Carglumic acid

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Carglumic acid
Carglumic acid.svg
Names
Trade namesCarbaglu, Ucedane
Other names(S)-2-ureidopentanedioic acid
  • (2S)-2-(carbamoylamino)pentanedioic acid
Clinical data
Drug classCarbamoyl phosphate synthetase 1 (CPS 1) activator[1]
Main usesHigh ammonia due to certain metabolic disorders[1]
Side effectsVomiting, abdominal pain, fever, tonsillitis, low red blood cells, diarrhea, infections, low red blood cells, low blood sugar, pancreatitis, electrolyte abnormalities, headache[1]
Pregnancy
category
  • AU: B1
Routes of
use
By mouth
Typical dose10 to 250 mg/kg per day[1]
External links
AHFS/Drugs.comMonograph
US NLMCarglumic acid
Legal
License data
Legal status
Pharmacokinetics
Bioavailability30%
Protein bindingUndetermined
MetabolismPartial
Elimination half-life4.3 to 9.5 hours
ExcretionFecal (60%) and kidney (9%, unchanged)
Chemical and physical data
FormulaC6H10N2O5
Molar mass190.155 g·mol−1
3D model (JSmol)
  • C(CC(=O)O)[C@@H](C(=O)O)NC(=O)N
  • 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 checkY
  • Key:LCQLHJZYVOQKHU-VKHMYHEASA-N checkY

Carglumic acid, sold under the brand name Carbaglu among others, is a medication used to treat high ammonia due to N-acetylglutamate synthase (NAGS) deficiency, propionic acidemia (PA) or methylmalonic acidemia (MMA).[1] It is taken by mouth.[1]

Common side effects include vomiting, abdominal pain, fever, tonsillitis, low red blood cells, diarrhea, infections, low red blood cells, low blood sugar, pancreatitis, electrolyte abnormalities, and headache.[1] Safety in pregnancy is unclear.[1] It is a carbamoyl phosphate synthetase 1 (CPS 1) activator, which works by activating an enzyme that breaks down ammonia.[1][2]

Carglumic acid was approved for medical use in Europe in 2003 and the United States in 2010.[1][2] It is available as a generic medication.[4] In the United Kingdom 60 tablets of 200 mg costs the NHS about £2,600 as of 2021.[4] This amount in the United States costs about 13,400 USD.[5] A generic version was approved in 2021 in the USA.[6]

Medical uses

Carglumic acid is indicated for the treatment of acute hyperammonemia and chronic hyperammonemia.[1][2][3]

Dosage

It is used at a dose of 10 to 250 mg/kg per day.[1] This is take as 2 to 4 doses.[1]

History

Carglumic acid is an orphan drug.[7][8]

References

  1. 1.00 1.01 1.02 1.03 1.04 1.05 1.06 1.07 1.08 1.09 1.10 1.11 1.12 1.13 "Carbaglu- carglumic acid tablet". DailyMed. Archived from the original on 9 June 2021. Retrieved 9 June 2021.
  2. 2.0 2.1 2.2 2.3 "Carbaglu EPAR". European Medicines Agency (EMA). Archived from the original on 9 June 2021. Retrieved 9 June 2021.
  3. 3.0 3.1 "Ucedane EPAR". European Medicines Agency (EMA). Archived from the original on 9 June 2021. Retrieved 9 June 2021.
  4. 4.0 4.1 BNF 81: March-September 2021. BMJ Group and the Pharmaceutical Press. 2021. p. 1118. ISBN 978-0857114105.
  5. "Carbaglu Prices, Coupons & Patient Assistance Programs". Drugs.com. Archived from the original on 20 April 2021. Retrieved 30 December 2021.
  6. Research, Center for Drug Evaluation and (10 February 2022). "2021 First Generic Drug Approvals". FDA. Archived from the original on 21 June 2022. Retrieved 22 October 2022.
  7. "Carglumic acid Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 17 June 2014. Archived from the original on 9 June 2021. Retrieved 9 June 2021.
  8. "Carglumic acid Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 20 January 1998. Archived from the original on 9 June 2021. Retrieved 9 June 2021.

External links

Identifiers:
  • "Carglumic acid". Drug Information Portal. U.S. National Library of Medicine. Archived from the original on 2021-06-09. Retrieved 2021-09-14.