Carglumic acid

Carglumic acid

Clinical data
AHFS/Drugs.com	Consumer Drug Information
License data	EU EMA: Carbaglu
Pregnancy category	unknown
Routes of administration	Oral
ATC code	A16AA05 (WHO)
Pharmacokinetic data
Bioavailability	30%
Protein binding	Undetermined
Metabolism	Partial
Biological half-life	4.3 to 9.5 hours
Excretion	Fecal (60%) and renal (9%, unchanged)
Identifiers
IUPAC name (2S)-2-(carbamoylamino)pentanedioic acid
Synonyms	(S)-2-ureidopentanedioic acid
CAS Number	1188-38-1
PubChem (CID)	121396
IUPHAR/BPS	7458
DrugBank	DB06775
ChemSpider	1265942 108351
UNII	5L0HB4V1EW
KEGG	D07130
ChEBI	CHEBI:71028
ChEMBL	CHEMBL1201780
ECHA InfoCard	100.116.323
Chemical and physical data
Formula	C6H10N2O5
Molar mass	190.2 g/mol
3D model (Jmol)	Interactive image
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 Key:LCQLHJZYVOQKHU-VKHMYHEASA-N
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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]

Adverse effects

The most common adverse effects include vomiting, abdominal pain, fever, and tonsillitis.^[4]

References

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".
4. Drugs.com: Professional Drug Facts for Carglumic Acid.