Drug

Eflornithine

Approved · EMA

Emerging researchLate-stage developmentRising momentum

Also known as 2-(difluoromethyl)ornithine, DFMO, Difluoromethylornithine, Iwilfin+3 more

2-(difluoromethyl)ornithine, DFMO, Difluoromethylornithine, Iwilfin, Vaniqa, alpha-difluoromethylornithine, (RS)-2,5-diamino-2-(difluoromethyl)pentanoic acid.

RxNorm569 ChEMBLCHEMBL830 ATCD11AX16 ATCL01XX79 ATCP01CX03UNIIZQN1G5V6SR

Research papers

Active clinical trials

Regulatory events

Latest activity

beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A Phase 1b/2 Trial Investigating the Safety and Efficacy of Oral AMXT 1501 and Oral DFMO in Combination With Standard of Care in Patients With Advanced Solid Tumors Who Progressed After Prior Therapies

Clinical trial2026-07-15Results expected Q1 2028 · ClinicalTrials.gov

A Phase II Open Label Basket Trial Study Using Eflornithine (DFMO) for Ewing Sarcoma and Osteosarcoma

Clinical trial2026-07-09Results expected Q2 2031 · ClinicalTrials.gov

Phase II Trial of Eflornithine (DFMO) and Etoposide for Relapsed/Refractory Neuroblastoma

Clinical trial2026-07-08Results expected Q4 2028 · ClinicalTrials.gov

A Phase I/II Study Using Eflornithine (DFMO) and AMXT 1501 for Relapsed and Refractory Neuroblastoma, CNS Tumors, and Sarcomas

Clinical trial2026-07-01Results expected Q2 2033 · ClinicalTrials.gov

An Open-label, Phase 1b Study to Evaluate the Safety and Tolerability of Eflornithine Plus Temozolomide in Patients With Newly Diagnosed Glioblastoma or Astrocytoma

Clinical trial2026-06-30Primary completion · ClinicalTrials.gov

Phase I/IIa Trial of Retifanlimab and Difluoromethylornithine (DFMO) in Patients With Progressive High-Grade Glioma

Clinical trial2026-06-24Results expected Q4 2030 · ClinicalTrials.gov

TArgeting Type 1 Diabetes Using POLyamines (TADPOL): A Randomized, Double-Masked, Placebo-Controlled Phase 2 Study to Evaluate the Efficacy and Safety of Difluoromethylornithine (DFMO) to Preserve Insulin Production in Type 1 Diabetes

Clinical trial2026-06-22Results expected Q4 2027 · ClinicalTrials.gov

Eflornithine plus Sulindac for Prevention of Progression in Familial Adenomatous Polyposis.

Research2020-09-01The New England journal of medicine

A subset analysis of a phase II trial evaluating the use of DFMO as maintenance therapy for high-risk neuroblastoma.

Research2020-05-24International journal of cancer

The effect of difluoromethylornithine on decreasing prostate size and polyamines in men: results of a year-long phase IIb randomized placebo-controlled chemoprevention trial.

Research2008-02-01Cancer epidemiology, biomarkers & prevention : a publication of the American Association for Cancer Research, cosponsored by the American Society of Preventive Oncology

Approval: Vaniqa (EMA)

Regulatory2001-03-19EMA

Recent advances in the biochemistry of polyamines in eukaryotes.

Research1986-03-01The Biochemical journal

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name

Eflornithine

Aliases & brands

2-(difluoromethyl)ornithineDFMODifluoromethylornithineIwilfinVaniqaalpha-difluoromethylornithine(RS)-2,5-diamino-2-(difluoromethyl)pentanoic acid

RxNorm CUI

569

ChEMBL ID

CHEMBL830

ATC codes

D11AX16L01XX79P01CX03

UNII

ZQN1G5V6SR

Regulatory jurisdictions

ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval

2001-03-19

Latest approval

2001-03-19

Authorities

EMA

Total events

emaEuropean Medicines Agency· 1 event

2001-03-19Approval

Approval: Vaniqa (EMA)

Indication: Treatment of facial hirsutism in women.

