Novartis Receives FDA Approval for Itvisma, Expanding Access to Gene Replacement Therapy for People of All Ages Living with Spinal Muscular Atrophy (SMA)

East Hanover, N.J., [November 24, 2025] - Novartis announced today that the U.S. Food and Drug Administration (FDA) has approved Itvisma (onasemnogene abeparvovec) for the treatment of children two years and older, as well as adolescents and adults living with spinal muscular atrophy (SMA) with a confirmed mutation in the survival motor neuron 1 (SMN1) gene. This approval makes Itvisma the first and only gene replacement therapy available for this broad population of people affected by SMA.

Itvismais uniquely designed to target the genetic root cause of SMA with a one-time, fixed-dose infusion that does not require adjustment for a patient’s age or body weight. By replacing the faulty or missing SMN1 gene, Itvisma enables the body to produce functional SMN protein, which is essential for healthy muscle function. This approach has the potential to improve motor abilities and reduce the need for ongoing, chronic SMA treatment.

The approval is based on data from the Phase III STEER registrational study and supported by the Phase IIIb STRENGTH open-label study. Across these trials, Itvisma showed statistically significant improvement in motor function and stabilization of muscle abilities not typically observed in the natural history of the disease, with benefits sustained for up to 52 weeks. The treatment’s safety profile was consistent across studies, with the most common adverse events being upper respiratory tract infection and fever (STEER) and common cold, fever, and vomiting (STRENGTH).

SMA is a rare, inherited neuromuscular disorder caused by a mutation or deletion in the SMN1 gene, which is responsible for producing most of the SMN protein needed to control muscle movement, breathing, and swallowing. Without adequate SMN protein, motor neurons deteriorate, leading to progressive muscle weakness and loss of function.

Anestimated 9,000 people in the United States live with SMA. While recent advances have improved outcomes for infants and young children, there remains an urgent unmet need for effective options that preserve motor function and quality of life for older children, teens, and adults.