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 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.
Clinical Evidence Supporting the Opinion
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 (HFMSE), compared with 0.51 points for placebo. Even small gains on this scale can translate into meaningful improvements in daily function, such as grasping objects or maintaining independence.
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.Community and Clinical Perspective
SMA Europe emphasized the importance of expanding treatment access, noting that older children and adults often face limited therapeutic options. Clinicians highlighted that preserving motor function-even modestly-can significantly impact quality of life for people living with a progressive neuromuscular disease.
Next Steps
A final decision from the European Commission is expected within approximately two months. If approved, 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.
NVS has traded between $104.93 and $170.46 over the past year. The stock is currently trading at $144.71, down 1.88%.
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