Has Zolgensma had safety problems?
In August 2022 Novartis reported the first two deaths from acute liver failure after Zolgensma treatment — foreshadowing the broader concerns about liver toxicity with AAV gene therapies that later surfaced dramatically with Sarepta's Elevidys.
Yes. Acute liver injury was a known risk listed on the label, but in August 2022 Novartis reported the first two deaths from acute liver failure, in patients in Russia and Kazakhstan, five to six weeks after infusion and following tapering of the steroids given alongside treatment.
Novartis said it would update the label to mention the deaths while maintaining that the therapy's overall risk-benefit profile is favourable, noting it had by then been used in more than 2,300 patients. The episode foreshadowed the concerns about liver toxicity with adeno-associated virus (AAV) gene therapies that later surfaced dramatically with Sarepta's Elevidys. For regulators, it underlined that one-time therapies still require long-term safety monitoring.