Eflornithine

Summary

Eflornithine (CHEMBL830) is an approved small-molecule trypanocidal drug (ATC D11AX16) targeting ODC1; indicated across 20 conditions including trypanosomiasis and anaplastic astrocytoma.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: D11AX16 (+2 more)
• Targets: 1 (ODC1)
• Indications: 20 conditions
• Clinical trials: 42
• Chemistry: 182.17 Da · C6H12F2N2O2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL830
Name	Eflornithine
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	3009
ChEBI	CHEBI:41948
ATC	D11AX16, L01XX79, P01CX03
Molecular formula	C6H12F2N2O2
Molecular weight	182.17
InChIKey	VLCYCQAOQCDTCN-UHFFFAOYSA-N

SMILES: C(CC(C(F)F)(C(=O)O)N)CN

IUPAC name: 2,5-diamino-2-(difluoromethyl)pentanoic acid

ChEBI definition: A fluoroamino acid that is ornithine substituted by a difluoromethyl group at position 2.

Pharmacological roles (ChEBI): trypanocidal drug.

Also known as: Eflornithine, Eflornitina, alpha-difluoromethylornithine, dl-alpha-difluoroornithine, eflornithine, Rac-eflornithine, SID90341137, dfmo, SID575150, difluoromethylornithine, alpha-Difluoromethylornithine, DFMO

Parent form; salt/anhydrous children: CHEMBL1201037

Patent coverage: 7,960 distinct patent families (27,744 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
ODC1	Ornithine decarboxylase	Inhibition	4.89		P11926

Broader ChEMBL bioactivity targets: 1 (assay-derived). Sample: Ornithine decarboxylase.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

Aggregated over 1 target gene(s): ODC1.

Top Reactome pathways

2 total, by targets touching each:

Pathway	Targets	Genes
Regulation of ornithine decarboxylase (ODC)	1	ODC1
Metabolism of polyamines	1	ODC1

Dominant GO biological processes

GO term	Targets
kidney development	1
polyamine metabolic process	1
cell population proliferation	1
positive regulation of cell population proliferation	1
response to virus	1
obsolete putrescine biosynthetic process from arginine, via ornithine	1
regulation of protein catabolic process	1
polyamine biosynthetic process	1

Indications & clinical

Indications

3 approved indications. FDA phase 4, plus an anticancer drug’s labelled cancer uses (which ChEMBL often logs at phase 3).

Indication	Phase	MONDO	EFO
colorectal neoplasm	3	MONDO:0005335	EFO:0004142
precancerous condition	3	MONDO:0021074	MONDO:0021074
neoplasm	3	MONDO:0005070	EFO:0000616

9 diseases in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
trypanosomiasis	3	MONDO:0000940	DOID:10113
anaplastic astrocytoma	3	MONDO:0016684	EFO:0002499
leishmaniasis	3	MONDO:0011989	EFO:0005044
neuroblastoma	2	MONDO:0005072	EFO:0000621
human African trypanosomiasis	2	MONDO:0005459	EFO:0005225
gastric neoplasm	2	MONDO:0021085	MONDO:0001056
type 1 diabetes mellitus	2	MONDO:0005147	MONDO:0005147
carcinoma	1	MONDO:0004993	EFO:0000313
glioblastoma	1	MONDO:0018177	EFO:0000519

6 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 42.

