A 2nd Patient Death Reported in Novartis’ Gene Therapy Trial

Just as Novartis and AveXis’ gene therapy, Zolgensma (onasemnogene abeparvovec-xioi) for spinal muscular atrophy (SMA) is progressing toward approval, a second patient death was reported. Last week, AveXis, a Novartis  company, announced interim data from its Phase III STR1VE clinical trial of Zolgensma in SMA Type 1. SMA is a severe neuromuscular disease caused by a mutation in the SMN1 gene, which codes for SMN, a protein necessary for motor neuron function. The disease is noted by the loss of motor neurons, which leads to progressive muscle weakness and paralysis. It is rare, occurring in about one in 10,000 live births. Type 1 is lethal and typically results in death by the age of two years. Zolgensma is a gene therapy in development as a one-time infusion for SMA Type 1. It uses a virus vector to deliver a copy of the human SMN gene. In December, the Food and Drug Administration (FDA) accepted AveXis’ Biologics License Application (BLA) for Zolgensma, with a target action date in May 2019. It is also expected to be approved in Japan and the European Union later this year.

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