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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.
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.
Irreversible inhibitor of ornithine decarboxylase (ODC1), a direct MYCN target, depleting polyamines required for neuroblastoma proliferation.
1.DFMO irreversibly inactivates ornithine decarboxylase
Oral, self-administered, so it is a Part D drug: covered through a stand-alone Part D plan or Medicare Advantage drug benefit, usually on the specialty tier with 25 to 33% coinsurance until the annual cap ($2,000 in 2025, $2,100 in 2026). Iwilfin for high-risk neuroblastoma maintenance; paediatric, so mostly Medicaid/CHIP and commercial family plans.
Covered for FDA-labelled and NCCN-listed uses, but almost always behind prior authorisation confirming diagnosis, biomarker and line of therapy; dispensed through a specialty pharmacy.
Part D out-of-pocket capped at $2,000 (2025) / $2,100 (2026). Medicare patients cannot use manufacturer co-pay cards; charity funds (PAN, HealthWell, CancerCare) and the Extra Help subsidy are the routes.
Sources: Medicare.gov: Drug coverage (Part D) · Medicare.gov: Costs for Medicare drug coverage (annual out-of-pocket cap). Not medical or financial advice; verify with your plan.
Sources: NICE search: eflornithine (DFMO). Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.
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First oral maintenance therapy; externally controlled evidence source
MAA submitted to EMA, UK, Australia, Switzerland source
| Region | Year | Indication |
|---|---|---|
| US | 2023 | Maintenance to reduce relapse risk in high-risk neuroblastoma after ≥PR to prior therapy including anti-GD2 |
| Adverse event | Any grade | Grade 3+ |
|---|---|---|
| Hearing loss | 27% | - |
| Otitis media | 27% | - |
| Neutropenia | 24% | - |
| Elevated ALT | 33% | - |
NMTRC003/003B; largely pre-existing from platinum. Events listed without rates were not read from a primary source; see the label. Blank cells mean the figure was not sourced, not that it is zero.
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Query for this drug: (TITLE:"Eflornithine" OR ABSTRACT:"Eflornithine" OR TITLE:"DFMO" OR ABSTRACT:"DFMO" OR TITLE:"Iwilfin" OR ABSTRACT:"Iwilfin") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Eflornithine (DFMO), not a curated reading list.
Shares High-risk neuroblastoma, Neuroblastoma (paediatric), Rare and paediatric cancers without markets.
Shares MYCN amplification, High-risk neuroblastoma, Neuroblastoma (paediatric).
Shares MYCN amplification, High-risk neuroblastoma, Neuroblastoma (paediatric).
Shares High-risk neuroblastoma, Neuroblastoma (paediatric), Rare and paediatric cancers without markets.
Shares High-risk neuroblastoma, Neuroblastoma (paediatric), Rare and paediatric cancers without markets.
Shares MYCN amplification, High-risk neuroblastoma, Neuroblastoma (paediatric).
Shares High-risk neuroblastoma, Neuroblastoma (paediatric), Rare and paediatric cancers without markets.