New data on safety and efficacy of Zolgensma for SMA

Swiss pharma giant Novartis (NOVN: VX) today presented 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).

Novartis noted that 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.

These data are among a Zolgensma data set being presented during the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference now taking lace in Orlando.