The FDA cleared Novartis’ Itvisma (onasemnogene abeparvovec) for spinal muscular atrophy (SMA) patients aged two and older, extending availability of a gene‑replacement option previously limited to infants. Novartis won approval based on a Phase 3 study showing intrathecal, fixed‑dose administration produced statistically significant motor function gains versus sham in a 127‑patient trial. Itvisma’s fixed intrathecal dose and broader age indication mark a commercial pivot: one‑time gene therapy access will no longer be limited to babies under two. Novartis highlighted manufacturing and dosing differences from its IV product Zolgensma and disclosed pricing and availability plans in its regulatory statements. The approval triggers discussions about long‑term monitoring, comparative use with chronic SMN‑modulating drugs, and reimbursement for an on‑label one‑time therapy.