Carglumic acid

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Carglumic acid
Carglumic acid.svg
Clinical data
Synonyms (S)-2-ureidopentanedioic acid
AHFS/Drugs.com Consumer Drug Information
License data
Pregnancy
category
  • unknown
Routes of
administration
Oral
ATC code
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
CAS Number
PubChem CID
IUPHAR/BPS
DrugBank
ChemSpider
UNII
KEGG
ChEBI
ChEMBL
ECHA InfoCard 100.116.323
Chemical and physical data
Formula C6H10N2O5
Molar mass 190.2 g/mol
3D model (JSmol)
 NYesY (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 expired on March 18, 2017.[3]

Adverse effects[edit]

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

References[edit]

  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.