Intrathecal onasemnogene abeparvovec shows promise in older children with SMA

Novartis has announced that its gene therapy, onasemnogene abeparvovec (OAV101 IT), has shown positive results in a late-stage trial for children aged 2 to 18 with spinal muscular atrophy (SMA).¹ This rare disorder impairs neuromuscular development, hindering patients’ ability to walk, talk and swallow. The therapy was approved in the US for treating children under 2 years of age in 209, after the FDA granted it Fast Track, Breakthrough Therapy and Priority Review designations.²  

In results from the phase III STEER study (NCT05089656), patients demonstrated improvements in motor functions such as sitting, crawling and standing compared to those who received a sham control. SMA, the leading genetic cause of infant deaths, affects roughly 1 in 10,000 people and is divided into five types based on symptom onset.³ The trial involved over 100 patients with type 2 SMA who had not received any prior treatment and could sit but had never walked. Common side effects included upper respiratory tract infections, fever and vomiting. 

Novartis intends to present the trial results to regulatory agencies next year and data are expected to be presented at a medical meeting this year.

References:

1. Novartis intrathecal onasemnogene abeparvovec Phase III study meets primary endpoint in children and young adults with SMA. Novartis. Available at: https://www.reuters.com/business/healthcare-pharmaceuticals/novartis-gene-therapy-helps-children-with-rare-muscle-disorder-study-2024-12-30/ (accessed 7 January 2025).
2. FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality. FDA.gov. Available at: https://www.fda.gov/news-events/press-announcements/fda-approves-innovative-gene-therapy-treat-pediatric-patients-spinal-muscular-atrophy-rare-disease (accessed 7 January 2025).
3. Nishio H, Tabe Eko Niba E, Saito T, et al. Spinal Muscular Atrophy: The Past, Present, and Future of Diagnosis and Treatment. Int J Mol Sci. 2023;24:11939. doi: 10.3390/ijms241511939.

Disclosures: This article was created by the touchNEUROLOGY team utilizing AI as an editorial tool (ChatGPT (GPT-4o) [Large language model]. https://chat.openai.com/chat.) The content was developed and edited by human editors. No funding was received in the publication of this article.