Gene therapy for SMA, available in the United States for patients of any age

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 IIIAu cours d’un essai clinique de phase III, un médicament, pour lequel on a déterminé lors d’essais antérieurs l’innocuité et le dosage optimum (essais de phase I et II), est administré à un grand groupe de malades, sur une longue durée, dans le but d’évaluer son efficacité thérapeutique en la comparant à celle d’un traitement de référence ou un placebo. Il permet aussi de mettre en évidence les interactions indésirables et les effets secondaires du traitement à moyen terme. Au terme de cet essai, le médicament peut obtenir une autorisation de mise sur le marché. 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.