Zolgensma (onasemnogene abeparvovec), the gene therapy developed by Novartis for spinal muscular atrophy (SMA), has shown efficacy in a Phase 3 study of patients aged two to less than 18 years who are able to sit but have never walked independently. SMA is a genetic neuromuscular disease caused by the lack of a functional SMN1 gene that affects muscle functions and basic movement. Zolgensma was first approved in the US in 2019, and one year later in the EU, as a paediatric drug for children less than two years old and administered as a single, one-time infusion.
The newest study in patients with SMA Type 2, covers a much broader population. The data were disclosed on 30 December 2024. In the Phase 3 STEER study, more than 100 patients were randomised to receive an intrathecal injection of the therapy compared with patients who received a sham procedure. The study met its primary endpoint showing an increase in motor function of patients receiving the therapy compared with the control group. According to Novartis, the safety profile of the therapy was favourable.
“Maintaining motor function is a key goal for many older patients with SMA. This may allow them the capacity to continue to propel their electric wheelchair, feed themselves with intact hand to mouth function, and perform other activities of daily living as independently as possible, Crystal Proud, a principal trial investigator, said in a prepared statement.
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