Safety of ZOLGENSMA

ZOLGENSMA® (onasemnogene abeparvovec-xioi) has an established safety profile demonstrated in 3 clinical studies and 1 observational long-term follow-up (LTFU) study.

  • 44 patients were treated with ZOLGENSMA and ranged in age from 0.3 to 7.9 months at the time of infusion
  • The most common side effects (5% or more) that occurred in patients treated with ZOLGENSMA were elevated liver enzymes and vomiting
  • Reports of pyrexia (or fever), thrombotic microangiopathy (TMA), thrombocytopenia, acute liver failure sometimes resulting in death, acute liver injury, and increased troponin were identified during postmarketing experience

Safety data update

  • As of June 2020, 102 patients have been treated with ZOLGENSMA intravenously (IV) in clinical studies*
  • The most common side effects (5% or more) that occurred in patients treated with ZOLGENSMA were elevated liver enzymes and vomiting
  • Safety data continue to be collected

*Patients from 5 open-label studies, including 2 completed and 3 ongoing clinical studies: at the time of the analysis START (completed, N = 15), STR1VE (completed, N = 22), STR1VE-EU (ongoing, N = 33), STR1VE-AP (ongoing, N = 2), SPR1NT (ongoing, N = 30). Three patients in the START study received a lower dose.

See results from the symptomatic SMA studies

See results

See results from the presymptomatic SMA study

See results

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.