Novartis Gene Therapies will initiate SMART, a Phase 3b clinical study to further evaluate the safety and efficacy of Zolgensma® (onasemnogene abeparvovec) in people living with SMA, weighing between 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 onasemnogene abeparvovec.
The global study is expected to enroll 24 symptomatic children with SMA from sites in Europe, North America, Australia and Taiwan and will follow the participants for a period of 12 months. All study site locations are still being finalised, with enrollment anticipated to start in September 2021. Novartis Gene Therapies expects only a few people in each country to be enrolled. Recruitment will be highly targeted to meet the enrollment criteria and led by the individual sites.