Carglumic acid

Carglumic acid
Names
Trade namesCarbaglu, Ucedane
Other names(S)-2-ureidopentanedioic acid
IUPAC name
  • (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)
    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 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 2 3 4 5 6 7 8 9 10 11 12 13 14 "Carbaglu- carglumic acid tablet". DailyMed. Archived from the original on 9 June 2021. Retrieved 9 June 2021.
    2. 1 2 3 4 "Carbaglu EPAR". European Medicines Agency (EMA). Archived from the original on 9 June 2021. Retrieved 9 June 2021.
    3. 1 2 "Ucedane EPAR". European Medicines Agency (EMA). Archived from the original on 9 June 2021. Retrieved 9 June 2021.
    4. 1 2 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.
    Identifiers:
    • "Carglumic acid". Drug Information Portal. U.S. National Library of Medicine. Archived from the original on 2021-06-09. Retrieved 2021-09-14.
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