On Monday, the U.S. Food and Drug Administration (FDA) approved Novartis AG’s (NYSE:NVS) Itvisma (onasemnogene abeparvovec-brve) for children two years and older, teens, and adults with spinal muscular atrophy (SMA).

The approval makes the gene replacement therapy the first and only one available for this broad population.

Itvisma addresses the genetic root cause of SMA. The one-time fixed dose does not need adjustment for age or body weight.

The patients with SMA have a confirmed mutation in the survival motor neuron 1 (SMN1) gene.

By replacing the SMN1 gene, Itvisma can improve motor function, offering the potential to reduce the ...