The U.S. Food and Drug Administration approved Novartis’ Itvisma (onasemnogene abeparvovec-brve) for patients aged 2 years and older with spinal muscular atrophy (SMA) caused by biallelic SMN1 mutations. Itvisma uses the same vectorized SMN1 payload as Zolgensma but delivers a lower-volume, fixed intrathecal dose suitable for older children and adults. Approval rested on a randomized Phase III study showing statistically significant motor function gains on the Hammersmith Functional Motor Scale versus sham control. The decision extends one-time gene-replacement therapy options beyond infants and effectively creates a gene therapy pathway for older SMA patients who previously relied on chronic RNA therapies. Novartis said Itvisma will be available in December and noted pricing and access discussions are ongoing given the therapy’s single-dose, high-cost profile.