Drug intelligence / Profile preview

eflornithine

Development stage
Approved
Lead developer
Bristol Myers Squibb
Modality
Small Molecules
Administration
Oral, Intravenous, Topical
01

Overview

Eflornithine is a small molecule drug that acts as an irreversible inhibitor of ornithine decarboxylase (ODC), an enzyme involved in polyamine synthesis. By inhibiting ODC, it blocks the production of polyamines necessary for cell growth and proliferation. Eflornithine was originally developed to treat African trypanosomiasis (sleeping sickness) and later approved for topical use to reduce unwanted facial hair in women (hirsutism). More recently, it has been approved as oral maintenance therapy to reduce relapse risk in adults and children with high-risk neuroblastoma who have responded at least partially to prior multiagent therapy including anti-GD2 immunotherapy. The drug is available in both topical and systemic formulations[1][3][4][5].

Brand names
VaniqaIwilfinFlorexaOrnidyl
Other names
eflornithine hydrochloride monohydrateeflornithine HCl hydratedifluoromethylornithine hydrochloride hydrate
02

Targets

ODC1 (Ornithine decarboxylase)

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