Zolgensma (onasemnogene abeparvovec) is the first approved gene therapy treatment in SMA via intravenous administration.
A new trial of the intrathecal administration gene therapy product is planned in over 100 participants with type II SMA, treatment-naïve and aged 2 to 18 years, able to sit up but having never walked. This randomized, double-blind, phase III STEER trial against a sham procedure will take place over an initial period of one year. At this time, patients who have received the sham procedure will receive the treatment and vice versa.
This trial is based on data from the STRONG trial, which shows significant improvement in driving force and clinical response in participants (aged 2 to 5 years). This phase I / II trial was partially suspended following inflammation of spinal ganglia in non-human primates, but the FDA has just lifted this hold.
Novartis announces lift of partial clinical trial hold and plans to initiate a new, pivotal Phase 3 study of intrathecal OAV-101 in older patients with SMA. Novartis press release, Aug 3rd, 2021.
