05:31 AM EST, 11/25/2025 (MT Newswires) -- Novartis ( NVS ) reported Monday that the US Food and Drug Administration has approved itvisma to treat spinal muscular atrophy, or SMA.

The gene replacement therapy is for children aged two years and older, teens, and adults, the company said, adding that the treatment addresses the genetic root cause of SMA with a one-time fixed dose that does not need to be adjusted for age or body weight.

Itvisma will be available in the US in December.

The approval is based on data from a registrational Phase 3 study supported by an open-label Phase 3b trial, the company said. Itvisma showed statistically "significant improvements" in motor function and stabilization of motor abilities "typically not seen in the natural history of the disease, with effects sustained over 52 weeks of follow-up."

Novartis ( NVS ) shares rose 1% in Tuesday's premarket activity.