Phase distribution

Phase	Trials
PHASE2	20
PHASE3	7
PHASE1/PHASE2	7
PHASE1	4
PHASE4	2
PHASE2/PHASE3	1
EARLY_PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT06976424	PHASE4	RECRUITING	Comparison of a Face Cream Plus a Laser Procedure and Face Cream Alone in Treatment of Excessive Face Hairs in Female
NCT04403282	PHASE4	TERMINATED	Topical Eflornithine for Pseudofolliculitis Barbae: Randomized Controlled Trial
NCT01349881	PHASE3	ACTIVE_NOT_RECRUITING	S0820, Adenoma and Second Primary Prevention Trial
NCT00003814	PHASE3	COMPLETED	Eflornithine in Treating Patients With Bladder Cancer
NCT00005884	PHASE3	COMPLETED	Eflornithine to Prevent Skin Cancer in Patients With Previously Treated Early Stage Skin Cancer
NCT00118365	PHASE3	COMPLETED	Eflornithine and Sulindac in Preventing Colorectal Cancer in Patients With Colon Polyps
NCT00146627	PHASE3	COMPLETED	Efficacy - Safety of Eflornithine-Nifurtimox Combination Versus Eflornithine to Treat Human African Trypanosomiasis
NCT01483144	PHASE3	COMPLETED	Trial of Eflornithine Plus Sulindac in Patients With Familial Adenomatous Polyposis (FAP)
NCT01685827	PHASE2/PHASE3	COMPLETED	Pivotal Study of Fexinidazole for Human African Trypanosomiasis in Stage 2
NCT02796261	PHASE3	UNKNOWN	Study to Evaluate Eflornithine + Lomustine vs Lomustine in Recurrent Anaplastic Astrocytoma (AA) Patients
NCT02559778	PHASE2	RECRUITING	Pediatric Precision Laboratory Advanced Neuroblastoma Therapy
NCT02679144	PHASE2	RECRUITING	Neuroblastoma Maintenance Therapy Trial
NCT04301843	PHASE2	RECRUITING	Eflornithine (DFMO) and Etoposide for Relapsed/Refractory Neuroblastoma
NCT04696029	PHASE2	RECRUITING	DFMO as Maintenance Therapy for Molecular High/Very High Risk and Relapsed Medulloblastoma
NCT05594563	PHASE2	RECRUITING	TArgeting Type 1 Diabetes Using POLyamines (TADPOL)
NCT06059118	PHASE2	RECRUITING	Difluoromethylornithine and High Dose Testosterone With Enzalutamide in Metastatic Castration-Resistant Prostate Cancer
NCT06465199	PHASE1/PHASE2	RECRUITING	Eflornithine (DFMO) and AMXT 1501 for Neuroblastoma, CNS Tumors, and Sarcomas
NCT06892678	PHASE1/PHASE2	RECRUITING	DFMO Maintenance for Patients With Relapsed/Refractory Ewing Sarcoma or Osteosarcoma
NCT07287917	PHASE1/PHASE2	RECRUITING	Study of AMXT 1501 and DFMO in Combination With Standard Therapies in Advanced Solid Tumors
NCT07321912	PHASE2	RECRUITING	Eflornithine (DFMO) for Ewing Sarcoma and Osteosarcoma
NCT07468136	PHASE1/PHASE2	NOT_YET_RECRUITING	Retifanlimab With or Without Difluoromethylornithine for the Treatment of Progressive High Grade Gliomas
NCT00003076	PHASE2	COMPLETED	Eflornithine to Prevent Cancer in Patients With Barrett’s Esophagus
NCT00006079	PHASE2	COMPLETED	Eflornithine To Prevent Cervical Cancer in Patients With Cervical Intraepithelial Neoplasia
NCT00006101	PHASE2	COMPLETED	Eflornithine in Treating Patients At High Risk of Developing Prostate Cancer
NCT00021294	PHASE2	COMPLETED	Eflornithine With or Without Triamcinolone in Preventing Nonmelanoma Skin Cancer in Patients With Actinic Keratosis
NCT00033371	PHASE2	COMPLETED	Celecoxib With or Without Eflornithine in Preventing Colorectal Cancer in Patients With Familial Adenomatous Polyposis
NCT00086736	PHASE2	COMPLETED	Neoadjuvant Eflornithine and Bicalutamide Compared With Eflornithine Alone, Bicalutamide Alone, and No Neoadjuvant Therapy in Treating Patients With Localized Prostate Cancer Undergoing Brachytherapy or Radical Prostatectomy
NCT00204789	PHASE2	COMPLETED	Difluoromethylornithine (DFMO) Chemoprevention of Skin Cancer in Organ Transplant Recipients
NCT00601640	PHASE2	COMPLETED	Eflornithine and/or Diclofenac in Treating Patients With Sun-Damaged Skin
NCT00983580	PHASE2	COMPLETED	Acetylsalicylic Acid and Eflornithine in Treating Patients at High Risk for Colorectal Cancer
NCT01636128	PHASE2	WITHDRAWN	Urinary Biomarker Study With Sulindac and Difluoromethylornithine
NCT02139397	PHASE1/PHASE2	COMPLETED	Study of Difluoromethylornithine (DFMO) in Combination With Bortezomib for Relapsed or Refractory Neuroblastoma
NCT02395666	PHASE2	COMPLETED	Preventative Trial of Difluoromethylornithine (DFMO) in High Risk Patients With Neuroblastoma That is in Remission
NCT02794428	PHASE2	COMPLETED	Chemoprevention of Gastric Carcinogenesis
NCT05500508	PHASE1/PHASE2	TERMINATED	Oral AMXT 1501 Dicaprate in Combination With IV DFMO
NCT06219174	PHASE1/PHASE2	SUSPENDED	Targeting ODC as an Immunotherapeutic Target in STK11 (LKB1) Pathway-Deficient NSCLC (DFMO)
NCT06957834	PHASE2	COMPLETED	Efficacy of Eflornithine Hydrochloride Cream in the Treatment of Melasma: A Randomized, Double-blinded, Split-face Controlled Study
NCT01059071	PHASE1	COMPLETED	Safety Study for Refractory or Relapsed Neuroblastoma With DFMO Alone and in Combination With Etoposide
NCT02030964	PHASE1	COMPLETED	N2012-01: Phase 1 Study of Difluoromethylornithine (DFMO) and Celecoxib With Cyclophosphamide/Topotecan
NCT02384889	PHASE1	COMPLETED	DFMO in Children With Type 1 Diabetes
NCT03536728	PHASE1	COMPLETED	Oral AMXT 1501 Dicaprate in Combination With DFMO
NCT05717153	EARLY_PHASE1	RECRUITING	Intratumoral Extracellular Metabolic Impact of DFMO and AMXT 1501 in Patients With Diffuse or High Grade Glioma

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

Related molecules

Related molecules

Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.

4 molecules share ≥1 primary target. Top 4 by shared-target count:

Molecule	Source	Status	Shared targets
RESVERATROL	ChEMBL + PubChem	Phase 3 (approved)	ODC1
GENISTEIN	ChEMBL + PubChem	Phase 2 (approved)	ODC1
EFLORNITHINE, (S)-	ChEMBL	Phase 2	ODC1
Vincristine	PubChem	Approved	ODC1