StoryRegulatory
EU approves Itvisma for SMA in children 2+ and adults
NovartisItvisma (onasemnogene abeparvovec-brve)
European Commission granted marketing authorization for Itvisma for SMA in children aged 2+ and adults with SMN1 mutations. First intrathecal gene therapy for this broad population.
- First gene therapy for SMA aged 2+ without weight restrictions
- STEER trial: 2.39-point HFMSE improvement at 52 weeks
- Intrathecal delivery enables older children and adults
- Benefits sustained through 52 weeks follow-up
- Second SMA approval after intravenous Zolgensma