Posted by Michael Wonder on 24 Nov 2025
Novartis receives FDA approval for Itvisma, the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy
24 November 2024 - Novartis today announced that the US FDA has approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of children two years and older, teens and adults living with spinal muscular atrophy with a confirmed mutation in the survival motor neuron 1 gene, making it the first and only gene replacement therapy available for this broad population.
Itvisma is uniquely designed to address the genetic root cause of spinal muscular atrophy with a one-time fixed dose that does not need to be adjusted for age or body weight.
