Carglumic acid
- US DailyMed: Carglumic acid
category
- AU: B1
administration
- A16AA05 (WHO)
- AU: S4 (Prescription only)[1]
- CA: ℞-only[2]
- US: ℞-only[3]
- EU: Rx-only[4][5]
- (2S)-2-(carbamoylamino)pentanedioic acid
- 1188-38-1
N
- 121396
- 7458
- DB06775
Y
- 1265942
N
- 108351
N
- 5L0HB4V1EW
- D07130
Y
- CHEBI:71028
N
- ChEMBL1201780
N
- DTXSID7046706
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Y
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Y
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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
- ^ "Prescription medicines: registration of new chemical entities in Australia, 2015". Therapeutic Goods Administration (TGA). 21 June 2022. Retrieved 10 April 2023.
- ^ "Health Canada New Drug Authorizations: 2015 Highlights". Health Canada. 4 May 2016. Retrieved 7 April 2024.
- ^ a b c d e "Carbaglu- carglumic acid tablet". DailyMed. Retrieved 9 June 2021.
- ^ a b "Carbaglu EPAR". European Medicines Agency (EMA). 17 September 2018. Retrieved 9 June 2021.
- ^ a b "Ucedane EPAR". European Medicines Agency (EMA). 17 September 2018. Retrieved 9 June 2021.
- ^ 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. S2CID 24604774.
- ^ "Drug Approval Package: Carbaglu (Carglumic Acid) Tablets". U.S. Food and Drug Administration (FDA). 16 February 2010. Retrieved 9 June 2021.
- ^ "Carglumic acid Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 17 June 2014. Retrieved 9 June 2021.
- ^ "Carglumic acid Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 20 January 1998. Retrieved 9 June 2021.
- ^ "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.
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- Carglumic acid
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- Carbohydrate metabolism: sucrase (Sacrosidase)
- alpha-glucosidase (Alglucosidase alfa, Avalglucosidase alfa, Cipaglucosidase alfa)
- Glycolipid/sphingolipid: glucocerebrosidase (Alglucerase
- Imiglucerase
- Taliglucerase alfa
- Velaglucerase alfa)
- alpha-galactosidase (Agalsidase alfa
- Agalsidase beta
- Pegunigalsidase alfa)
- Glycosaminoglycan: iduronidase (Laronidase)
- arylsulfatase B (Galsulfase)
- iduronate-2-sulfatase (Idursulfase)
- idursulfase beta
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