Novartis Receives FDA Approval for Spinal Muscular Atrophy Gene Replacement Therapy

On Nov 26, 2025

The U.S. Food and Drug Administration (FDA) has approved Novartis’ Itvisma^® (onasemnogene abeparvovec-brve) for the treatment of children two years and older, teens and adults living with spinal muscular atrophy (SMA) with a confirmed mutation in the survival motor neuron 1 (SMN1) gene. This is the first and only gene replacement therapy available for this broad population. Itvisma is designed to address the genetic root cause of SMA with a one-time fixed dose that does not need to be adjusted for age or body weight. By replacing the SMN1 gene, Itvisma can improve motor function, offering the potential to reduce the need for chronically administered treatment associated with other available therapies for this population.

“The FDA’s approval of intrathecal onasemnogene abeparvovec is a game-changing advance, expanding the use of transformational gene replacement therapy for SMA across age groups,” said John W. Day, MD, PhD, professor of neurology and pediatrics, director, division of neuromuscular medicine at Stanford University School of Medicine, and co-director of Stanford’s Neuro IGNITE Center. “This achievement is not only a significant step forward for SMA – it also signals new possibilities for the broader field of neurological disorders and genetic medicine.”

“This new route of administration for a single dose of gene replacement therapy can mean so much more than what is measured by numbers on a functional motor scale – it could mean greater independence and freedom in activities of daily life,” said Kenneth Hobby, president, Cure SMA. “The SMA disease landscape has dramatically changed over the last six years, when the first gene therapy was approved. This is another welcome advancement, and it represents real progress in expanding access for many older patients and addressing the unmet needs that remain in our community.”

SMA is a rare, genetic neuromuscular disease caused by a mutated or missing SMN1 gene. The SMN1 gene is responsible for producing most of the SMN protein a body needs for muscle function, including breathing, swallowing and basic movement. Without it, motor neurons are irreversibly lost, leading to progressive, debilitating muscle weakness. A second gene, the SMN2 gene, produces a small fraction (~10%) of functional SMN protein compared with the SMN1 gene. Individuals with more copies of the SMN2 gene generally have a less severe form of SMA than those with fewer copies.

Approximately 9,000 people in the US live with SMA, and though there have been advancements in treating the disease, unmet needs remain for older children, teens, and adults in preserving motor neurons and maintaining physical strength.

Itvisma will be available in the US in December. Novartis Patient Support is available to help eligible patients get started on treatment. Patients and providers can call 1-855-441-4363 for personalized assistance, including help understanding insurance coverage and identifying potential financial assistance options.

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