What is ZOLGENSMA?

ZOLGENSMA® (onasemnogene abeparvovec-xioi) is a gene therapy approved by the US Food and Drug Administration (FDA) for children less than 2 years old with spinal muscular atrophy (SMA). ZOLGENSMA is:

  • A one-time-only dose
  • Given intravenously (IV)
  • An infusion that takes 60 minutes

Download A Caregiver’s Guide
to ZOLGENSMA

What is ZOLGENSMA?

ZOLGENSMA® (onasemnogene abeparvovec-xioi) is a gene therapy approved by the US Food and Drug Administration (FDA) for children less than 2 years old with spinal muscular atrophy (SMA). ZOLGENSMA is:

  • A one-time-only dose
  • Given intravenously (IV)
  • An infusion that takes 60 minutes

Download A Caregiver’s Guide
to ZOLGENSMA

Designed to target and replace

ZOLGENSMA is designed to target the genetic root cause of SMA by replacing the function of the missing or nonworking survival motor neuron 1 (SMN1) gene.

The new gene tells motor neuron cells to produce more survival motor neuron (SMN) protein. Motor neuron cells need SMN protein to survive and support muscle functions.

Time is of the essence

With each day that passes without treatment, motor neuron cells become weaker and weaker and eventually die. Once these cells stop working, they cannot be brought back. ZOLGENSMA is designed to produce SMN protein in the motor neuron cells, which preserves valuable muscular function needed for children to survive. While ZOLGENSMA keeps motor neurons working to preserve strength in the muscles, it is not a cure and cannot reverse damage already caused by SMA before treatment.

On treatment day, we were excited, we were hopeful. We were so happy that today we are making a difference. Today we’re going to do something that’s going to help him.

Laura, mother of Donovan
Donovan, treated at ~2 months and pictured at 2½ years, has SMA Type 1.

Alive and without breathing support

One clinical study, called STR1VE, is ongoing and includes only children with SMA Type 1. In this study, about 91% (20/22) of patients were alive and did not need permanent breathing support as of March 2019. In the natural history of SMA, patients with SMA Type 1 are not able to sit without help and about 25% are alive without permanent breathing support at 14 months of age.

Learn more about the clinical studies

What is permanent breathing support?

Permanent breathing support was defined as tracheostomy or the need for continuous use of a machine to help breathe for at least 16 hours every day for 2 weeks or more in a child who did not have a severe and short-lasting, reversible illness or surgery.

What is natural history?

Natural history refers to the progression of a disease in a person over time without treatment.

Download a Caregiver's Guide to ZOLGENSMA

Help for caregivers

A Caregiver’s Guide to ZOLGENSMA covers everything from facts about SMA and how ZOLGENSMA works to results from the clinical studies and steps to starting treatment.

Download the guide

Important Safety Information

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

  • Liver enzymes could become elevated and cause 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.

Important Safety Information

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

  • Liver enzymes could become elevated and cause 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.

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

  • 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.

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 respiratory syncytial virus (RSV) is recommended.

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.

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 the 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 AveXis at 833-828-3947.

Please see the Full Prescribing Information.