NCIt definition : An orally active, synthetic structural analogue of N-acetylglutamate (NAG) and carbamoyl
phosphate synthetase 1 (CPS 1) activator, with ammonia lowering activity. NAG, which
is formed by the hepatic enzyme N-acetylglutamate synthase (NAGS), is an essential
allosteric activator of the enzyme carbamoyl phosphate synthetase 1 (CPS 1). CPS 1
plays an essential role in the urea cycle and converts ammonia into urea. Upon oral
administration, carglumic acid can replace NAG in NAGS deficient patients and activates
CPS 1, which prevents hyperammonaemia.;