Carglumic acid

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Carglumic acid
Carglumic acid.svg
Systematic (IUPAC) name
(2S)-2-(carbamoylamino)pentanedioic acid
Clinical data
AHFS/ Consumer Drug Information
Licence data EMA:Link
Pregnancy cat.
  • unknown
Legal status
Routes Oral
Pharmacokinetic data
Bioavailability 30%
Protein binding Undetermined
Metabolism Partial
Half-life 4.3 to 9.5 hours
Excretion Fecal (60%) and renal (9%, unchanged)
CAS number 1188-38-1 N
ATC code A16AA05
PubChem CID 121396
DrugBank DB06775
ChemSpider 108351 YesY
KEGG D07130 YesY
Synonyms (S)-2-ureidopentanedioic acid
Chemical data
Formula C6H10N2O5 
Mol. mass 190.2 g/mol
 N (what is this?)  (verify)

Carglumic acid is an orphan drug, marketed by Orphan Europe under the trade name Carbaglu. Carglumic acid is used for the treatment of hyperammonaemia in patients with N-acetylglutamate synthase deficiency.[1][2] The initial daily dose ranges from 100 to 250 mg/kg, adjusted thereafter to maintain normal plasma levels of ammonia.

The US FDA approved it for treatment of hyperammonaemia on March 18, 2010. Orphan Drug exclusivity expires on March 18,2017.[3]


  1. ^ 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. 
  2. ^ 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. 
  3. ^ "Patent and Exclusivity Search Results".