Carglumic acid

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.

Carglumic acid
Clinical data
Other names(S)-2-ureidopentanedioic acid
AHFS/Drugs.comMonograph
License data
Pregnancy
category
  • unknown
Routes of
administration		By mouth
ATC code
Pharmacokinetic data
Bioavailability30%
Protein bindingUndetermined
MetabolismPartial
Elimination half-life4.3 to 9.5 hours
ExcretionFecal (60%) and kidney (9%, unchanged)
Identifiers
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 mass190.2 g/mol g·mol−1
3D model (JSmol)
 NY (what is this?)  (verify)

The US FDA approved it for treatment of hyperammonaemia on March 18, 2010. Orphan Drug exclusivity expired 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.
This article is issued from Wikipedia. The text is licensed under Creative Commons - Attribution - Sharealike. Additional terms may apply for the media files.