Zolgensma is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA).

Zolgensma belongs to a group of drugs called gene therapy. It is designed to target the genetic root cause of SMA by replacing the function of the missing or nonworking SMN1 gene with a new, working copy of a human SMN gene.

Zolgensma comes in an injectable form to be given as a one-time infusion into the vein (IV). A course of an oral corticosteroid will be started the day before the Zolgensma infusion to help manage elevated liver enzyme reactions to Zolgensma by the body’s immune system.

Common side effects include elevated liver enzymes and vomiting.

Zolgensma is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA).

This medication may be prescribed for other uses. Ask your doctor or pharmacist for more information.

Serious side effects have been reported with Zolgensma. See the "Zolgensma Precautions" section.

The most common side effects include:

• Elevated liver enzymes
• Vomiting

These are not all of the possible side effects of Zolgensma.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Tell your child's healthcare provider about all the medicines your child is taking including prescription and non-prescription medicines, vitamins, and herbal supplements. 

Talk with your child's healthcare provider to decide if adjustments to the vaccination schedule are needed to accommodate treatment with a corticosteroid.

Zolgensma may cause serious side effects including:

• Serious liver injury. Liver enzymes could become elevated and may reflect acute serious liver injury in children who receive Zolgensma. Patients will receive an oral corticosteroid before and after infusion with Zolgensma and will undergo regular blood tests to monitor liver function. Contact the patient’s doctor immediately if the patient’s skin and/or whites of the eyes appear yellowish, or if the patient misses a dose of the corticosteroid or vomits it up.
• Temporary decreased platelet counts (thrombocytopenia). Your child's healthcare provider will monitor platelet counts before Zolgensma treatment and on a regular basis afterwards (weekly for the first month; every other week for the second and third months until platelet counts return to baseline).
• Temporary elevated blood levels of a certain heart muscle protein (Troponin-I). In clinical trials, some patients had elevated Troponin-I levels, which may indicate heart injury. 

What to watch for before and after Zolgensma infusion:

• Viral respiratory infections before or after Zolgensma infusion can lead to more serious complications. Contact the patient’s doctor immediately if you see signs of a possible viral respiratory infection such as coughing, wheezing, sneezing, runny nose, sore throat, or fever. 
• Decreased platelet counts could occur following infusion with Zolgensma. Seek immediate medical attention if a patient experiences unexpected bleeding or bruising.

Important Vaccination Information:

• Talk with the patient’s doctor to decide if adjustments to the vaccination schedule are needed to accommodate treatment with a corticosteroid.
• Protection against respiratory syncytial virus (RSV) is recommended.

Take precautions with the patient’s bodily waste. Temporarily, small amounts of Zolgensma may be found in the patient’s stool. Use good hand hygiene when coming into direct contact with bodily waste for 1 month after infusion with Zolgensma. Disposable diapers should be sealed in disposable trash bags and thrown out with regular trash.

Medicines can interact with certain foods. In some cases, this may be harmful and your doctor may advise you to avoid certain foods. In the case of Zolgensma there are no specific foods that you must exclude from your diet when receiving this medication.

Before starting Zolgensma, tell your doctor about all of your child's medical conditions including if your child:

• shows signs of a possible viral respiratory infection such as coughing, wheezing, sneezing, runny nose, sore throat, or fever. Viral respiratory infection before or after Zolgensma infusion can lead to serious complications.

Talk with the patient’s doctor to decide if adjustments to the vaccination schedule are needed to accommodate treatment with a corticosteroid.

Tell your doctor about all the medicines your child is taking including prescription and non-prescription medicines, vitamins, and herbal supplements.

Tell your doctor if you are pregnant or plan to become pregnant.

There are no available data regarding Zolgensma use in pregnant women. No animal reproductive and developmental toxicity studies have been conducted with Zolgensma.

Tell your healthcare provider if you are breastfeeding or plan to breastfeed.

There is no information available on the presence of Zolgensma in human milk, the effects on the breastfed infant, or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for Zolgensma and any potential adverse effects on the breastfed child from Zolgensma or from the underlying maternal condition.

Confirm a diagnosis of SMA through a genetic test

Before Zolgensma can be given, a diagnosis of SMA has to be confirmed. If needed, your doctor will order a genetic test to confirm the SMN1 gene is missing or nonworking.

