Novartis received U.S. approval to extend its SMN1 gene‑replacement therapy to patients aged two and older under a new formulation called Itvisma. The approval follows a Phase 3 trial showing intrathecal dosing improved motor function versus sham control in pediatric and adult patients with less severe SMA presentations. Itvisma is a fixed‑dose intrathecal formulation of onasemnogene abeparvovec, distinct from the original weight‑based IV Zolgensma approved for infants. Novartis said the label gives older children, teens and adults a one‑time gene therapy option where none previously existed, shifting treatment choices alongside chronic SMN‑modulating drugs.