Overview of the ZOLGENSMA
clinical studies

The safety and efficacy of ZOLGENSMA® (onasemnogene abeparvovec-xioi) have been evaluated in clinical studies and continues to be evaluated in long-term follow-up. ZOLGENSMA has been shown to stop the progression of spinal muscular atrophy (SMA).

These studies enrolled patients with SMA who were treated after symptoms appeared (symptomatic) and patients treated before symptoms appeared (presymptomatic). ZOLGENSMA was given through an IV infusion. All patients received ZOLGENSMA and there was no placebo group.

See results of the STR1VE and START studies See results of the SPR1NT study
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Overview of the ZOLGENSMA
clinical studies

The safety and efficacy of ZOLGENSMA® (onasemnogene abeparvovec-xioi) have been evaluated in clinical studies and continues to be evaluated in long-term follow-up. ZOLGENSMA has been shown to stop the progression of spinal muscular atrophy (SMA).

These studies enrolled patients with SMA who were treated after symptoms appeared (symptomatic) and patients treated before symptoms appeared (presymptomatic). ZOLGENSMA was given through an IV infusion. All patients received ZOLGENSMA and there was no placebo group.

See results of the STR1VE and START studies See results of the SPR1NT study
Donovan, treated at ~2 months and pictured at 4 years, was diagnosed with SMA Type 1.

Patients treated after SMA symptoms appeared (symptomatic)

STR1VE study

This study followed the START study.

Participants: 22* patients with SMA Type 1 (had 2 copies of SMN2 backup gene, showed symptoms of SMA, and were 6 months of age or younger at the time of infusion)

Goal: Establish efficacy and safety of ZOLGENSMA

*One patient was initially not part of the data set but is included in the final data analysis.

START study

This was the first study of ZOLGENSMA.

Participants: 15 patients with SMA Type 1 (had 2 copies of SMN2 backup gene, showed symptoms of SMA, and were 8 months of age or younger at the time of infusion)

Goal: Establish safety and determine the appropriate dose of ZOLGENSMA

START LTFU study (ongoing)

In addition, participants in the START study were invited to enroll in a long-term follow-up (LTFU). The ongoing 15-year observational study enrolled 13 of 15 patients from the START study and is intended to monitor ongoing safety.

See the results of these studies

Patients treated before SMA symptoms appeared (presymptomatic)

SPR1NT study

This study includes patients who did not have symptoms of SMA.

Participants: 29 patients younger than 6 weeks of age with 2 or 3 copies of the SMN2 backup gene

Goal: Establish efficacy and safety of ZOLGENSMA and provide further results

See the results of this study

Safety of ZOLGENSMA

The safety of ZOLGENSMA has been evaluated in patients who ranged in age from 0.3 to 7.9 months at the time of infusion.

See the safety profile

How does SMA progress without treatment?

Learn about the main types of SMA and the typical progression of the disease without treatment.

See SMA types

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.