Has Zolgensma had safety issues since approval?
Yes — in August 2022 Novartis reported the first two deaths from acute liver failure after more than 2,300 treatments, prefiguring the broader AAV liver-toxicity concerns that later emerged with Sarepta's Elevidys.
Yes. Acute liver injury was a known risk on the label, which is why patients receive steroids around the infusion. In August 2022 Novartis reported the first two deaths from acute liver failure, in patients in Russia and Kazakhstan, occurring five to six weeks after infusion and after steroid tapering. Novartis said it would add the deaths to the label and that the therapy's benefit-risk remained favourable after more than 2,300 treatments. European regulators subsequently considered new liver monitoring guidance. These risks are shared by other AAV gene therapies, as Sarepta's Elevidys later showed. For families, the risks are weighed against a disease that is often fatal without treatment.