carglumic acid at a glance
Medicine class
An amino-acid derivative that acts like the natural urea-cycle activator N-acetylglutamate.
Usual uses
Hyperammonaemia caused by primary N-acetylglutamate synthase deficiency or by isovaleric, methylmalonic or propionic acidaemia.
How it is taken
Disperse the tablet in water and take it immediately before food or a feed. It may also be given through a nasogastric tube.
Specialist treatment
Treatment is initiated and supervised by a clinician experienced in metabolic disorders.
What is carglumic acid used for?
Licensed uses are hyperammonaemia associated with specific inherited metabolic disorders.
Primary N-acetylglutamate synthase deficiency.
Isovaleric acidaemia.
Methylmalonic acidaemia.
Propionic acidaemia.
How does carglumic acid work?
Carglumic acid acts like N-acetylglutamate, activating carbamoyl phosphate synthetase 1—the first enzyme in the liver’s urea cycle. This helps convert toxic ammonia into urea for removal.
carglumic acid preparations and strengths
Dispersible tablet
Route: Oral
Strengths: 200 mg
How to take carglumic acid
Use carglumic acid exactly as directed by the specialist metabolic team.
Disperse the prescribed tablet portion in 5–10 mL of water.
Take the dispersion immediately; it has a slightly acidic taste.
Take it before meals or feeds.
It may be given through a nasogastric tube using a syringe when clinically required.
Tell the specialist team about kidney impairment because closer oversight and treatment adjustment may be needed.
carglumic acid side effects
Common or expected effects
- Increased sweating has been reported commonly in people treated for N-acetylglutamate synthase deficiency.
Get urgent medical advice
- A slow heart rate has been reported uncommonly in organic acidaemia. Seek prompt assessment if accompanied by faintness, breathlessness or marked weakness.
- Get emergency help for a rash with facial or throat swelling or difficulty breathing.
- Seizures, severe confusion or reduced consciousness may indicate worsening hyperammonaemia and require emergency assessment.
carglumic acid in pregnancy
Human pregnancy information is limited, and the product information advises caution. Discuss pregnancy planning or suspected pregnancy promptly with the specialist metabolic and maternity teams. Do not stop treatment independently.
carglumic acid while breastfeeding
Breastfeeding during treatment is contraindicated for the selected products. Human milk transfer is unknown; discuss alternatives with the metabolic and maternity teams.
carglumic acid interactions
No specific interaction studies have been performed. The metabolic team and pharmacist should review all prescribed, over-the-counter and complementary products.
Common questions about carglumic acid
Answers are fully visible for fast scanning and source review.
What is carglumic acid used for?
It treats raised blood ammonia caused by primary N-acetylglutamate synthase deficiency or associated with isovaleric, methylmalonic or propionic acidaemia.
Can the tablets be swallowed whole?
The tablets should be dispersed in water and taken immediately rather than swallowed whole.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.