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# Gene Therapy for Broader Spinal Muscular Atrophy Range
- URL: https://www.fdaweb.com/gene-therapy-for-broader-spinal-muscular-atrophy-range/
- Published: 2025-11-25T12:00:00.000Z
- Updated: 2026-09-14T15:27:43.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5160281

FDA has approved Novartis’ Itvisma (onasemnogene abeparvovec-brve), a new formulation of a gene therapy for spinal muscular atrophy (SMA), allowing the treatment for adults and children two years of age and older with confirmed mutations in the SMN1 gene. Itvisma contains the same active ingredient as Zolgensma, the intravenous gene therapy approved in 2019 for infants under two years old, according to an [agency release](https://www.fda.gov/news-events/press-announcements/fda-approves-gene-therapy-treatment-spinal-muscular-atrophy?ref=fdaweb.com).

The new product uses a more concentrated formulation delivered through a single intrathecal injection — directly into the cerebrospinal fluid — enabling treatment of older, heavier patients without weight-based dosing, it says. By injecting the therapy directly into the central nervous system, Itvisma aims to deliver the gene to motor neurons more efficiently, restoring production of survival motor neuron protein and halting further neurodegeneration.

Approval is based on data from a Phase 3 clinical trial in children over two years, additional mechanistic data, and supportive evidence from Zolgensma’s efficacy and safety record. FDA says the manufacturer provided sufficient justification to extend use to adults with SMA, though the agency cautioned that adults with chronic medical conditions may face higher risks of liver and cardiac side effects. The Itvisma label includes a Boxed Warning for hepatotoxicity similar to that of Zolgensma, reflecting observed liver-related adverse events in clinical studies.