# Eflornithine (DFMO)

Source: https://onco.cc/drugs/eflornithine/  
OnCo record `eflornithine` (Treatment). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Eflornithine (DFMO) is an old sleeping-sickness drug repurposed as the first oral maintenance therapy for high-risk neuroblastoma, approved in December 2023 to reduce relapse after immunotherapy.

## Summary

Approved 13 December 2023 (US WorldMeds) for adults and children with high-risk neuroblastoma who achieved at least a partial response to prior multiagent, multimodality therapy including anti-GD2 immunotherapy. Evidence: single-arm NMTRC003/003B (n=105) compared with an external control from ANBL0032 (EFS HR 0.48, OS HR 0.32), a controversial externally controlled approval. EU applications filed 2025. Two years of twice-daily tablets.

## Fields

- Kind: Treatment
- Status: approved
- Last checked: 2026-09-07
- Brand: Iwilfin
- Modality: Small-molecule ornithine decarboxylase inhibitor
- Mechanism: Irreversible inhibitor of ornithine decarboxylase (ODC1), a direct MYCN target, depleting polyamines required for neuroblastoma proliferation.
- Approvals: US 2023: Maintenance to reduce relapse risk in high-risk neuroblastoma after ≥PR to prior therapy including anti-GD2
- Dosing: Oral; Body-surface-area-based dose twice daily for 2 years (e.g. 768 mg twice daily for BSA >1.5 m2)

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Eflornithine
- FDA label (DailyMed): https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Eflornithine

## Connected records

- cancers: [High-risk neuroblastoma](https://onco.cc/cancers/neuroblastoma-high-risk/), [Neuroblastoma (paediatric)](https://onco.cc/cancers/neuroblastoma/)
- companies: [US WorldMeds](https://onco.cc/companies/us-worldmeds/)
- terms: [MYCN amplification](https://onco.cc/terms/mycn-amplification/)
- trials: [NMTRC003/003B (DFMO maintenance)](https://onco.cc/trials/nmtrc003/), [S0820, Adenoma and Second Primary Prevention Trial](https://onco.cc/trials/nct01349881/), [Study of AMXT 1501 and DFMO in Combination With Standard Therapies in Advanced Solid Tumors](https://onco.cc/trials/nct07287917/)
- bottlenecks: [No incentive to repurpose cheap drugs](https://onco.cc/bottlenecks/b-generic-repurposing/), [Rare and paediatric cancers without markets](https://onco.cc/bottlenecks/b-rare-cancers/)

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