82% of SMA children treated pre-symptomatically with Zolgensma achieved developmental motor milestones

Swiss Pharma giant Novartis (NOVN: VX) has announced positive new data about Zolgensma (onasemnogene abeparvovec), an essential, one-time treatment and the only gene therapy for spinal muscular atrophy (SMA), from two clinical trials including the completed two-copy cohort of the Phase III SPR1NT clinical trial that demonstrates age-appropriate development when used pre-symptomatically and rapid, clinically meaningful efficacy in symptomatic children, even those with severe SMA at baseline, as well as and the completed Phase III STR1VE-EU trial which demonstrated rapid improvements in motor function following treatment with Zolgensma.

Highlights of the trials’ results

The data will be presented at the European Academy for Neurology (EAN) Virtual Congress 2021 (June 19–22), but the main findings are as follows: