Efficacy of N-carbamoyl-L-glutamic acid for the treatment of inherited metabolic disorders.

ABSTRACT:

Introduction

N-carbamoyl-L-glutamic acid (NCG) is a synthetic analogue of N-acetyl glutamate (NAG) that works effectively as a cofactor for carbamoyl phosphate synthase 1 and enhances ureagenesis by activating the urea cycle. NCG (brand name, Carbaglu) was recently approved by the United States Food and Drug Administration (US FDA) for the management of NAGS deficiency and by the European Medicines Agency (EMA) for the treatment of NAGS deficiency as well as for the treatment of hyperammonenia in propionic, methylmalonic and isovaleric acidemias in Europe.

Areas covered

The history, mechanism of action, and efficacy of this new drug are described. Moreover, clinical utility of NCG in a variety of inborn errors of metabolism with secondary NAGS deficiency is dis

SUBMITTER: Chapel-Crespo CC 

PROVIDER: S-EPMC6054484 | biostudies-literature | 2016

REPOSITORIES: biostudies-literature