Carglumic acid is an analog of N-acetylglutamate (NAG) used for the treatment of acute and chronic hyperammonemia in patients with N-acetylglutamate synthase (NAGS) deficiency. This rare genetic disorder results in elevated blood levels of ammonia, which can eventually cross the blood–brain barrier and cause neurologic problems, cerebral edema, coma, and death.
- Mechanism
- Curator reviewed
Carglumic acid is a synthetic structural analogue of N-acetylglutamate (NAG), which is an essential allosteric activator of the liver enzyme carbamoyl phosphate synthetase 1 (CPS1). CPS1 is found in the mitochondria and is the first enzyme of the urea cycle, which converts ammonia into urea. Carglumic acid acts as a replacement for NAG in NAGS deficiency patients by activating CPS1 but it does not help to regulate the urea cycle.
- Primary indication
- For the treatment of acute and chronic hyperammonaemia in patients with N-acetylglutamate synthase (NAGS) deficiency.Curator reviewed · 4 structured indications
- Formula / weight
- C6H10N2O5 · 190.154 g/mol (avg)
- First approval
- Canada, 2019 · United States, 2010 · European Union, 2016
- Also known as
- Carbamino-L-glutamic acid · Carbamylglutamic acid · N-Carbamylglutamate · Ureidoglutaric acid
- Code names
- OE-312
- Brand names
Resolves to
LCQLHJZYVOQKHU-VKHMYHEASA-NSMILESNC(=O)N[C@@H](CCC(O)=O)C(O)=OWhat you can answer from here — as of June 15, 2026
Which drugs share a target with Carglumic acid, and which of those have an active Phase 3 trial?
Create a free account or log in to explore the full data card.
Create Account