AveXis presents Zolgensma® data at EPNS

Janssen presents initial results for BCMA CAR-T therapy

AveXis has presented new data on Zolgensma® at the 2019 European Paediatric Neurology Society (EPNS) Congress. The data continue to show the 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 the SPR1NT study supports critical importance of early intervention in pre-symptomatic SMA patients, leading to age appropriate major milestone gain.

In addition, updated results from the 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.

An additional oral presentation highlighted interim results from the long-term follow-up of the Phase 1 START study. Patients in the 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 .

SMA is a rare and devastating genetic disease that leads to progressive muscle weakness, paralysis and, when left untreated in its most severe form (Type 1), death. The disease affects 550-600 infants in Europe.

Dr Olga Santiago, Chief Medical Officer, AveXis, said: “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.

“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.”

The 2019 EPNS Congress takes place in Athens, Greece, from 17-21 September.