Agenzia Italiana del Farmaco
Direct communication with healthcare professionals on ZOLGENSMA (onasemnogene abeparvovec) - Direct communication with healthcare professionals on ZOLGENSMA (onasemnogene abeparvovec)
Direct communication with healthcare professionals on ZOLGENSMA (onasemnogene abeparvovec)
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.
Published on: 16 February 2023