Your guide to ZOLGENSMA

A Caregiver’s Guide to ZOLGENSMA is specifically designed to help caregivers learn more about spinal muscular atrophy (SMA) and ZOLGENSMA^® (onasemnogene abeparvovec-xioi). It covers many things from what causes SMA to how ZOLGENSMA works in the body and the necessary steps to start treatment. You will also learn about other families and their experiences with ZOLGENSMA and how they’re doing after treatment.

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Your guide to ZOLGENSMA

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Overview of the guide

Learn more about each section and what to expect at every step toward treatment.

What is SMA?

This section of the guide helps you understand what causes SMA and why it’s critical to talk to your child’s doctor immediately if you see any of the signs and symptoms of SMA. It also explains the importance of survival motor neuron (SMN) protein in the body and the role of the backup gene.

How ZOLGENSMA works

This section explains how ZOLGENSMA treats SMA with a one-time-only dose. You will learn how the new gene reaches its destination, how it replaces the function of the missing or nonworking gene, and what the new gene does to start helping the body function properly.

A Caregiver's Guide to ZOLGENSMA® (onasemnogene abeparvovec-xioi) - Results from the ZOLGENSMA clinical studies

ZOLGENSMA clinical studies overview

Several years of clinical studies have taken place to ensure the safety, efficacy, and appropriate dosing of ZOLGENSMA. This section will highlight the 3 clinical studies (2 completed and 1 ongoing) and 1 long-term follow-up study that demonstrate how ZOLGENSMA helps stop the progression of SMA.

A Caregiver's Guide to ZOLGENSMA® (onasemnogene abeparvovec-xioi) - Steps to starting ZOLGENSMA

See the steps to starting ZOLGENSMA

Once you and your doctor have chosen ZOLGENSMA, it’s important to take the necessary steps to start treatment and learn about the resources available to your family. This section covers the types of tests needed before the infusion and how the OneGene Program^® offers guidance and support at every step of the treatment journey.

Learn more about SMA and ZOLGENSMA

Download A Caregiver’s Guide to ZOLGENSMA

Important Safety Information

What is the most important information I should know about ZOLGENSMA?

ZOLGENSMA can increase liver enzyme levels and cause acute serious liver injury or acute liver failure which could result in death.
Patients will receive an oral corticosteroid before and after infusion with ZOLGENSMA and will undergo regular blood tests to monitor liver function.

Important Safety Information

What is the most important information I should know about ZOLGENSMA?

ZOLGENSMA can increase liver enzyme levels and cause acute serious liver injury or acute liver failure which could result in death.
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, if the patient misses a dose of corticosteroid or vomits it up, or if the patient experiences a decrease in alertness.

What should I watch for before and after infusion with ZOLGENSMA?

Infections before or after ZOLGENSMA infusion can lead to more serious complications. Caregivers and close contacts with the patient should follow infection prevention procedures. Contact the patient’s doctor immediately if the patient experiences 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 generally occur within the first two weeks after ZOLGENSMA infusion. 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.

What do I need to know about vaccinations and ZOLGENSMA?

Talk with the patient’s doctor to decide if adjustments to the vaccination schedule are needed to accommodate treatment with a corticosteroid.
Protection against influenza and respiratory syncytial virus (RSV) is recommended and vaccination status should be up-to-date prior to ZOLGENSMA administration. Please consult the patient’s doctor.

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 patient body waste for one month after infusion with ZOLGENSMA. Disposable diapers should be sealed in disposable trash bags and thrown out with regular trash.

What are the possible or likely side effects of ZOLGENSMA?

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

Indication

What is ZOLGENSMA?
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 was not evaluated in patients with advanced SMA.

The safety information provided here is not comprehensive. Talk to the patient’s doctor about any side effects that bother the patient or that don’t 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.

Please see the Full Prescribing Information.