Swiss pharmaceutical company Novartis Pharma AG (SIX:NOVN) (NYSE:NVS) announced on Monday that it has received approval from the US Food and Drug Administration (FDA) for Itvisma (onasemnogene abeparvovec-brve) for the treatment of children two years and older, teens, and adults living with spinal muscular atrophy (SMA) with a confirmed mutation in the survival motor neuron 1 (SMN1) gene.

According to Novartis, Itvisma becomes the first and only gene replacement therapy available for this broad population.

Itvisma is designed to address the genetic root cause of SMA with a one-time fixed dose that does not need to be adjusted for age or body weight. By replacing the SMN1 gene, Itvisma can improve motor function, offering the potential to reduce the need for chronically administered treatment associated with other available therapies for this population.

The FDA's approval is based on data from the registrational Phase lll STEER study, and is supported by the open-label Phase lllb STRENGTH study. Itvisma showed statistically significant improvements in motor function and stabilisation of motor abilities typically not seen in the natural history of the disease, with effects sustained over 52 weeks of follow-up.