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Overview: Zolgensma is a one-time treatment for certain people with spinal muscular atrophy. Zolgensma provides the instructions for your child’s body to make a protein that is needed for muscles to work normally. Your health care provider will give your child this medicine by an injection into their vein (IV). Liver damage is a common side effect. Tell your child’s health care provider right away if your child is not eating well; unusually tired or weak; scratching; vomiting; or has a fever, light-colored poop, dark-colored urine (pee), or a yellowish hue in their skin or the whites of their eyes.

Uses

What is Zolgensma used for?

Zolgensma (onasemnogene abeparvovec-xioi) is commonly used to treat certain people with spinal muscular atrophy.

Zolgensma may also be used for other conditions as determined by your health care provider.

How does Zolgensma work (mechanism of action)?

Zolgensma is a medicine known as a gene replacement therapy. This medicine provides the instructions for your child’s body to make a protein that is needed for muscles to work normally. People without enough of this protein may have muscle weakness that can cause problems in breathing or walking.

How is Zolgensma supplied (dosage forms)?

Zolgensma is available in an injectable form that is given by a health care provider.

How should I store Zolgensma?

Zolgensma is usually given by a health care provider in a hospital or clinic. You will not store it at home.

Side Effects

What are the most common side effects of Zolgensma?

The most common side effects of Zolgensma are listed below. Tell your health care provider if your child has any of these side effects.

  • Liver damage (see below)
  • Vomiting

There may be other side effects of Zolgensma that are not listed here. Contact your health care provider if you think your child is having a side effect of a medicine. In the U.S., you can report side effects to the FDA at www.fda.gov/medwatch or by calling 800-FDA-1088 (800-332-1088). In Canada, you can report side effects to Health Canada at www.health.gc.ca/medeffect or by calling 866-234-2345.

What are the serious side effects of Zolgensma?

While less common, the most serious side effects of Zolgensma are described below, along with what to do if they happen.

Severe Allergic Reactions. Zolgensma may cause allergic reactions, which can be serious. Get help right away if your child has any of the following symptoms of a serious allergic reaction.

  • Breathing problems or wheezing
  • Racing heart
  • Fever or general ill feeling
  • Swollen lymph nodes
  • Swelling of the face, lips, mouth, tongue, or throat
  • Trouble swallowing or throat tightness
  • Itching or skin rash
  • Bumps on the skin called hives that can be red, pink, white, or brown depending on your skin tone
  • Nausea or vomiting
  • Dizziness, feeling lightheaded, or fainting
  • Stomach cramps
  • Joint pain

Severely Low Platelet Levels. Zolgensma can cause your child to have a severely low number of platelets in their blood, also called thrombocytopenia. Platelets help to form blood clots. Having a low level of platelets may increase the risk of bleeding. Keep all appointments to have your child’s blood checked, which will likely be needed for several months. Tell your health care provider if your child has any of the following symptoms of low platelet levels.

  • Easy bruising or bleeding
  • Bleeding that does not stop
  • Nosebleeds

Liver Damage. Liver damage, also called hepatotoxicity, can happen after Zolgensma use. Liver damage can be life-threatening. Keep all appointments to have your child’s blood checked, which will likely be needed for several months. Call your health care provider right away if your child has any of the following symptoms of liver damage.

  • Nausea or vomiting
  • Stomach or belly pain
  • Fever
  • Weakness, unusual tiredness, or decreased alertness
  • Itching
  • Loss of appetite
  • Light-colored poop
  • Dark-colored urine (pee)
  • Your skin or the whites of your eyes turning yellowish in color (also called jaundice)

Infusion Reactions. Zolgensma can cause reactions that can be serious when it is given to your child. Your child’s infusion may need to be slowed or stopped. Tell your child’s health care provider right away if your child looks or acts differently, has trouble breathing, or gets a rash or hives during the infusion.

Small Blood Vessel Clots. Zolgensma may cause a life-threatening condition called thrombotic microangiopathy (TMA). Small blood clots form in the blood vessels and can damage your child’s kidneys. Keep all your appointments to have your child’s blood checked. Tell your health care provider right away if your child has any of the following symptoms.

  • Fewer wet diapers
  • Unusual bruising or bleeding
  • Seizures

Warnings & Precautions

Who should not use Zolgensma?

Allergies to Ingredients. People who are allergic to any of the following should not use Zolgensma.

  • Onasemnogene abeparvovec-xioi
  • Zolgensma
  • Any of the ingredients in the specific product dispensed

Your pharmacist can tell you all of the ingredients in Zolgensma.

What should I know about Zolgensma before using it?

Zolgensma will be injected into your child’s vein (intravenous) by a health care provider in a hospital or clinic.

Keep all appointments for blood tests.

Your child will likely get a medicine called a corticosteroid before getting Zolgensma and afterward for a period of time. Tell your child’s health care provider right away if your child misses a corticosteroid dose or if your child throws up after taking a corticosteroid.

Both you and your child need to stay away from people who are sick. Wash your hands often, and stay away from crowded spaces. Ask anyone you live with to also try to stay well. If your child gets sick around the time Zolgensma is given, your child may have a more serious illness. Tell your health care provider right away if your child is sick or you think that your child may be sick before or after they get this medicine. Symptoms your child may have if they are sick include cough, wheezing, runny nose, sneezing, sore throat, or fever.

Carefully throw away your child’s poop for one month after they get Zolgensma. Throw away dirty disposable diapers in trash bags that you then place into regular trash. Anyone who comes into contact with the child’s poop needs to carefully wash their hands.

What should I tell my health care provider before using Zolgensma?

Tell your child’s health care provider about all of your child’s health conditions and any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements your child is using. This will help them know if Zolgensma is right for your child.

In particular, make sure that you discuss any of the following.

Current and Past Health Conditions. Tell your child’s health care provider if your child has any of the following.

  • Current infection
  • Liver problems

Vaccines. Talk to your child’s health care provider about any vaccines that your child may need. Your child should not receive certain vaccines right before or while using Zolgensma because they likely will also be using another medicine called a corticosteroid. Tell your child’s health care provider about any vaccines your child has recently received or has scheduled.

Pregnancy. People who can become pregnant are not usually prescribed Zolgensma.

Breastfeeding. People who can breastfeed are not usually prescribed Zolgensma.

How can I get more information about Zolgensma?

For more information about Zolgensma, you can visit the drugmaker’s website at www.zolgensma.com or call them at 855-441-4363.

Interactions

Does Zolgensma interact with foods or drinks?

There are no known interactions between Zolgensma and foods or drinks.

Does Zolgensma interact with other medicines (drug interactions)?

There are no known interactions between Zolgensma and other medicines. Always tell your child’s health care provider about any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements your child is using.

Does interact with other drugs you are taking?

Enter your medication into the WebMD interaction checker

Overdose/Missed Dose

What should I do if I accidentally use too much Zolgensma?

If you or someone else has used too much Zolgensma, get medical help right away, call 911, or contact a Poison Control center at 800-222-1222.

What should I do if I miss a dose of Zolgensma?

Zolgensma is usually given as a one-time dose by a health care provider in a hospital or clinic.

If you miss your child’s appointment to get Zolgensma, call to make another appointment as soon as possible.

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