Novartis scores a first with FDA approval for Itvisma

The US Food and Drug Administration (FDA) has approved 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, making it the first and only gene replacement therapy available for this broad population.

Developed by Swiss pharma giant Novartis (NOVN: VX), Itvisma is uniquely 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. The treatment will carry a wholesale acquisition cost of $2.59 million. The active ingredient in Itvisma is identical to Novartis’ Zolgensma but formulated at a different concentration.

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.