New data show significant therapeutic benefit of Zolgensma in prolonging event-free survival in SMA Type 1

AveXis, which was  acquired in an $8.7 billion deal by Swiss pharma giant Novartis (NOVN: VX) last year, today announced that new interim data from the Phase III SPR1NT trial in pre-symptomatic patients as well as interim data from the ongoing Phase III STR1VE clinical program for Zolgensma (onasemnogene abeparvovec-xioi) showed positive outcomes, demonstrating age‑appropriate major milestone gain with pre‑symptomatic treatment and prolonged event-free survival in patients with spinal muscular atrophy (SMA) Type 1.

An additional oral presentation highlighted interim results from the long-term follow-up of the Phase 1 START study. These data will be presented during the 2019 European Pediatric Neurology Society (EPNS) Congress.

"For families who never expected their children to reach meaningful motor milestones, the results we’re presenting at EPNS demonstrate the life-changing impact Zolgensma can have on children with SMA Type 1,” said Dr Olga Santiago, chief medical officer, AveXis. “It is critical to diagnose SMA and begin treatment as early as possible in order to stop irreversible motor neuron loss and make the achievement of major motor milestones such as crawling, sitting and walking a possibility,” she noted.