Two Children Died After Zolgensma Gene Therapy

Novartis reported that two children died approximately five to six weeks after receiving an infusion of Zolgensma (onasemnogene abeparvovec-xioi), its gene therapy for spinal muscular atrophy (SMA).

The company notified the FDA and other regulators about the deaths from acute liver failure that occurred in Russia and Kazakhstan.

Zolgensma received FDA approval in May 2019 for treatment of pediatric patients less than two years old with SMA. The approval included limitations of use, including on repeat administrations of the therapy, because of concerns about efficacy and safety.

The drug’s prescribing information includes a boxed warning about the risk of acute serious liver injury and of acute liver failure.