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.