CARBAGLU
CARGLUMIC ACID
What it does
Carglumic acid is a medicine used to help manage certain metabolic disorders.
Commonly used for: urea cycle disorder, hyperammonaemia
Read more in plain English ↓Plain-language summary for general understanding - not medical advice. Always follow your pharmacist/doctor.
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Sourcing - Kenya onlyRegistration & product details
Source: South African Health Products Regulatory Authority · fetched 2026-04-15 21:16:39 · updated 2026-10-21 04:00:54
About this medicine
Carglumic acid is a medicine used to help manage certain metabolic disorders.
What it treats
- urea cycle disorder
- hyperammonaemia
How it works
Carglumic acid helps to remove excess ammonia from the blood, which can be harmful.
Who it's for
This medicine is for people with specific genetic conditions that affect how their body processes proteins.
AI-assisted summary grounded in BNF data - general information only, not medical advice. Always confirm with your pharmacist or doctor.
Clinical monograph: carglumic
Carglumic acid is a medication primarily used as a treatment for hyperammonemia due to deficiencies in the urea cycle, specifically carbamoyl phosphate synthetase 1 (CPS1) deficiency. It acts as a substitute for N-acetylglutamate, which is a key activator of CPS1, thereby facilitating the conversion of ammonia to urea. Carglumic acid helps reduce ammonia levels in the blood, which can be toxic and lead to severe neurological complications.
Indications
- Hyperammonemia due to urea cycle disorders
- Carbamoyl phosphate synthetase 1 deficiency
Dosage
Children: Refer to the BNF for Children for appropriate dosing recommendations based on age and weight.
Adults: Refer to the prescribing information or clinical guidelines for exact dosing recommendations based on individual patient needs.
Mechanism of action
Carglumic acid functions as a positive allosteric modulator of carbamoyl phosphate synthetase 1 (CPS1). By mimicking N-acetylglutamate, it enhances the enzyme's activity, promoting the conversion of ammonia into urea in the liver. This mechanism is crucial for detoxifying ammonia, especially in patients with urea cycle disorders.
Pharmacodynamics
Carglumic acid increases the enzyme activity of CPS1, thereby improving the body's ability to eliminate ammonia. The reduction of ammonia levels leads to a decrease in the risk of hyperammonemic crises, which can cause severe neurological damage. The drug's pharmacodynamic effects are particularly significant in managing urea cycle disorders, where ammonia accumulation poses a substantial health risk.
Pharmacokinetics
Carglumic acid is absorbed rapidly after oral administration, with peak plasma concentrations typically reached within a few hours. It has a half-life that allows for effective dosing intervals to manage ammonia levels effectively. The drug is primarily metabolized in the liver, with renal excretion being a significant pathway for elimination. Its pharmacokinetic profile supports its use in acute and chronic management of hyperammonemia.
Adverse effects
- Hyperammonaemia
- Hypoglycaemia
- Nausea
- Vomiting
- Diarrhea
- Fatigue
- Irritability
Precautions
- Monitor for signs of hyperammonaemia
- Use with caution in patients with renal impairment
Pregnancy
Carglumic acid should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. Consult healthcare professionals for risk assessment.
Breast-feeding
It is not known if carglumic acid is excreted in human milk. Caution should be exercised when administering to nursing mothers.
Storage
Store at room temperature, away from light and moisture. Keep out of reach of children.
Formulations
- Carglumic acid oral tablets
- Carglumic acid oral solution
AI-synthesized from BNF references - general information only, not a substitute for professional medical advice or the current BNF. Verify doses with a pharmacist.