Zolgensma can cause side effects that range from mild to serious. More common side effects include vomiting and liver test irregularities. If you’re concerned about side effects from Zolgensma, talk with your doctor about coping tips or other treatment options.

Zolgensma (onasemnogene abeparvovec-xioi) is used to treat spinal muscular atrophy (SMA) in certain children under 2 years.

Keep reading to learn about the common, mild, and serious side effects Zolgensma can cause. To learn more about this medication, including its uses, dosage, and cost, read this Zolgensma overview.

Zolgensma has a boxed warning. A boxed warning is the most serious warning from the Food and Drug Administration (FDA). For details, see the “Boxed warning for Zolgensma” section.

Zolgensma can cause certain side effects, some more common than others. These side effects may be temporary, lasting a few days to weeks. Talk with your child’s doctor if side effects do not go away, get worse, or become too difficult to tolerate.

Zolgensma’s more commonly reported side effects include vomiting and increased liver enzyme levels.

For more details, see the “Tips for managing side effects” and “Boxed warning for Zolgensma” sections.

A mild side effect reported with Zolgensma is vomiting, which should be temporary. But if your child has symptoms that are ongoing or bothersome, talk with your doctor or pharmacist. For information about what you can do for vomiting, see the “Tips for managing side effects” section.

Zolgensma may cause mild side effects other than those listed here. See the drug’s prescribing information for details.

Zolgensma may cause serious side effects. The following list may not include all possible serious side effects of the drug. For more information, you can refer to the drug’s prescribing information.

If your child develops serious side effects with Zolgensma use, call your doctor right away. If the side effects seem life threatening or you think it’s a medical emergency, immediately call 911 or your local emergency number.

Serious side effects that have been reported include:

For more information about some of these side effects, see the “Tips for managing side effects” and “Boxed warning for Zolgensma” sections.

Note: After the Food and Drug Administration (FDA) approves a drug, it tracks and reviews side effects of the medication. Sharing your child’s experience helps make medication safer for everyone by giving doctors and researchers more information about how the drug works in real life. If your child develops a side effect during treatment with Zolgensma and you want to tell the FDA about it, visit MedWatch or call 800-FDA-1088.

Allergic reaction

For some people, Zolgensma infusion can cause an allergic reaction, including an infusion-related reaction.

In general, symptoms of an allergic reaction can be mild or serious.

What might help

If your child has mild symptoms of an allergic reaction, such as a mild rash, call the doctor right away. They may suggest a treatment to manage the symptoms.

If your child has symptoms of a severe allergic reaction, such as swelling or difficulty breathing, call 911 or your local emergency number right away. These symptoms could be life threatening and require immediate medical care. For more information about infusion-related reactions, see “Tips for managing side effects.”

Zolgensma has a boxed warning. A boxed warning is the most serious warning from the FDA.

Risk of liver injury and acute liver failure

Zolgensma can increase liver enzyme levels and may cause liver injury and acute (sudden) liver failure. Increased enzyme levels were a common side effect reported in studies of Zolgensma. In some children, this has progressed to liver injury and liver failure.

Your child may have a higher risk of this side effect if they already have liver problems before receiving the drug.

Symptoms of liver injury (damage) can include:

What you can do

To help prevent liver damage, your child will receive corticosteroids before and after their Zolgensma infusion.

The doctor will also evaluate your child’s liver function before and after the infusion. They’ll continue monitoring for up to 3 months following the infusion.

If your child has increased liver enzyme levels or other abnormal liver test results, the doctor will closely monitor for worsening enzyme levels and/or symptoms of liver injury. If they suspect liver injury, they’ll refer your child to a liver specialist for additional treatment.

To learn more about this warning, talk with your child’s doctor.

There may be things you can do to help make certain Zolgensma side effects more manageable. If they have ongoing, worsening, or bothersome side effects, talk with your child’s doctor. They can suggest tips for managing them.

Vomiting

Vomiting was a more common side effect reported in studies of Zolgensma. Steps you can take to manage your child’s vomiting include:

  • offering electrolyte drinks, such as Pedialyte
  • giving smaller meals throughout the day (if your baby is breastfeeding, continue to breastfeed often)
  • giving an over-the-counter antiemetic drug to treat vomiting, with the doctor’s guidance

Infusion-related reaction

Some children receiving Zolgensma have had an infusion-related reaction, but it was an uncommon side effect reported in studies of Zolgensma. Infusion-related reactions can include allergic reactions and anaphylaxis (a life threatening allergic reaction).

Symptoms of an infusion-related reaction can include skin rash and hives, vomiting, and breathing problems.

If your child has a reaction to Zolgensma, the doctor will stop the infusion and treat the symptoms. They may decide to resume the infusion depending on your child’s condition. For more information about this side effect, talk with your child’s doctor.

Consider the following important information before your child receives Zolgensma.

Zolgensma has a boxed warning. A boxed warning is the most serious warning from the FDA. For details, see “Boxed warning for Zolgensma.”

Other precautions

Before Zolgensma treatment starts, you and the doctor will discuss your child’s health history. Zolgensma may not be right for your child if they have certain medical conditions or other factors affecting their health. Be sure to talk with the doctor if any of the following apply to your child:

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Disclaimer: Healthline has made every effort to make certain that all information is factually correct, comprehensive, and up to date. However, this article should not be used as a substitute for the knowledge and expertise of a licensed healthcare professional. You should always consult your doctor or another healthcare professional before taking any medication. The drug information contained herein is subject to change and is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. The absence of warnings or other information for a given drug does not indicate that the drug or drug combination is safe, effective, or appropriate for all patients or all specific uses.