Carglumic acid

Carglumic acid, sold under the brand name Carbaglu among others, is used for the treatment of hyperammonaemia.[1][4][5]

Carglumic acid
Clinical data
Trade namesCarbaglu, Ucedane
Other names(S)-2-ureidopentanedioic acid
AHFS/Drugs.comMonograph
License data
Pregnancy
category
  • AU: B1
Routes of
administration		By mouth
ATC code
Legal status
Legal status
Pharmacokinetic data
Bioavailability30%
Protein bindingUndetermined
MetabolismPartial
Elimination half-life4.3 to 9.5 hours
ExcretionFecal (60%) and kidney (9%, unchanged)
Identifiers
IUPAC name
  • (2S)-2-(carbamoylamino)pentanedioic acid
CAS Number
PubChem CID
IUPHAR/BPS
DrugBank
ChemSpider
UNII
KEGG
ChEBI
ChEMBL
CompTox Dashboard (EPA)
ECHA InfoCard100.116.323
Chemical and physical data
FormulaC6H10N2O5
Molar mass190.155 g·mol−1
3D model (JSmol)
SMILES
  • C(CC(=O)O)[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 Y
  • Key:LCQLHJZYVOQKHU-VKHMYHEASA-N Y
 NY (what is this?)  (verify)

Carglumic acid is a carbamoyl phosphate synthetase 1 (CPS 1) activator.[1]

The most common adverse effects include vomiting, abdominal pain, pyrexia (fever), and tonsillitis, anemia, diarrhea, ear infection, other infections, nasopharyngitis, decreased hemoglobin levels, and headache.[1]

It was approved for medical use in the United States in March 2010.[6] Carglumic acid is an orphan drug.[7][8]

Medical uses

Carglumic acid is indicated for the treatment of acute hyperammonemia and chronic hyperammonemia.[1][2][3]

References
  1. "Carbaglu- carglumic acid tablet". DailyMed. Retrieved 9 June 2021.
  2. "Carbaglu EPAR". European Medicines Agency (EMA). Retrieved 9 June 2021.
  3. "Ucedane EPAR". European Medicines Agency (EMA). Retrieved 9 June 2021.
  4. Caldovic L, Morizono H, Daikhin Y, Nissim I, McCarter RJ, Yudkoff M, Tuchman M (2004). "Restoration of ureagenesis in N-acetylglutamate synthase deficiency by N-carbamylglutamate". J Pediatr. 145 (4): 552–4. doi:10.1016/j.jpeds.2004.06.047. PMID 15480384.
  5. Elpeleg O, Shaag A, Ben-Shalom E, Schmid T, Bachmann C (2002). "N-acetylglutamate synthase deficiency and the treatment of hyperammonemic encephalopathy". Ann Neurol. 52 (6): 845–9. doi:10.1002/ana.10406. PMID 12447942. S2CID 24604774.
  6. "Drug Approval Package: Carbaglu (Carglumic Acid) Tablets". U.S. Food and Drug Administration (FDA). 16 February 2010. Retrieved 9 June 2021.
  7. "Carglumic acid Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 17 June 2014. Retrieved 9 June 2021.
  8. "Carglumic acid Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 20 January 1998. Retrieved 9 June 2021.


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