carglumic acid   Click here for help

GtoPdb Ligand ID: 7458

Synonyms: N-carbamylglutamate | Carbaglu® | OE-312
Approved drug
carglumic acid is an approved drug (EMA (2003), FDA (2010))
Compound class: Synthetic organic
Comment: Carglumic acid is an orphan drug.
2D Structure
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Physico-chemical Properties
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Hydrogen bond acceptors 7
Hydrogen bond donors 4
Rotatable bonds 6
Topological polar surface area 129.72
Molecular weight 190.06
XLogP -1.78
No. Lipinski's rules broken 0
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Canonical SMILES OC(=O)CCC(C(=O)O)NC(=O)N
Isomeric SMILES OC(=O)CC[C@@H](C(=O)O)NC(=O)N
InChI InChI=1S/C6H10N2O5/c7-6(13)8-3(5(11)12)1-2-4(9)10/h3H,1-2H2,(H,9,10)(H,11,12)(H3,7,8,13)/t3-/m0/s1
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Summary of Clinical Use Click here for help
Approved to treat hyperammonaemia in patients with N-acetylglutamate synthase deficiency, or of other aetiologies.
Mechanism Of Action and Pharmacodynamic Effects Click here for help
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.
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