The FDA approved Novartis’ intrathecal gene therapy Itvisma (onasemnogene abeparvovec) for patients aged two years and older with spinal muscular atrophy (SMA) due to biallelic SMN1 mutations. Novartis reported pivotal Phase III data showing statistically significant motor‑function gains on the Hammersmith scale versus sham control at one year, supporting an intrathecal, fixed‑dose regimen for older children and adults. The approval extends gene‑replacement therapy beyond infants and creates a new one‑time therapeutic option for a broader SMA population; Novartis tested a lower‑volume formulation designed for intrathecal delivery. Payers, centers and clinicians will now evaluate the place of Itvisma amid existing chronic SMA therapies.