Carglumic acid
Clinical data | |
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AHFS/Drugs.com | Consumer Drug Information |
License data | |
Pregnancy category |
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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 | |
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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 |
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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
- ↑ 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.
- ↑ 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.
- ↑ "Patent and Exclusivity Search Results".
- ↑ Drugs.com: Professional Drug Facts for Carglumic Acid.
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