Skip to content

Father pushing two young children on a tire swing

Father pushing two young children on a tire swing

The studies behind ZOLGENSMA

Louis has 2 copies of SMN2 gene
Treated at ~2½ months old

The studies behind ZOLGENSMA

ZOLGENSMA^® (onasemnogene abeparvovec-xioi) has been studied in children with spinal muscular atrophy (SMA), including those with no known SMA symptoms as well as those already showing symptoms at the time of treatment.

SMA can take away vital motor neurons, which may lead to a loss of muscle function. Once these neurons are lost, they can’t be regained. The good news is that early detection and diagnosis are widely available and early treatment with ZOLGENSMA can stop the progression of SMA. It’s important to remember that the sooner a child is treated, the more their muscle function can be preserved. Many parents may get their child’s SMA diagnosis before symptoms appear (also known as presymptomatic); others may learn of their child’s diagnosis after their child starts having symptoms (also known as symptomatic).

Choose below to learn about results in children treated:

Before Showing SMA
Symptoms (Presymptomatic)

After Showing SMA
Symptoms (Symptomatic)

Important Safety Information

Expand to see more

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.
There is a theoretical risk of tumor development with gene therapies such as ZOLGENSMA. Contact the patient’s doctor and Novartis Gene Therapies, Inc. (1-833-828-3947) if a tumor develops.

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 1-833-828-3947.

Please see the FullPrescribing Information.

Tap or scroll down to read full Important Safety Information and Indication

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.
There is a theoretical risk of tumor development with gene therapies such as ZOLGENSMA. Contact the patient’s doctor and Novartis Gene Therapies, Inc. (1-833-828-3947) if a tumor develops.

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 1-833-828-3947.

Please see the Full Prescribing Information.

This website may use cookies to improve the site and your experience. See how Novartis Gene Therapies, Inc. collects, uses, and shares your personal information in our Privacy Policy.

The information provided in this site is intended only for audiences of the United States. This information does not take the place of talking to your healthcare professional about medical conditions and treatments. If you have questions about ZOLGENSMA after reading this information, ask your healthcare professional.