Novartis SMA gene therapy shows sustained benefit in late-stage studies

Novartis (NYSE:NVS) unit AveXis announces updated data from the Phase 3 SPR1NT and STR1VE studies evaluating gene therapy Zolgensma (onasemnogene abeparvovec-xioi) in patients with spinal muscular atrophy type 1 (SMA1). The results were presented at the European Neurology Society Congress in Athens.

Zolgensma continues to demonstrate significant therapeutic benefit in prolonging event-free survival compared to natural history.

Data support the critical importance of early intervention in presymptomatic SMA patients.

Patients in long-term follow-up study continue to achieve developmental milestones.

The FDA approved Zolgensma in May for SMA.

https://seekingalpha.com/news/3500405-novartis-sma-gene-therapy-shows-sustained-benefit-late-stage-studies