An increasing number of targeted agents are now entering early phase paediatric trials including the lead Novartis MDM2 antagonist, HDM‐201, as part of the Next Generation Personalised Neuroblastoma Therapy (NEPENTHE) trial (NCT02780128) and the dual MDM2/MDMX inhibitor ALRN‐6924 (NCT03654716). The gene discussed is MDM2; the disease is neuroblastoma.