Results from the symptomatic SMA studies of ZOLGENSMA

SYMPTOMATIC STUDY RESULTS

The clinical studies of ZOLGENSMA® (onasemnogene abeparvovec-xioi), STR1VE and START, enrolled patients who showed symptoms (symptomatic) of spinal muscular atrophy (SMA) before treatment. In addition, there is a voluntary, ongoing 15-year long-term follow-up (LTFU) observational study that is monitoring the safety of many patients from the START study. Learn more about these studies and see the results.

STR1VE study

The purpose of the STR1VE study was to establish the efficacy and safety of ZOLGENSMA. Results reported are at the end of the study.

STR1VE participants

The study enrolled 22* symptomatic patients diagnosed with SMA Type 1, who had 2 copies of the SMN2 backup gene, and were 6 months of age or younger at the time of IV infusion.

  • The average age at dosing was 3.7 months (range 0.5-5.9 months)
  • Patients received the therapeutic dose of ZOLGENSMA (dose approved by the FDA)
  • Patients were followed through their 18 months of age study visit

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

START study

The START study was the first ZOLGENSMA clinical study. The purpose was to establish safety and find the appropriate dose of ZOLGENSMA. Results from the START study are further supported by the STR1VE study results (both are measured against the natural history of SMA).

START participants

START results

START LTFU study

The purpose of the START long-term follow-up (LTFU) study is to monitor the safety of ZOLGENSMA over 15 years. Ongoing results of the START LTFU study show the safety and efficacy of ZOLGENSMA up to 5 years after treatment and 5 years of age or older.

START LTFU participants

START LTFU results

See results from the ongoing presymptomatic SMA study

See results

See the safety data of ZOLGENSMA in clinical studies

See ZOLGENSMA safety

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.