India, April 24 -- Novartis (NVS) has received a positive opinion from the European Medicines Agency's CHMP for Itvisma, bringing the one-time gene replacement therapy a step closer to approval for older children, teens, and adults living with spinal muscular atrophy. The recommendation supports its use in patients aged two years and older with 5q SMA caused by bi-allelic SMN1 mutations.
Spinal muscular atrophy (SMA) is a rare genetic neuromuscular disease that leads to progressive muscle weakness affecting mobility, breathing, and daily independence. While treatment options have expanded for infants and young children, older patients have historically had fewer opportunities to benefit from advanced therapies.
Itvisma (intrathecal onasem...