Nov 24 (Reuters) – (This Nov 24 story has been corrected to fix the pricing for Zolgensma to $2.5 million from $2.1 million in paragraph 4)<\/p>\n
The U.S. Food and Drug Administration has approved Novartis’ (NOVN.S), opens new tab<\/a> gene therapy for patients with a rare muscle disorder, the drugmaker said on Monday.<\/p>\n Sign up here.<\/a><\/p>\n The therapy, branded as Itvisma, was approved for the treatment of spinal muscular atrophy patients of age two years and older who have a confirmed mutation in the survival motor neuron 1 gene.<\/p>\n Itvisma contains the same active ingredient as the Swiss drugmaker’s older therapy, Zolgensma, which is approved in the U.S. to treat SMA patients less than 2 years of age.<\/p>\n The new treatment has a wholesale acquisition cost of $2.59 million, compared with $2.5 million for Zolgensma.<\/p>\n Itvisma is the first and only gene replacement therapy available for the broad population, Novartis said.<\/p>\n “(This) gives patients even more choice, which for any patient is a good thing,” Tracey Dawson, U.S. Therapeutic Area Head of Neuroscience at Novartis, told Reuters ahead of the approval.<\/p>\n In a late-stage trial, treatment with Itvisma led to a statistically significant 2.39-point improvement on a scale that assesses motor ability and disease progression.<\/p>\n Spinal muscular atrophy is a rare, genetic neuromuscular disease caused by a mutated or missing SMN1 gene, which is responsible for the production of a protein needed for muscle function, including breathing, swallowing and basic movement.<\/p>\n It is the leading genetic cause of infant deaths and about 9,000 people in the U.S. live with the condition.<\/p>\n Unlike Zolgensma, which is administered intravenously based on patient weight, Itvisma is a concentrated formulation administered directly to the central nervous system through the spinal cord. The new treatment does not need to be adjusted for the patient’s weight, the company said.<\/p>\n Both therapies replace the SMN1 gene, offering the potential to reduce the need for chronically administered treatment associated with other available therapies for this population.<\/p>\n Zolgensma generated $925 million in global sales in the first nine months of 2025.<\/p>\n Reporting by Sriparna Roy in Bengaluru and Bhanvi Satija in London; Editing by Leroy Leo<\/p>\n Our Standards: The Thomson Reuters Trust Principles., opens new tab<\/a><\/p>\n Purchase Licensing Rights<\/a> Sriparna reports on pharmaceutical companies and healthcare in the United States. She has a master’s degree in English literature and post graduate diploma in broadcast journalism. <\/p>\n![\"Sriparna](\"https:\/\/www.europesays.com\/ch\/wp-content\/uploads\/2026\/03\/https:\/\/cloudfront-us-east-2.images.arcpublishing.com\/reuters\/H24NV3AFCZCWPC463OG3JRYY7U.jpg\")
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