AveXis presents new data at EPNS continuing to show significant therapeutic benefit of Zolgensma in prolonging event-free survival now up to 5 years of age in patients with spinal muscular atrophy (SMA) Type 1

New interim data from SPR1NT study supports critical importance of early intervention in pre-symptomatic SMA patients, leading to age-appropriate major milestone gain. Updated results from global STR1VE study demonstrate that Zolgensma (onasemnogene abeparvovec-xioi) has significant therapeutic benefit in prolonging event-free survival in SMA Type 1 patients versus natural history Patients in START long-term follow-up study (cohort 2), who are an average age of 4.2 years (oldest patient is 5 years), continue to achieve developmental milestones Basel, September 19, 2019 - AveXis, a Novartis company, today announced that new interim data from the Phase 3 SPR1NT trial in pre-symptomatic patients as well as interim data from the ongoing Phase 3 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 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 Paediatric 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 Olga Santiago, M.D. Chief Medical Officer, AveXis.