Novartis wins FDA approval for Itvisma gene therapy to treat older SMA patients

The Swiss drugmaker Novartis has secured U.S. regulatory approval for Itvisma (onasemnogene abeparvovec-brve), a gene replacement therapy for individuals aged two years and older with Spinal Muscular Atrophy (SMA) and a confirmed SMN1 mutation. Itvisma represents the first one-time gene therapy available for a broader SMA population beyond infants.

The treatment delivers a functional copy of the SMN1 gene via an injection into the spinal fluid, eliminating the need for weight-based dosing. Clinical trials demonstrated statistically significant improvements in motor function and stabilization of disease progression. Novartis emphasises that this approval could reduce or even eliminate the requirement for chronic therapy in older patients. With this milestone, Itvisma becomes a major extension of Novartis’s commitment to gene therapy and could reshape the standard of care for older children, teens, and adult SMA patients.