By Maitane Sardon


Novartis AG said that two children who received its gene therapy for a neuromuscular disease died after the treatment.

The patients developed acute liver failure about five to six weeks after being administered the gene therapy, Zolgensma, which is used to treat spinal muscular atrophy, a muscle-wasting condition.

"While acute liver failure is a known adverse event, these are the first fatal cases of acute liver failure," the Swiss pharma major said in an emailed statement Friday.

Novartis said the children had also received corticosteroids, which are used alongside Zolgensma to prevent complications that can lead to severe liver damage.

The deaths occurred in Russia and Kazakhstan, Novartis said.

"Pending health authority review and approval, the Zolgensma label will be updated to inform prescribers that fatal acute liver failure has been reported," Novartis said.


Write to Maitane Sardon at


(END) Dow Jones Newswires

August 12, 2022 05:20 ET (09:20 GMT)

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