%)
Vomiting (7%)

Other Side Effects

Fever

Serious Side Effects

Contact your healthcare provider immediately if you experience any of the following.

Liver failure: vomiting, right upper stomach pain, dark urine, yellow skin or eyes
Heart problems: changes in heart rate, bluish or grayish color of the skin, or fast or difficulty breathing
Thrombotic microangiopathy (TMA): fever, very high blood pressure, kidney problems, stomach pain, diarrhea, stroke, confusion, heart problems, or eye damage.

Source: DailyMed

The following side effects have also been reported

Side effects that you should report to your care team as soon as possible:

Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat
Hemolytic anemia—unusual weakness or fatigue, dizziness, headache, trouble breathing, dark urine, yellowing skin or eyes
Infection—fever, chills, cough, sore throat, wounds that don't heal, pain or trouble when passing urine, general feeling of discomfort or being unwell
Infusion reactions—chest pain, shortness of breath or trouble breathing, feeling faint or lightheaded
Kidney injury—decrease in the amount of urine, swelling of the ankles, hands, or feet
Liver injury—right upper belly pain, loss of appetite, nausea, light-colored stool, dark yellow or brown urine, yellowing skin or eyes, unusual weakness or fatigue
Unusual bruising or bleeding

Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):

Vomiting

Pros and cons of Zolgensma

Pros

Convenient, one-time infusion treatment

Treats SMA by focusing on the genetic problem

Help babies achieve certain milestones, such as sitting by themselves for 30 seconds or more

Cons

Given as an infusion into the vein, so it might be uncomfortable for small children

Not approved to treat children with advanced SMA, such as complete paralysis of arms and legs, or using a permanent ventilator

Child must get regular lab work for at least 3 months after treatment

Pharmacist tips for Zolgensma

Zolgensma can raise the liver levels and cause sudden liver problems. Your child will be monitored regularly for their liver levels before and after treatment. Let your provider know if your child experiences signs of liver problems, such as yellowing of the skin and eyes, vomiting, or becoming less alert.

It's very important that you follow the instructions on giving your child corticosteroids every day to lower the risk of liver problems. Let your provider know if your child misses a dose of corticosteroids, so they can instruct you on what to do.

Let your provider know if your child experiences any unexpected bruising or bleeding, seizures, or low urine amount, because these could be signs of a blood problem.

Take extra care to not touch your child's stools (feces) for 1 month after the Zolgensma infusion. Small amounts of the virus vector can come out with the feces (stools), so make sure to handle all of your child's waste properly. Wash your hands with soap and water after you change your child's diapers. Seal diapers in trash bags before throwing it into regular trash. Wash your hands properly if you come in contact with your child's stools.

Frequently asked questions about Zolgensma

Can Zolgensma be given to a child who currently has an infection?

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.

Can my baby be treated with Zolgensma if they're born pre-term?

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.

When and why will my child get corticosteroids?

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.

How often does my child needs to get blood work done?

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.

Will Zolgensma interfere with my child's vaccinations?

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.

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What are the risks and warnings for Zolgensma?

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.

Liver damage

Risk factors: Liver problems | Liver infection

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.

Low platelet level

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.

Damaged blood vessels and clotting problems

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.

Risk of heart problems

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.