Drug intelligence / Profile preview

Lorenzo's oil

Development stage
Phase 3
Lead developer
Croda International
Modality
Small Molecules
Administration
Oral
01

Overview

Lorenzo's oil is a combination drug consisting of a 4:1 mixture of glyceryl trioleate (the triacylglycerol form of oleic acid) and glyceryl trierucate (the triacylglycerol form of erucic acid), derived from olive and rapeseed oils[2][3][4]. It is used as an investigational therapy for adrenoleukodystrophy (ALD) and adrenomyeloneuropathy (AMN), rare inherited disorders that cause accumulation of very long-chain fatty acids (VLCFAs) in tissues due to mutations in the ABCD1 gene[1][9]. The mechanism involves competitive inhibition of endogenous VLCFA synthesis by providing unsaturated fatty acids that reduce their accumulation in plasma and tissues[9]. While it can lower VLCFA levels and may delay or prevent neurological symptoms if started before onset, it does not reverse established neurological damage. Its use is experimental; adverse effects include thrombocytopenia and possible liver enzyme elevations[7].

Brand names
Lorenzo's oil
Other names
Erucic acidOleic acidGlyceryl trierucateGlyceryl trioleateGlycerol trierucate oilGlycerol trioleate oilHuile de Lorenzo
02

Targets

ELOVL1

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