Synonyms: N-carbamylglutamate | Carbaglu® | OE-312
carglumic acid is an approved drug (EMA (2003), FDA (2010))
Compound class:
Synthetic organic
Comment: Carglumic acid is an orphan drug.
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No information available. |
Summary of Clinical Use |
Approved to treat hyperammonaemia in patients with N-acetylglutamate synthase deficiency, or of other aetiologies. |
Mechanism Of Action and Pharmacodynamic Effects |
Carglumic acid is a synthetic structural analogue of N-acetylglutamate (NAG). NAG is an essential allosteric activator of carbamoyl-phosphate synthase 1 (CPS1, P31327) in the liver. CPS1 is a mitochondrial enzyme, responsible for catalysing the first step of the urea cycle, which converts ammonia into urea. Drug-induced enhanced activity of CPS1 therefore helps reduce blood ammonia levels in patients with hyperammonaemia. |
External links |
For extended ADME data see the following: Electronic Medicines Compendium (eMC) Drugs.com European Medicines Agency (EMA) |