Dinutuximab (ch14.18; Unituxin™) is a chimeric human-mouse monoclonal antibody 
that binds to the glycolipid antigen disialoganglioside, which is highly 
expressed on the surface of neuroblastoma cells. This intravenous drug is 
approved in the EU and USA as combination therapy with granulocyte-macrophage 
colony-stimulating factor (GM-CSF), interleukin (IL)-2 and isotretinoin for the 
postconsolidation treatment of patients with high-risk neuroblastoma. In a 
multinational, phase III study in this patient population, event-free survival 
(EFS) benefits with the dinutuximab-containing regimen versus isotretinoin alone 
were observed at the time of the primary (p = 0.0115) and confirmatory 
(p = 0.0330) efficacy analyses, although the observed p-value for the 
between-group difference in EFS for the primary efficacy analysis did not cross 
the prespecified boundary for statistical significance (p < 0.0108). Significant 
and sustained (5 years) overall survival benefits were seen with the 
dinutuximab-containing regimen versus isotretinoin alone. Despite pretreatment 
with analgesics, antihistamines and antipyretics, serious adverse reactions have 
been reported with the dinutuximab-containing regimen, with infusion reactions 
and neuropathy prompting the US FDA to issue boxed warnings. Dinutuximab 
administered in combination with GM-CSF, IL-2 and isotretinoin represents an 
important advance in the postconsolidation treatment of patients with high-risk 
neuroblastoma, with its benefits outweighing its risks in a patient population 
with a poor prognosis and limited therapeutic options.