Treating hyperammonemia due to NAGS deficiency with CARBAGLU
CARBAGLU® (carglumic acid) is a synthetic structural analogue of
N-acetylglutamate (NAG), which is an essential allosteric activator of carbamoyl phosphate synthetase 1 (CPS1) in liver mitochondria.
- CPS1 is the first enzyme of the urea cycle, which converts ammonia into urea.
- NAG is the product (cofactor) of N-acetylglutamate synthase (NAGS), a mitochondrial enzyme.
- Carglumic acid acts as a replacement for NAG in NAGS deficiency patients by activating CPS1.1
- CARBAGLU thus acts as a key to start the urea cycle, enabling it to function as it should to maintain normal blood ammonia levels.2