FDA Greenlights Novartis' Gene Replacement Treatment for SMA

Key Takeaways NVS gained FDA approval for Itvisma, a gene replacement therapy for a broad SMA population.The therapy delivers a functional SMN1 gene in a single fixed dose to improve motor function.Phase III studies showed significant motor gains, stable abilities, and consistent safety through 52 weeks.Novartis (NVS) obtained FDA approval for onasemnogene abeparvovec-brve, a gene replacement therapy, for children two years and older, teens, and adults with spinal muscular atrophy (SMA).The therapy was appr ...