The FDA approved Novartis’ Itvisma (onasemnogene abeparvovec) for spinal muscular atrophy (SMA) patients aged two years and older, extending access to gene replacement therapy beyond infants. Itvisma uses intrathecal delivery of an AAV vector at a fixed dose designed for older children, teens and adults who carry biallelic SMN1 mutations. Novartis based approval on a Phase 3 study showing statistically significant motor-function gains versus sham and evidence of stabilization in patients who had stopped other SMA treatments. The intrathecal route and fixed dosing aim to address biodistribution and safety considerations in larger patients. The decision opens a one‑time gene therapy option to a broader SMA population and will prompt payer discussions on long‑term benefit, follow-up registries and how gene therapy fits with chronic SMN‑modulating medicines.