Overview
- Itvisma (onasemnogene abeparvovec-brve) is cleared for children two years and older, teens, and adults with a confirmed SMN1 mutation.
- The one-time, fixed-dose intrathecal therapy delivers a functional SMN1 gene and does not require age or weight dose adjustments.
- Approval draws on Phase 3 STEER and Phase 3b STRENGTH data showing stabilization and motor-function improvement through 52 weeks.
- Novartis says the therapy may lessen the burden of chronically administered treatments for eligible patients.
- The decision makes Itvisma the first gene therapy option authorized in the U.S. for this broader SMA population.