eflornithine   Click here for help

GtoPdb Ligand ID: 5176

Synonyms: 2-(difluoromethyl)ornithine (DMFO) | difluoromethylornithine | Iwilfin® | Ornidyl® | Vaniqa®
Approved drug
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.
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2D Structure
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Physico-chemical Properties
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Hydrogen bond acceptors 4
Hydrogen bond donors 3
Rotatable bonds 5
Topological polar surface area 89.34
Molecular weight 182.09
XLogP -2.71
No. Lipinski's rules broken 0
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Canonical SMILES NCCCC(C(=O)O)(C(F)F)N
InChI InChI=1S/C6H12F2N2O2/c7-4(8)6(10,5(11)12)2-1-3-9/h4H,1-3,9-10H2,(H,11,12)
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Summary of Clinical Use Click here for help
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 Click here for help
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].
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