Important Safety Information
What is the most important information I should know about
- ZOLGENSMA can cause acute serious liver injury. Liver enzymes could become
elevated and may reflect acute serious liver injury in children who receive
- 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
- 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
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.
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
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
Please see the Full Prescribing Information.