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

38 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme

CLINICALTRIALS.GOV · LIVE REGISTRY

registered trials across all phases

LATEST COMPLETION 2024

Active studies

Recruiting

Late-stage (III+)

Completed

Discontinued

PHASE DISTRIBUTIONn = 38

Early Phase 11Phase 15Phase 1 / 27Phase 218Phase 2 / 31Phase 36

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

A Randomized Phase 3 Open-Label Study To Evaluate the Efficacy and Safety of Eflornithine With Lomustine Compared to Lomustine Alone in Patients With AA That Progress/Recur After Irradiation and Adjuvant Temozolomide Chemotherapy

Unknown statusPH IIIOrbus Therapeutics, Inc.n=3432023

Phase III Trial of the Safety and Efficacy of Eflornithine Combined With Sulindac Compared to Eflornithine, Sulindac as Single Agents in Patients With Familial Adenomatous Polyposis

CompletedPH IIICancer Prevention Pharmaceuticals, Inc.n=1712019

Efficacy and Safety of Fexinidazole Compared to Nifurtimox-Eflornithine Combination Therapy (NECT) in Patients With Late-stage Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: Pivotal, Non-inferiority, Multicentre, Randomised, Open-label Study

CompletedPH II/IIIDrugs for Neglected Diseasesn=3942017

A Phase III Randomized, Double-Blind, Placebo-Controlled Clinical Trial of the Combination of DFMO and Sulindac to Decrease the Rate of Recurrence of Adenomatous Polyps in the Colon

CompletedPH IIINational Cancer Institute (NCI)n=3752008

Clinical Study Comparing the Nifurtimox-Eflornithine Combination With the Standard Eflornithine Regimen for the Treatment of Trypanosoma Brucei Gambiense Human African Trypanosomiasis in the Meningoencephalitic Phase

CompletedPH IIIDrugs for Neglected Diseasesn=2802008

Recent completions

Trials that read out recently, adding to the completed evidence base.

Targeted Chemoprevention of Gastric Carcinogenesis in High Risk Populations

CompletedPH IIVanderbilt-Ingram Cancer Centern=912024

A Phase I/II Trial of DFMO in Combination With Bortezomib in Patients With Relapsed or Refractory Neuroblastoma

CompletedPH I/IIGiselle Shollern=162024

N2012-01: Phase 1 Study of Difluoromethylornithine (DFMO) and Celecoxib With Cyclophosphamide/Topotecan for Patients With Relapsed or Refractory Neuroblastoma

CompletedPH INew Approaches to Neuroblastoma Therapy Consortiumn=302023

A Phase II Preventative Trial of DFMO (Eflornithine HCl) as a Single Agent in Patients With High Risk Neuroblastoma in Remission

CompletedPH IIGiselle Shollern=1402023

Phase I Dose-Finding, Safety Study of Oral AMXT 1501 Dicaprate and Difluoromethylornithine (DFMO) in Patients With Advanced Solid Tumors

CompletedPH IAminex Therapeutics, Inc.n=562023

Targeting Polyamines Using DFMO in Persons With Type 1 Diabetes: A Randomized, Double-Masked, Placebo-Controlled Phase I Study to Evaluate the Safety, Tolerability, and Initial Pharmacodynamics of Multiple Ascending Doses

CompletedPH IIndiana Universityn=412020

Research activity

4 papers

Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.

Publications over time

19862020

Most influential

Recent advances in the biochemistry of polyamines in eukaryotes.

The Biochemical journal · 1986 · 1,096 cites

The effect of difluoromethylornithine on decreasing prostate size and polyamines in men: results of a year-long phase IIb randomized placebo-controlled chemoprevention trial.

Cancer epidemiology, biomarkers & prevention : a publication of the American Association for Cancer Research, cosponsored by the American Society of Preventive Oncology · 2008 · 70 cites

Eflornithine plus Sulindac for Prevention of Progression in Familial Adenomatous Polyposis.

The New England journal of medicine · 2020 · 54 cites

A subset analysis of a phase II trial evaluating the use of DFMO as maintenance therapy for high-risk neuroblastoma.

International journal of cancer · 2020 · 49 cites

Related drugs

1 matches

Drugs sharing diseases, protein targets and literature with this one. Ranked by graph evidence (shared targets + diseases weighted above co-mentions).

Ornithine

Frequently co-studied with Eflornithine in the research literature.

1 shared paper

References & data sources

RxNorm (U.S. National Library of Medicine) — drug identity
ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
Europe PMC — research literature
ClinicalTrials.gov — clinical trials
Regulatory event sources are credited in the Regulatory Timeline above.