Novartis Advances SMA Care with Positive CHMP Backing for Itvisma, Expanding Gene Therapy Access Across Age Groups

Novartis has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) for Itvisma, an intrathecal gene therapy developed for the treatment of spinal muscular atrophy (SMA). The therapy is designed to tackle the underlying genetic cause of SMA by delivering a functional copy of the SMN1 gene, targeting to restore survival motor neuron protein production essential for motor neuron function. The favorable CHMP recommendation represents an essential regulatory milestone for the therapy and signals potential growth of advanced treatment options for patients with SMA, mainly beyond traditionally treated pediatric populations.

According to Precedence Research, the global neuroimmunology market size was estimated at USD 37.80 billion in 2025 and is predicted to increase from USD 41.15 billion in 2026 to approximately USD 91.88 billion by 2035, expanding at a CAGR of 8.90% from 2026 to 2035. The neuroimmunology market is driven by the surging global incidence of neurological as well as autoimmune disorders, such as multiple sclerosis, Alzheimer's, and Parkinson's, combined with breakthroughs in targeted drug discovery and advanced diagnostic imaging.

One of the key aspects of this development is the therapy’s focus on wider patient eligibility, including older children, adolescents, and adults living with SMA. This could address a significant unmet demand within the neuromuscular disease landscape, where therapeutic access and effectiveness across age groups remain ongoing clinical considerations. The positive regulatory momentum may also strengthen Novartis’ position in the competitive SMA therapeutics market, which continues to evolve via innovation in gene therapy, RNA-based approaches, and disease-modifying treatments.

Another notable point is the growing role of gene replacement technologies in neurological diseases. Itvisma’s intrathecal administration strategy thus reflects continued efforts to optimize delivery methods aiming at the central nervous system, potentially influencing future therapeutic development in rare neurological disorders. While the news is mainly aligned with the SMA therapeutics, rare disease, and even neurological treatment markets, it also highlights the broader shift toward precision medicine alongside genetic interventions in neuroscience.

From a market perspective, the positive CHMP opinion may lead to increased commercial and clinical interest in next-generation neurological therapies, thus reinforcing momentum in the fields of gene therapy, rare neuromuscular disorders, and even advanced CNS therapeutics.