Novartis proposes positive data from the SMART study of Zolgensma in older and heavier children with SMA at the 2024 MDA conference

Novartis revealed new data affirming the clinical advantages of Zolgensma (onasemnogene abeparvovec), the sole one-time gene therapy for spinal muscular atrophy (SMA).

The SMART study's final data showcase Zolgensma's safety and efficacy in SMA patients weighing 8.5 kg to 21 kg, with most discontinuing other therapies. These results, presented at the 2024 Muscular Dystrophy Association (MDA) Conference, supplement real-world data in countries without age restrictions.

SMART assessed Zolgensma's safety and efficacy in pediatric SMA patients with specific gene mutations. Most maintained motor milestones during the one-year study, with mean increases in total Upper Limb Module and Hammersmith Functional Motor Scale scores. The study reported manageable adverse events like transient thrombocytopenia and increases in transaminases.

Zolgensma, approved in over 51 countries, targets the genetic cause of SMA. Novartis is committed to advancing SMA treatments through ongoing clinical trials and exploring intrathecal administration. SMA, a rare neuromuscular disease, results from a lack of functional SMN1 gene, leading to motor neuron loss and muscle function impairment. Early treatment initiation is crucial for halting disease progression.