FDA Approves Novartis Gene Therapy Itvisma for Expanded SMA Patient Population

Washington, Nov 24, 2025 — The U.S. Food and Drug Administration (FDA) has approved Itvisma, a gene therapy developed by Novartis, for a broader group of patients with spinal muscular atrophy (SMA), expanding its use to individuals aged two years and older.

Itvisma targets mutations in the SMN1 gene, which are responsible for the progressive neuromuscular degeneration seen in SMA patients. Previously, gene therapy for SMA was largely limited to infants and very young children. This new approval significantly broadens access to gene-based treatment for older pediatric and potentially adolescent patients.

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Unlike earlier intravenous gene therapies, Itvisma is delivered via intrathecal injection, allowing the therapeutic agent to be administered directly into the spinal canal. This approach is designed to enhance delivery to the central nervous system while potentially reducing systemic exposure and related risks.

Clinical data released by Novartis indicate that treated patients demonstrated meaningful improvements in motor function, muscle control, and overall quality of life. The company stated that the therapy will be rolled out through specialized treatment centers across the United States, with future plans to expand its global access.

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Industry observers view this approval as a significant milestone for the gene therapy field, reflecting an ongoing shift toward broader patient inclusivity and long-term clinical application. It also signals continued regulatory support for next-generation genetic medicines as they move beyond rare pediatric indications into wider patient populations.

Source: Reuters, FDA approves Novartis gene therapy Itvisma for broader spinal muscular atrophy patients, November 24, 2025.