ZOLGENSMA (onasemnogene abeparvovec) - Fatal cases of acute liver failure.

Summary:

• Fatal cases of acute liver failure have been reported in patients treated with onasemnogene abeparvovec. 
• Liver function should be monitored before treatment and regularly for at least 3 months after infusion. 
• Promptly assess patients with worsening liver function tests and/or signs or symptoms of acute illness.  
• If patients do not respond adequately to corticosteroids, consult a pediatric gastroenterologist or hepatologist and consider adjustment of the corticosteroid regimen. 
• Corticosteroids should not be tapered until liver function tests become unremarkable (normal clinical examination, total bilirubin, and ALT and AST levels below 2 × ULN).  
• Inform caregivers about the serious risk of hepatic injury and the need for periodic monitoring of liver function.