New data revealed for Novartis SMA gene therapy

According to new data in older and heavier children treated with Novartis’ spinal muscular atrophy (SMA) gene therapy, nearly all participants in the Phase IIIb trial maintained or improved motor milestones after 52 weeks.

Final data from the Phase IIIb SMART study of Zolgensma^® (onasemnogene abeparvovec) highlighted its safety and efficacy in children weighing ≥ 8.5kg to ≤ 21kg, with a mean age of 4.69 years.

Findings from the study demonstrated that most children maintained motor milestones throughout the one-year study.

According to Novartis, key data highlights include:

• Most patients maintained or improved motor milestones from baseline at the time of final analysis
• There were 95.8 percent of patients who could sit with slight support who still met this milestone at Week 52
• Three patients achieved the milestone of newly standing with support and one achieved newly walking with support at Week 52
• All patients (100 percent) who could walk at baseline maintained this milestone until end of study.

Importance of the new data for the Novartis gene therapy

This data is “the first Zolgensma open-label clinical study to include older and heavier, as well as previously treated patients,” according to Dr Sandra Reyna, Chief Scientific Advisor and Head of Global Medical Engagement for SMA at Novartis.

“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,” stated Dr Hugh McMillan, Pediatric Neurologist. “These data inform the use of Zolgensma in children up to 21kg, supporting the use of a one-time gene replacement therapy as a therapeutic option for SMA in a broader population.”

The company also shared that overall, “the new clinical results supplement emerging real-world experience” and use of the treatment in older and heavier children “in countries where authorised use is not restricted by age.”

These data are among a Zolgensma data set being presented during the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, Novartis confirmed.

The data release follows Novartis’ announcement last year demonstrating that the gene therapy enabled long-term follow up trial participants to experience 100 percent achievement of all assessed milestones in those given Zolgensma prior to SMA symptom onset.