Between January 2019 and September 2023, the pharmacovigilance database of the European Medicines Agency collected 661 individual reports of adverse reactions linked to Zolgensma®, representing a total of 2744 adverse reactions.
- In more than 92% of cases, only Zolgensma® was suspected.
- More than 50% of adverse reactions were classified as serious: 21% resulted in hospitalisation or prolonged hospitalisation, and 4.3% were life-threatening.
- One hundred and thirty adverse events were fatal in 39 children, 32 of whom were aged between two months and two years. No fatal adverse events were reported in children aged 3 to 11 years.
- The most common adverse events were fever (6.3%), vomiting (5.1%), increased aspartate aminotransferase (ASAT) (4.70%), increased alanine aminotransferase (ALAT) (4.40%), thrombocytopenia (4.30%).
- There were 59 adverse cardiac events, including 14 tachycardias, 11 bradycardias, nine cardiac arrests and four cardiorespiratory arrests.
The authors conclude that hepatic and cardiac monitoring is necessary when taking Zolgensma®. They emphasise that the rarity of a disease makes post-marketing pharmacovigilance all the more important.
