Zolgensma is a gene therapy used for spinal muscular atrophy (SMA). SMA is an inherited disorder that causes skeletal muscle weakness and wasting, which worsens with age.

SMA is caused when a gene, usually SMA1, is missing or doesn't work properly. Zolgensma provides a new functional or working copy of a human SMN gene.

Zolgensma is a one-time treatment given via an intravenous (IV) infusion. It was approved by the US Food and Drug Administration (FDA) in 2019.

What is Zolgensma used for?

Zolgensma is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). Zolgensma is given as a one-time infusion into a vein. Zolgensma has not been evaluated in patients with advanced SMA.

• Zolgensma can increase liver enzyme levels and cause acute serious liver injury or acute liver failure.
• Your child will receive an oral corticosteroid before and after infusion with Zolgensma and will undergo regular blood tests to monitor liver function.
• Contact your child's doctor immediately if the patient’s skin and/or whites of the eyes appear yellowish, if they miss a dose of corticosteroid or vomits it up, or if the patient experiences a decrease in alertness.

• Zolgensma is administered by IV infusion over 60 minutes.
• Zolgensma is administered alongside corticosteroid therapy. Corticosteroid therapy is started one day prior to infusion of Zolgensma and continued for a total of 30 days.
• Contact your child's doctor immediately if your child's skin and/or whites of the eyes appear yellowish, if they miss a dose of corticosteroid or vomits it up, or if the patient experiences a decrease in alertness.
• Before and after your child receives Zolgensma watch for:
  • Infections before or after Zolgensma infusion can lead to more serious complications. Contact the patient’s doctor immediately if you see any signs of a possible 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 the patient experiences unexpected bleeding or bruising.
  • Thrombotic microangiopathy (TMA) has been reported to occur approximately one week after Zolgensma infusion. Caregivers should seek immediate medical attention if the patient experiences any signs or symptoms of TMA, such as unexpected bruising or bleeding, seizures, or decreased urine output.

Do I need to 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.

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

Before receiving Zolgensma, talk with your child's doctor about:

• Vaccinations to decide if adjustments to the vaccination schedule are needed to accommodate treatment with a corticosteroid.
• Protection against respiratory syncytial virus (RSV), which is recommended.

The most common side effects that occurred in patients treated with Zolgensma were elevated liver enzymes and vomiting.

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

Call your child's doctor for medical advice about side effects that bother you or do not go away.

You are encouraged to report suspected side effects by contacting the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch, or Novartis Gene Therapies, Inc. at 833-828-3947.

Adjustments may need to be made to your child's vaccination schedule to accommodate treatment with a corticosteroid. Talk with your child's doctor.