Dinutuximab received a priority review and orphan drug designation. Approval was based on the results of an open-label trial in 226 patients with high-risk neuroblastoma that had at least a partial response to induction chemotherapy, autologous stem cell transplantation, and radiation. Patients were randomized to receive a combination of dinutuximab, GM-CSF, IL-2, and isotretinoin, or isotretinoin alone. At 2 years, the event-free survival rate, the primary endpoint, was 66% with the dinutuximab regimen and 46% with isotretinoin alone (p<0.01). The overall survival rate was 86% with the dinutuximab regimen compared to 75% with isotretinoin alone (p<0.02).2 The recommended dose of dinutuximab is 17.5 mg/m² daily infused IV over 10-20 hours for 4 consecutive days for up to 5 cycles. Dinutuximab can cause life-threatening infusion reactions, severe pain requiring treatment with IV opioids, peripheral neuropathy, capillary leak syndrome, visual disturbances, hemolytic-uremic syndrome, and other serious adverse effects. The cost for one 17.5 mg single-use vial is $7,500.3