Wedoany.com Report-Nov. 25, Novartis (NOVN.S) announced on Monday that the U.S. Food and Drug Administration has approved its gene therapy, Itvisma, for individuals with spinal muscular atrophy who are two years of age and older and have a confirmed mutation in the survival motor neuron 1 gene.

The logo of Swiss drugmaker Novartis is pictured at the company's French headquarters in Rueil-Malmaison near Paris, France, April 22, 2020.

Itvisma shares the same active ingredient as Novartis's earlier therapy, Zolgensma, which received approval for treating spinal muscular atrophy in patients under two years old. The new therapy is administered directly into the central nervous system via the spinal cord, offering a concentrated formulation that does not require dosage adjustments based on patient weight, unlike the intravenous method used for Zolgensma.

The wholesale acquisition cost for Itvisma is set at $2.59 million, compared to $2.1 million for Zolgensma. Novartis described Itvisma as the first and only gene replacement therapy available for this wider patient population, providing an additional treatment option.

Tracey Dawson, U.S. Therapeutic Area Head of Neuroscience at Novartis, told Reuters: "(This) gives patients even more choice, which for any patient is a good thing."

In a late-stage clinical trial, Itvisma demonstrated a statistically significant improvement of 2.39 points on a motor function assessment scale that evaluates disease progression and physical abilities.

Spinal muscular atrophy is a rare genetic neuromuscular condition resulting from a mutation or absence of the SMN1 gene, which produces a protein essential for motor neuron survival and muscle function. The disease affects abilities such as breathing, swallowing, and movement, and it represents the primary genetic cause of infant mortality. Approximately 9,000 individuals in the United States currently live with the condition.

Both Itvisma and Zolgensma work by delivering a functional copy of the SMN1 gene, potentially decreasing reliance on ongoing treatments typically required for managing spinal muscular atrophy.

In the first nine months of 2025, Zolgensma achieved global sales of $925 million.

The approval of Itvisma expands access to gene therapy for older children, adolescents, and adults with spinal muscular atrophy, addressing a critical gap in treatment options for this progressive disorder. By targeting the genetic root cause directly at the site of motor neuron impact, the therapy aims to halt or slow disease advancement more effectively in this age group. Novartis's ongoing advancements in this area underscore a broader commitment to neurology, including efforts in neuroimmunology, neurodegeneration, and neuromuscular conditions, with the goal of enhancing patient outcomes across diverse lifespans. The introduction of Itvisma not only builds on the success of Zolgensma but also reflects evolving delivery methods that prioritize precision and accessibility in rare disease management.