Eflornithine — Drug Monograph
Brand names: Iwilfin
Drug class: Other
Mechanism of Action
Irreversible inhibitor of ornithine decarboxylase (ODC), the rate-limiting enzyme in polyamine biosynthesis. ODC is a transcriptional target of the MYCN oncogene, which is amplified in high-risk neuroblastoma. By depleting the polyamines (putrescine, spermidine, spermine) that tumor cells need for proliferation and survival, eflornithine suppresses residual disease and reduces the risk of relapse.
FDA Indications
- To reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma (HRNB) who have achieved at least a partial response to prior multiagent, multimodality therapy including anti-GD2 immunotherapy (FDA approved 2023)
Common Side Effects
- Hearing loss / otitis media
- Pyrexia
- Diarrhea
- Cough
- Vomiting
- Elevated ALT/AST
Clinical Pearl
Eflornithine is the first oral maintenance therapy approved to reduce relapse in high-risk neuroblastoma after completion of anti-GD2 immunotherapy. It targets the MYCN-driven polyamine pathway via ODC inhibition — a fundamentally different mechanism from the cytotoxic and immunotherapy backbone that precedes it.