Novartis on Monday presented new data that continue to support the clinical benefits of Zolgensma (onasemnogene abeparvovec) which is 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 company said in a statement.
These data are among a Zolgensma data set being presented during the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in Orlando, Florida, March 3 – 6.
“The results from the SMART study provide evidence that Zolgensma is clinically beneficial for older and heavier patients with SMA, many of whom have had prior treatment with another disease-modifying therapy,” said Dr. Hugh McMillan, Pediatric Neurologist. “These data inform the use of Zolgensma in children up to 21 kg, supporting the use of a one-time gene replacement therapy as a therapeutic option for SMA in a broader population.”
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.
According to the company, 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.
“This data – the first Zolgensma open-label clinical study to include older and heavier, as well as previously treated, patients – should build confidence among caregivers and healthcare professionals as they make informed treatment decisions, consistent with their local product label, for the studied patient population,” said Dr. Sandra P. Reyna, Chief Scientific Advisor and Head of Global Medical Engagement for SMA at Novartis. “We remain committed to reimagining possibilities for the SMA community.”
