Novartis  has received approval from the U.S. Food and Drug Administration for its new gene therapy, Itvisma, designed to treat patients aged two years and older suffering from spinal muscular atrophy (SMA) with a confirmed mutation in the survival motor neuron 1 (SMN1) gene. The approval marks a major advancement in therapies for this rare, genetic neuromuscular disease, which is the leading genetic cause of infant deaths and affects about 9,000 people in the United States.

Itvisma contains the same active ingredient as Novartis' earlier therapy, Zolgensma, which is approved for SMA patients under two years old. The newly approved treatment features a concentrated formulation administered directly to the central nervous system through the spinal cord, eliminating the need for weight-based dosing required by Zolgensma.

Clinical trials have shown promising results, with Itvisma therapy leading to a statistically significant improvement in motor ability and a reduction in disease progression. The wholesale acquisition cost for Itvisma is set at $2.59 million, compared with $2.1 million for Zolgensma.

Both Itvisma and Zolgensma act by replacing the missing or mutated SMN1 gene, offering the potential to decrease reliance on chronically administered treatments. The approval provides patients and physicians with a new option, expanding treatment possibilities for those living with SMA.

Zolgensma generated $925 million in global sales in the first nine months of 2025, highlighting the demand for effective gene therapies in addressing rare disorders. Novartis emphasized that the approval of Itvisma gives patients greater choice, an important step forward in the fight against spinal muscular atrophy.