Novartis’ Itvisma moves closer to EU approval for broader SMA population
Novartis received a positive CHMP opinion for Itvisma in spinal muscular atrophy patients aged 2 years and older. A European Commission decision is expected within about two months.
Novartis 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. A final decision from the European Commission is expected within approximately two months.
The CHMP recommendation is based on the Phase III STEER study, supported by the Phase IIIb STRENGTH and Phase I/II STRONG trials. In STEER, Itvisma achieved its primary endpoint, demonstrating a statistically significant 2.39-point improvement on the Hammersmith Functional Motor Scale, compared with 0.51 points for placebo. Benefits were observed in both treatment-naïve and previously treated patients, with effects sustained over 52 weeks. Safety findings were consistent across all supporting studies.
Itvisma (intrathecal onasemnogene abeparvovec) is designed to address the genetic root cause of SMA by delivering a functional copy of the SMN1 gene through a single intrathecal dose. Unlike chronic therapies that require ongoing administration, Itvisma aims to provide sustained SMN protein expression with one treatment.
The FDA cleared the treatment in November 2025 for patients 2 years and older with a confirmed mutation in the SMN1 gene, widening access beyond the infant population that has defined the therapy’s use since its original approval in 2019. If approved in the EU, Itvisma would become the first and only gene replacement therapy available in the EU for this broader SMA population, complementing the established role of Zolgensma in infants and young children.
Spinal muscular atrophy 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.