Carglumic acid analogue restores urea cycle by activating carbamoyl phosphate synthetase.

Italian pharmaceutical group Recordati confirmed FDA approval of Carbaglu® for the treatment/maintenance therapy of acute/chronic hyperammonemia due to deficiency of the hepatic enzyme N-acetylglutamate syntase (NAGS deficiency). Clearance covers therapy for both pediatric and adult patients.

The NDA for Carbaglu was submitted by rare disease therapeutics company Orphan Europe, which was acquired by Recordati in 2007. Carbaglu was sanctioned in Europe in 2003. Recordati points out that the drug is the only specific treatment for hyperammonemia due to NAGS deficiency.

Hyperammonemia due to NAGS deficiency is a urea cycle disorder (UCD) that results from a deficiency in one of the six enzymes involved in urea synthesis, Orphan Europe explains. The inherited metabolic disease leads to extremely high plasma levels of ammonia, which leads to permanent and irreversible damage to the central nervous system.

Carbaglu is a functional analogue of N-acetyl glutamate that restores the urea cycle by activating the enzyme carbamoyl phosphate synthetase. The treatment effectively controls blood levels of ammonia to physiologically acceptable levels. It may also need to be accompanied by dietary protein restriction with hypercaloric value and the provision of arginine to enhance availability of ornithine, the firm adds.

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