Zolgensma

Generic name: onasemnogene abeparvovec-xioi
Dosage form: intravenous (infusion) injection
Drug class: Miscellaneous uncategorized agents

Medically reviewed by Philip Thornton, DipPharm. Last updated on Jun 12, 2023.

What is Zolgensma?

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.

Important information

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.

What should I tell my doctor before receiving Zolgensma?

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.

How should I receive Zolgensma?

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.

Dosing information

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

What are the side effects of Zolgensma?

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.

Interactions

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

Storage

Zolgensma is shipped frozen (≤ -60°C [-76°F]) in 10 mL vials with 2 fill volumes (either 5.5 mL or 8.3 mL).

What are the ingredients in Zolgensma?

Active ingredients: Adeno-associated virus

Inactive ingredients: tromethamine, magnesium chloride, sodium chloride, poloxamer 188, water.

Zolgensma is manufactured, packaged and distributed by Novartis Gene Therapies, Inc. 2275 Half Day Road, Suite 200, Bannockburn, IL 60015 USA.

Popular FAQ

Why is it so expensive?

Zolgensma costs $2.1 million for the one-time-only treatment. The reason Zolgensma is so expensive is because that is the price Novartis has decided it is worth because it “dramatically transforms the lives of families affected by this devastating disease” and the claimed cost of bringing new drugs to market. But this price is not without controversy. Continue reading

Zolgensma vs Spinraza: What are the key differences?

Zolgensma (onasemnogene abeparvovec-xioi) and Spinraza (nusinersen sodium) are both treatments for people with spinal muscular atrophy (SMA). There are a number of key differences between the two treatments, including:

Which patients with SMA they are approved for use in
How often they are administered
The type of treatments they are
How they work
How well they work

Is Zolgensma a cure for SMA?

No, Zolgensma (onasemnogene abeparvovec-xioi) is not a cure for SMA (spinal muscular atrophy). In addition, it cannot reverse any damage to motor neurons already caused by SMA before treatment. Continue reading

How is Zolgensma administered?

Zolgensma is given as a one-time intravenous (IV) infusion into a vein at a treatment center. The infusion takes approximately one hour. Your healthcare provider can direct you to the closest treatment center. Continue reading