Synonyms: 2-(difluoromethyl)ornithine (DMFO) | difluoromethylornithine | Iwilfin® | Ornidyl® | Vaniqa®
eflornithine is an approved drug (FDA (2000), EMA (2001))
Compound class:
Synthetic organic
Comment: Eflornithine is an irreversible ornithine decarboxylase inhibitor [1,4] and is a partial inhibitor of arginase enzymes [6]. Marketed formulations may contain eflornithine hydrochloride (PubChem CID 57004).
Eflornithine has recently been reported to provide neuroprotective effects in a mouse model of Alzheimer's disease (AD) [3]. The authors identify microglial-induced arginine depletion as a causative factor in AD pathophysiology in their model, focussing on inhibition of arginine catabolising enzymes as a novel mechanism by which to halt the neurodegeneration observed in AD. These findings suggest that eflornithine could be used as a starting point for medicinal chemists to identify novel compounds with anti-arginase activity in humans. ![]() Ligand Activity Visualisation ChartsThese are box plot that provide a unique visualisation, summarising all the activity data for a ligand taken from ChEMBL and GtoPdb across multiple targets and species. Click on a plot to see the median, interquartile range, low and high data points. A value of zero indicates that no data are available. A separate chart is created for each target, and where possible the algorithm tries to merge ChEMBL and GtoPdb targets by matching them on name and UniProt accession, for each available species. However, please note that inconsistency in naming of targets may lead to data for the same target being reported across multiple charts. ✖ |
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No information available. |
Summary of Clinical Use ![]() |
This drug is used topically to treat facial hirsutism (excessive hair growth) and by injection for African sleeping sickness (trypanosomiasis). The EMA granted eflornithine orphan designation in 2016 for the treament of glioma [2]. In December 2023, the FDA granted authorisation for eflornithine to be used as an oral maintenance therapy for high-risk neuroblastoma. |
Mechanism Of Action and Pharmacodynamic Effects ![]() |
Eflornithine irreversibly inhibits ornithine decarboxylase, an enzyme that catalyses the conversion of ornithine to putrescine, and plays an important role in cell division and proliferation in the hair follicle. In sleeping sickness it is believed eflornithine kills trypanosomes by inhibiting their ornithine decarboxylase enzyme, which in these organisms is required for cell division [4]. |
External links ![]() |
For extended ADME data see the following: Electronic Medicines Compendium (eMC) Drugs.com European Medicines Agency (EMA) |