Dear SMA Community,
Novartis Gene Therapies is committed to working with our partners in the SMA community to make a difference in the diagnosis, treatment, and care of those affected by SMA. We would like to announce that we will initiate SMART, a Phase 3b clinical study to further evaluate the safety and efficacy of Zolgensma® (onasemnogene abeparvovec-xioi) in patients with spinal muscular atrophy (SMA) weighing ≥ 8.5 kg and ≤ 21 kg, following a single intravenous (IV) infusion. Clinical data from this study will supplement emerging real-world evidence and use of Zolgensma.
The global study is expected to enroll 24 symptomatic children with SMA from sites in Europe, North America, Australia, and Taiwan, and will follow patients for a period of 12 months. All study site locations are still being finalized, with enrollment anticipated to start in September 2021. We expect only a few patients in each country to be enrolled and anticipate enrollment to be filled quickly. Recruitment will be highly targeted to meet the enrollment criteria and led by the individual sites. Physicians and caregivers looking for more information about the SMART study should refer to https://clinicaltrials.gov/ct2/show/NCT04851873 or contact the Novartis Medical Information team at [email protected].
We continue to invest in new research and development programs to better serve the SMA community. We thank you for your engagement and will continue to keep you informed of our progress.
Please continue reading for Important Safety Information, and please see accompanying Full Prescribing Information, including Boxed Warning.
Sincerely,
Your Novartis Gene Therapies Team
About the SMART trial
- SMART is a Phase 3b, open-label, single-arm, multicenter study designed to further evaluate the safety, tolerability, and efficacy of a single IV infusion of Zolgensma in patients who have symptomatic SMA with bi-allelic mutations in the SMN1 gene and any copy number of the SMN2 gene and weigh ≥ 8.5 kg and ≤ 21 kg.
- The global study is expected to enroll 24 patients and will follow participants for a period of 12 months. After study completion, participants will be invited to enroll into a long-term, follow-up study to collect additional safety and efficacy data.
Questions & Answers on SMART Study
What is the purpose of the SMART study?
The SMART study will further expand the clinical evidence beyond the patient population studied in clinical trials conducted by Novartis Gene Therapies to date.
How can patient families enroll in the study? Where can they go for more information?
All site locations are still being finalized and we expect to have additional details to share towards the middle of this year. It is important to note that enrollment is not anticipated to start until September 2021. Enrollment will be for 24 symptomatic children across sites in Europe, North America, Australia and Taiwan. As such, we expect only a few patients in each country to be enrolled and anticipate enrollment to be filled quickly. Recruitment will be highly targeted to meet the enrollment criteria and led by the individual sites. Physicians and caregivers looking for more information about the SMART study should refer to https://clinicaltrials.gov/ct2/show/NCT04851873 or contact the Novartis Medical Information team at [email protected].
Why is this study limited to 24 patients?
It is important to keep in mind this is a clinical study designed to extend the clinical evidence beyond the patient population studied in Zolgensma trials conducted by Novartis Gene Therapies to date. The purpose of the study is to get additional data on the use of Zolgensma, in a timely fashion, which then can be used to inform physicians and caregivers.
We remain unwavering in our commitment to advancing global access for babies and children with SMA, but the SMART study is not designed as an access pathway for Zolgensma. Currently, Zolgensma is approved in 39 countries and regions around the world and we continue to collaborate with all stakeholders, including health authorities and reimbursement bodies to enable access to Zolgensma for babies and children with SMA. Earlier this year, we also reaffirmed our commitment to continue the global Managed Access Program (MAP) with up to 100 doses planned in 2021 to make Zolgensma available to eligible patients with SMA who are under the age of two in countries where Zolgensma has not received regulatory approval. More information about MAP can be found here.
Indication and Important Safety Information for ZOLGENSMA® (onasemnogene abeparvovec-xioi)
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.
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.
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.
US-ZOL-21-0171 04/2021