A new indication for gene therapy in proximal spinal muscular atrophy (SMA), involving a single intrathecal injection of Itvisma, has just been approved by the FDA (Food and Drug Administration) for patients over the age of two only.
This follows positive results from the STRENGTH and STEER Phase III clinical trials, which showed stabilization or improvement in motor function with this product in older patients (aged 2 to 18 years).
Zolgensma was already available for patients under two years of age, as a single intravenous injection.
This new product will be available in the United States in December. The European Medicines Agency (EMA) is currently reviewing the application and is expected to issue its opinion in mid-2026.
Novartis receives FDA approval for Itvisma®, the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA). Novartis. 24 November 2025 press release.
