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
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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
Natalie, treated at ~14 months and pictured at 1½ years, was diagnosed with SMA Type 2.

Designed to target the genetic root cause of SMA

ZOLGENSMA is designed to target the genetic root cause of SMA with a one-time-only dose 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.

Learn more about the SMN1 gene

Time is of the essence

With each day that passes without treatment, motor neuron cells become weak and eventually lose all function and die. As a result, things like breathing, eating, swallowing, and speaking become difficult to impossible. Once these cells stop working, the condition is likely to become life threatening.

ZOLGENSMA is designed to deliver a new, working copy of the SMN gene to produce SMN protein, which preserves motor neuron cells throughout the body. In turn, ZOLGENSMA stops the progression of SMA and sustains the remaining muscle function needed for children to survive. The earlier treatment with ZOLGENSMA is given, the sooner muscle function may be preserved. ZOLGENSMA is not a cure and cannot reverse the damage already caused by SMA before treatment.

After treatment with ZOLGENSMA, we saw Brady again. He eats on his own, uses utensils and he eats everything by mouth.

Nicole, mother of BradyWatch Brady’s video

The efficacy and safety of ZOLGENSMA have been established in clinical studies

ZOLGENSMA has been evaluated in clinical studies and continues to be evaluated in long-term follow-up. The purpose of these clinical studies is to evaluate the safety and efficacy of ZOLGENSMA.

The studies are also evaluating how ZOLGENSMA may help patients achieve important milestones like breathing, eating, speaking, and sitting up without support.

See milestones achieved in the clinical studies

What is natural history?

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

Download PDF for A Caregiver's Guide to ZOLGENSMA® (onasemnogene abeparvovec-xioi)

A Caregiver’s Guide to ZOLGENSMA

What causes SMA? How does ZOLGENSMA work in the body? What are the results of the clinical studies? How do you start treatment with ZOLGENSMA? Get answers to these questions and many more with this in-depth guide created just for caregivers.

Download the guide

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.