Drug intelligence / Profile preview

N-(phosphonoacetyl)-L-ornithine

Development stage
Preclinical
Modality
Small Molecules
01

Overview

N-(Phosphonoacetyl)-L-Ornithine is a synthetic small molecule that acts as a potent, competitive bisubstrate analog inhibitor of ornithine transcarbamylase (OTC), an enzyme essential for the urea cycle. By mimicking the transition state of the OTC-catalyzed reaction, it binds to the catalytic site and disrupts enzymatic activity, thereby inhibiting conversion of ornithine and carbamoyl phosphate to citrulline. This compound has been used primarily in biochemical research to study OTC function and structure, with applications in understanding genetic disorders such as ornithine transcarbamylase deficiency (OTCD). It is not approved for clinical use but has been investigated experimentally for its inhibitory properties[1][5][7].

Other names
N(delta)-(phosphonoacetyl)-L-ornithine
02

Targets

OTC (Ornithine carbamoyltransferase)

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