Novartis’ Itvisma (onasemnogene abeparvovec), a re‑formulated gene therapy for spinal muscular atrophy, received FDA approval for patients aged two years and older, expanding a one‑time gene replacement option beyond infants. The intrathecal, fixed‑dose formulation enables treatment for older children and adults and was evaluated in a randomized Phase III study demonstrating motor function improvements versus sham controls. Itvisma’s approval offers a one‑time gene therapy alternative to chronic SMN‑upregulating treatments, with Novartis positioning the product as a potential durable option for a broader SMA population. The company emphasized the intrathecal delivery route and a simplified dosing strategy for older patients. Payers and clinical centers will evaluate long‑term data, safety monitoring logistics for older recipients, and comparative outcomes versus established therapies when integrating Itvisma into SMA care pathways.