The U.S. Food and Drug Administration approved Novartis gene therapy for spinal muscular atrophy on Nov. 24, marking a major development for patients living with the rare neuromuscular disorder. Novartis said the treatment, branded as Itvisma, is cleared for patients aged two and older who have a confirmed mutation in the survival motor neuron 1 gene, which is essential for producing a protein critical for muscle function.
Wider treatment access
Itvisma expands the company’s gene therapy portfolio for spinal muscular atrophy, commonly known as SMA. The therapy contains the same active ingredient as Zolgensma, Novartis’ earlier treatment approved in the U.S. for patients younger than two. With the new authorization, Novartis gene therapy now offers a gene replacement therapy that covers a broader age range, addressing a long-standing gap for families seeking options beyond early childhood.
Novartis said Itvisma is the first gene replacement therapy available for this wider patient population. Tracey Dawson, the U.S. therapeutic area head of neuroscience at Novartis gene therapy, said the approval increases treatment choices for families managing the progressive condition. “This gives patients even more choice, which for any patient is a good thing,” Dawson said.
The company set the wholesale acquisition cost for Itvisma at $2.59 million, compared with $2.1 million for Zolgensma. Both therapies target the same genetic deficiency but differ in administration method and eligible patient groups.
Trial results and clinical impact
The FDA approval is based on late-stage clinical trial results that showed meaningful improvements for treated patients. Novartis reported that participants receiving Itvisma recorded a statistically significant 2.39-point increase on a motor-function scale that measures mobility, muscle strength, and disease progression. These results indicate that delivering a functional copy of the SMN1 gene may slow the deterioration associated with SMA.
Spinal muscular atrophy results from a missing or mutated SMN1 gene, which prevents the body from producing adequate levels of a protein required for essential muscle activities, including breathing, swallowing, and basic movement. The disease is the leading genetic cause of infant deaths. According to Novartis, about 9,000 people in the U.S. live with SMA, and many face long-term physical limitations that worsen without intervention.
The gene therapies developed for SMA aim to address the root cause rather than only managing symptoms. By delivering a functional SMN1 gene, treatments such as Itvisma and Zolgensma offer a one-time approach that seeks to provide lasting benefit.
Administration differences and future outlook
Itvisma differs from Zolgensma in its method of administration. While Zolgensma is delivered intravenously and requires dosing adjustments based on a patient’s weight, Itvisma is formulated for direct delivery to the central nervous system through the spinal cord. Novartis said this concentrated approach eliminates the need for weight-based dosing and is designed for patients beyond infancy who may require an alternative route of administration.
Both therapies, however, share the same goal: replacing the defective gene with a functional copy. The company said these treatments may reduce the need for chronic therapies often used to manage symptoms throughout a patient’s life.
Zolgensma generated $925 million in global sales in the first nine months of 2025, reflecting strong demand for gene-based treatments. The approval of Itvisma expands the company’s presence in the SMA market and may position Novartis gene therapy to broaden its gene therapy strategy across other neuromuscular conditions.
Industry analysts have noted increasing interest in gene replacement treatments as regulatory agencies continue to clear new therapies targeting rare diseases. With Itvisma’s approval, patients and caregivers have access to additional treatment options that address the underlying genetic cause rather than relying solely on long-term maintenance therapies.
As Novartis gene therapy prepares for a broader rollout in the U.S., the company said it will work with clinicians and treatment centers to support adoption of the therapy. The FDA’s decision marks another step in the ongoing development of gene therapies aimed at improving outcomes for patients living with rare, life-limiting disorders.
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