Novartis receives positive CHMP opinion for Itvisma® for spinal muscular atrophy (SMA)
Novartis receives positive CHMP opinion for Itvisma® for spinal muscular atrophy (SMA)
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- If approved, Itvisma (intrathecal onasemnogene abeparvovec) will be first and only gene replacement therapy for children two years and older, teens and adults with SMA in the European Union
- Itvisma demonstrated clinically meaningful and statistically significant improvement in motor function in Phase III STEER study
- One-time dose of Itvisma replaces SMN1 gene, with potential to reduce need for chronic SMA treatment