Two children die of liver failure after being treated with most expensive gene therapy drug Zolgensma, cause under investigation

On August 11, 2022, two children died of acute liver failure under further investigation after treatment with Novartis' (NYSE: NVS) gene therapy drug Zolgensma (onasemnogene abeparvovec) for spinal muscular atrophy in Russia and Kazakhsta.

In May 2019, AveXis, a subsidiary of Novartis, announced that the gene therapy drug Zolgensma was approved by the FDA for the treatment of Spinal Muscular Atrophy (SMA) patients under 2 years of ag.

In January 2022, Novartis (Novartis, NYSE: NVS) submitted a clinical trial application for Spinal Muscular Atrophy (SMA) AAV gene therapy drug OAV101 injection (Zolgensma) in China, which has obtained an implied license for clinical trial.

The data of Zolgensma’s Phase 3 STR1VE trial abroad and the completed Phase 1 START trial data show that Zolgensma significantly improves the survival rate of patient.

Related reports:

JAMA Neurol: Spinal muscular atrophy patients still achieve long-term clinical remission 5 years after Zolgensma treatment

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About SMA

SMA is a rare inherited neuromuscular disorder, the most common genetic disorder that causes death in children, caused by mutations in the SMN1 gene, which encodes the survivin motor neuron protei.