Novartis Presents New Data on Safety and Efficacy of Zolgensma, Including Maintained and Improved Motor Milestones in Older and Heavier Children with SMA


  • The SMART study supplements a growing body of evidence on the use of Zolgensma in a patient population older and heavier (1.5 – 9.1 years of age) than the children treated in previous clinical studies1-6
  • Nearly all patients treated maintained or improved motor milestones after 52 weeks, with most switching to the one-time gene therapy from another chronically administered disease-modifying therapy 1-6
  • The SMART study is the first open-label clinical study of Zolgensma to include previously treated patients1-6

Novartis today announced new data that continue to support the clinical benefits of Zolgensma® (onasemnogene abeparvovec), the only one-time gene therapy for the treatment of spinal muscular atrophy (SMA). Final data from the SMART study highlight the safety and efficacy profile of Zolgensma in children with SMA weighing ≥ 8.5 kg to ≤ 21 kg, with a mean age of 4.69 years, most of whom (21/24, 87.5%) had discontinued use of another disease modifying therapy at the time of treatment. The new clinical results supplement emerging real-world experience and use of this innovative therapy in older and heavier children in countries where authorized use is not restricted by age.

The primary study objective was to evaluate the safety and tolerability of Zolgensma in older and heavier patients than were treated in previous clinical studies. The majority of patients in the study experienced increases in transaminases and transient thrombocytopenia; all cases were asymptomatic and managed with appropriate monitoring and treatment, as described in the product labeling. No new safety signals were observed in the study.

Most patients in the SMART study maintained motor milestones observed at baseline throughout the one-year study. The mean increase in total Revised Upper Limb Module (RULM) score was 2 points and a mean increase in total Hammersmith Functional Motor Scale – Expanded (HFMSE) score was 3.7 points. Four patients demonstrated new development milestones at week 52.


About SMART Study
SMART was a Phase 3b, open-label, single-arm, multicenter study designed to evaluate the safety, tolerability and efficacy of a one-time IV infusion of Zolgensma in pediatric patients who have symptomatic SMA with bi-allelic mutations in the SMN1 gene, any copy number of the SMN2 gene, and weigh ≥ 8.5 kg and ≤ 21 kg. The study enrolled a total of 24 patients with heterogeneous phenotypes of SMA across three weight brackets (8.5 - 13 kg; >13 – 17 kg; >17 – 21 kg), ranging in age from ~18 months to 9 years (mean age of 4.69 years).

Three patients were treatment-naïve to previous SMN-dependent therapies; 21 were treatment-experienced and discontinued risdiplam or nusinersen before enrollment in the study. The SMART study was the first open-label clinical study of Zolgensma to include previously treated patients. 1-6



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