Your doctor should perform an AAV9 antibody test

An AAV9 antibody test measures the amount of anti-AAV9 antibodies in your child’s blood. If your child’s immune system has built up a certain level of anti-AAV9 antibodies, he or she may not qualify for Zolgensma right away. If your child’s anti-AAV9 antibodies are too high, your doctor has the option to retest. Speak with your doctor about the AAV9 antibody test.

Your doctor should perform baseline liver function, platelet, and troponin-I tests

Your doctor should perform blood tests to check your child’s liver function and to establish baseline levels for other markers in the blood. These measurements before dosing will help your doctor and care team monitor your child’s levels after dosing. Your doctor will give you a prescription for an oral corticosteroid to start the day before your child’s infusion.

Connect with OneGene Program

The OneGene Program is a dedicated resource brought to you by AveXis. It is designed to support and guide you and your family before, during, and after treatment. After the Zolgensma Prescription Form and Patient Consent Form are received, a team of highly trained and dedicated people from the OneGene Program will call you to discuss the variety of patient resources that are available to support your family.

Prepare for Treatment Day

If not completed already, your doctor should perform blood tests to check your child’s liver function and to establish baseline levels for other markers in the blood. These measurements before dosing will help your doctor and care team monitor your child’s levels after dosing. Your doctor will give you a prescription for an oral corticosteroid to start the day before your child’s infusion.

Viral respiratory infection before or after Zolgensma infusion can lead to serious complications. Contact your doctor immediately if you see signs of a possible viral respiratory infection such as coughing, wheezing, sneezing, runny nose, sore throat, or fever.

A course of an oral corticosteroid should be started the day before infusion with Zolgensma. This helps manage elevated liver enzyme reactions to Zolgensma by the body’s immune system.

On Treatment Day

Give your child the second dose of the oral corticosteroid on the day of infusion as prescribed to help manage reactions to Zolgensma by the body’s immune system. The actual infusion will take 60 minutes. However, ask your doctor or care team for additional details on the schedule for the day. Remember to talk to your doctor and care team about any family members you would like to have with you on treatment day. You may have the option of having your Patient Resource Manager present on the day of treatment to support your family.

Post-treatment plan

Before you leave the hospital, talk with your doctor about post-treatment follow-up and additional monitoring. You will continue to give your child the corticosteroid as prescribed by your doctor. Your doctor will monitor your child’s liver function for at least 3 months after infusion through weekly clinical exams and blood tests for the first month and every other week for the second and third months. Your doctor will determine when to gradually reduce the dose of the corticosteroid and when to stop it.

• You should contact your doctor immediately if a dose of the corticosteroid is missed or vomited up
• Talk to your doctor about any side effects

In addition to liver function, your doctor will monitor other markers through blood tests, which may require weekly appointments:

• Weekly platelet counts for the first month and then every other week for the second and third months until platelet counts return to baseline
• Weekly troponin-I for the first month and then monthly for the second and third months until troponin-I level returns to baseline

The recommended dose of Zolgensma is 1.1 × 10¹⁴ vector genomes per kilogram (vg/kg) of body weight.

Zolgensma is usually administered by a healthcare provider in a medical setting making it unlikely for an overdose to occur. However, if overdose is suspected, seek emergency medical attention.

• Product is shipped and delivered frozen (≤ -60 °C [-76 °F]) in clear vials.

• Upon receipt, immediately place the kit in a refrigerator at 2°C to 8°C (36°F to 46°F). 

• ZOLGENSMA is stable for 14 days from receipt when stored at 2°C to 8°C (36°F to 46°F).

• DO NOT REFREEZE.

• Must use within 14 days of receipt.

WARNING: ACUTE SERIOUS LIVER INJURY 

• Acute serious liver injury and elevated aminotransferases can occur with ZOLGENSMA. 
• Patients with pre-existing liver impairment may be at higher risk. 
• Prior to infusion, assess liver function of all patients by clinical examination and laboratory testing (e.g., hepatic aminotransferases [aspartate aminotransferase (AST) and alanine aminotransferase (ALT)], total bilirubin, and prothrombin time). Administer systemic corticosteroid to all patients before and after ZOLGENSMA infusion. Continue to monitor liver function for at least 3 months after infusion.