Novartis gets positive EU opinion for Itvisma, a gene therapy for spinal muscular atrophy in patients 2 years and older

Novartis announced that the European Medicines Agency's CHMP gave a positive opinion for Itvisma, a gene replacement therapy for spinal muscular atrophy (SMA) in children aged two and older, teens, and adults. Itvisma is designed as a one-time intrathecal treatment that replaces the defective SMN1 gene, improving motor function and potentially reducing the need for ongoing SMA treatments. The recommendation was based on clinical trial data showing significant motor function improvements. The European Commission is expected to make a final approval decision within about two months, which could expand treatment options for a broader SMA patient population in Europe.