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
 ☒NcheckY (what is this?)  (verify)

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]

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.[6][7]

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. Retrieved 9 June 2021.
  2. 2.0 2.1 2.2 2.3 "Carbaglu EPAR". European Medicines Agency (EMA). Retrieved 9 June 2021.
  3. 3.0 3.1 "Ucedane EPAR". European Medicines Agency (EMA). 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. Retrieved 30 December 2021.
  6. "Carglumic acid Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 17 June 2014. Retrieved 9 June 2021.
  7. "Carglumic acid Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 20 January 1998. Retrieved 9 June 2021.

External links

Identifiers: