Overview of the ZOLGENSMA
clinical studies

The safety and efficacy of ZOLGENSMA® (onasemnogene abeparvovec-xioi) have been established in 3 clinical studies (2 completed and 1 ongoing) and 1 long-term follow-up study. 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 over 60 minutes. All patients received ZOLGENSMA and there was no placebo group.

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

The safety and efficacy of ZOLGENSMA® (onasemnogene abeparvovec-xioi) have been established in 3 clinical studies (2 completed and 1 ongoing) and 1 long-term follow-up study. 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 over 60 minutes. All patients received ZOLGENSMA and there was no placebo group.

See results of the STR1VE and START studies See results of the ongoing SPR1NT study

Patients treated after SMA symptoms appeared (symptomatic)

STR1VE study (completed)

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 (completed)

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 voluntarily 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.

Patients treated before SMA symptoms appeared (presymptomatic)

SPR1NT study (ongoing)

This study includes patients who did not have symptoms of SMA. These patients continue to be evaluated.

Participants: 29 patients with 2 or 3 SMN2 backup gene copies

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

Safety overview 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.

How does SMA progress without treatment?

Learn about the primary 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 cause acute serious liver injury. Liver enzymes could become elevated and may reflect acute serious liver injury in children who receive ZOLGENSMA.
  • 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 cause acute serious liver injury. Liver enzymes could become elevated and may reflect acute serious liver injury in children who receive ZOLGENSMA.
  • 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 ZOLGENSMA?

  • 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 the patient experiences unexpected bleeding or bruising.
  • Thrombotic microangiopathy (TMA) has been reported to occur approximately one week after ZOLGENSMA infusion. Caregivers should 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 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.

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.