Novartis presets new data on Zolgensma

Novartis presented new data that continue to support the clinical benefits of Zolgensma. Final data from the SMART study highlight the safety and efficacy profile of Zolgensma in children with SMA weighing greater than or equal to 8.5 kg to less than or equal to 21 kg, with a mean age of 4.69 years, most of whomhad discontinued use of another disease modifying therapy at the time of treatment. 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 score was 2 points and a mean increase in total Hammersmith Functional Motor Scale – Expanded score was 3.7 points. Four patients demonstrated new development milestones at week 52.