2 Children Die After Taking Novartis Gene Therapy

Novartis says two children died of acute liver failure weeks after being administered Zolgensma (onasemnogene abeparvovec-xioi), a gene therapy indicated for treating patients under age 2 with spinal muscular atrophy. The therapy currently carries a Boxed Warning for acute liver failure. The deaths were first reported by Stat.

“While this is important safety information, it is not a new safety signal and we firmly believe in the overall favorable risk/benefit profile of Zolgensma, which to date has been used to treat more than 2,300 patients worldwide across clinical trials, managed access programs, and in the commercial setting,” the company said in statement.

Novartis says it is notifying FDA and other health authorities in countries where Zolgensma is approved. Additionally, the company is updating the approved label to describe the acute liver failure fatalities.