Carglumic acid

Carglumic acid

Clinical data
Trade names	Carbaglu, Ucedane
Other names	(S)-2-ureidopentanedioic acid
AHFS/Drugs.com	Monograph
License data	US  DailyMed:  Carglumic acid
Pregnancy category	AU:B1
Routes of administration	By mouth
ATC code	A16AA05 ( WHO )
Legal status
Legal status	AU: S4 (Prescription only) [1] CA: ℞-only [2] US: ℞-only [3] EU:Rx-only [4] [5]
Pharmacokinetic data
Bioavailability	30%
Protein binding	Undetermined
Metabolism	Partial
Elimination half-life	4.3 to 9.5 hours
Excretion	Fecal (60%) and kidney (9%, unchanged)
Identifiers
IUPAC name (2S)-2-(carbamoylamino)pentanedioic acid
CAS Number	1188-38-1  N
PubChem CID	121396
IUPHAR/BPS	7458
DrugBank	DB06775  Y
ChemSpider	1265942  N 108351  N
UNII	5L0HB4V1EW
KEGG	D07130  Y
ChEBI	CHEBI:71028  N
ChEMBL	ChEMBL1201780  N
CompTox Dashboard (EPA)	DTXSID7046706
ECHA InfoCard	100.116.323
Chemical and physical data
Formula	C6H10N2O5
Molar mass	190.155 g·mol−1
3D model (JSmol)	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 Y Key:LCQLHJZYVOQKHU-VKHMYHEASA-N Y
NY  (what is this?)    (verify)

Carglumic acid, sold under the brand name Carbaglu among others, is used for the treatment of hyperammonaemia. ^{[3] [6] [7]} Carglumic acid is a carbamoyl phosphate synthetase 1 (CPS 1) activator. ^[3]

The most common adverse effects include vomiting, abdominal pain, pyrexia (fever), and tonsillitis, anemia, diarrhea, ear infection, other infections, nasopharyngitis, decreased hemoglobin levels, and headache. ^[3]

It was approved for medical use in the United States in March 2010. ^[8] Carglumic acid is an orphan drug. ^{[9] [10]} It is available as a generic medication. ^[11]

Medical uses

Carglumic acid is indicated for the treatment of acute hyperammonemia and chronic hyperammonemia. ^{[3] [4] [5]}

References

1. "Prescription medicines: registration of new chemical entities in Australia, 2015". Therapeutic Goods Administration (TGA). 21 June 2022. Retrieved 10 April 2023.
2. "Health Canada New Drug Authorizations: 2015 Highlights". Health Canada . 4 May 2016. Retrieved 7 April 2024.
3. "Carbaglu- carglumic acid tablet". DailyMed. Retrieved 9 June 2021.
4. "Carbaglu EPAR". European Medicines Agency (EMA). 17 September 2018. Retrieved 9 June 2021.
5. "Ucedane EPAR". European Medicines Agency (EMA). 17 September 2018. Retrieved 9 June 2021.
6. 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.
7. 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. S2CID   24604774.
8. "Drug Approval Package: Carbaglu (Carglumic Acid) Tablets". U.S. Food and Drug Administration (FDA). 16 February 2010. Archived from the original on October 30, 2014. Retrieved 9 June 2021.
9. "Carglumic acid Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 17 June 2014. Retrieved 9 June 2021.
10. "Carglumic acid Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 20 January 1998. Archived from the original on June 9, 2021. Retrieved 9 June 2021.
11. "Competitive Generic Therapy Approvals". U.S. Food and Drug Administration (FDA). 29 June 2023. Archived from the original on 29 June 2023. Retrieved 29 June 2023.