Novartis receives FDA approval for Itvisma

Novartis (NVS) “announced that the US Food and Drug Administration has approved Itvisma, onasemnogene abeparvovec-brve, for the treatment of children two years and older, teens and adults living with spinal muscular atrophy with a confirmed mutation in the survival motor neuron 1 gene, making it the first and only gene replacement therapy available for this broad population. Itvisma is uniquely designed to address the genetic root cause of SMA with a one-time fixed dose that does not need to be adjusted for age or body weight.1 By replacing the SMN1 gene, Itvisma can improve motor function, offering the potential to reduce the need for chronically administered treatment associated with other available therapies for this population. The approval of Itvisma is based on data from the registrational Phase lll STEER study and supported by the open-label Phase lllb STRENGTH study. Itvisma showed statistically significant improvements in motor function and stabilization of motor abilities typically not seen in the natural history of the disease, with effects sustained over 52 weeks of follow-up.2,3 Additionally, Itvisma demonstrated a safety profile with adverse events that were consistent across both studies.2,3 The most common adverse events in the STEER study were upper respiratory tract infection and pyrexia, and the most common adverse events in the STRENGTH study were common cold, pyrexia, and vomiting.2,3 These data were presented at the 2025 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference. Approximately 9,000 people in the US live with SMA, and though there have been advancements in treating the disease, unmet needs remain for older children, teens, and adults in preserving motor neurons and maintaining physical strength. Itvisma will be available in the US in December. Novartis Patient Support is available to help eligible patients get started on treatment.”