Novartis receives FDA approval for Itvisma®, the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA)

Core Viewpoint - Novartis announced the FDA approval of Itvisma (onasemnogene abeparvovec-brve) as the first and only gene replacement therapy for spinal muscular atrophy (SMA) in children aged two years and older, teens, and adults, addressing the genetic cause of the disease with a one-time fixed dose [2][3][6] Group 1: Product Details - Itvisma is designed to replace the mutated SMN1 gene, improving motor function and potentially reducing the need for chronic treatments associated with other therapies [2][7] - The approval is based on data from the Phase III STEER study and the Phase IIIb STRENGTH study, which showed significant improvements in motor function and stabilization over 52 weeks [3][7] - The most common adverse events reported were upper respiratory tract infections, pyrexia, common cold, and vomiting, with a consistent safety profile across studies [3][4] Group 2: Market Impact - Approximately 9,000 individuals in the US are affected by SMA, with unmet needs particularly among older children, teens, and adults [5] - The approval of Itvisma represents a significant advancement in SMA treatment, expanding access for a broader population and addressing ongoing unmet needs [4][6] Group 3: Company Commitment - Novartis aims to transform care for SMA by providing innovative, one-time therapies that empower patients of all ages [6][11] - The company has exclusive worldwide licenses for various gene replacement therapy delivery methods, enhancing its position in the SMA treatment landscape [10]