Health Canada approves sale of Carbaglu for deficient liver enzymes

Orphan Europe announced that Health Canada has authorized the sale of Carbaglu as adjunctive therapy for the treatment of acute hyperammonemia or as maintenance therapy for chronic hyperammonemia due to the deficiency of N-aceytlglutamate synthase, a liver enzyme, in pediatric and adult patients, according to a press release from the manufacturer.

N-aceytlglutamate synthase (NAGS) deficiency is a rare urea cycle disorder (UCD), according to the release. UCDs are inherited hepatic metabolic disorders caused by a deficiency in liver enzymes involved in the transformation of ammonia into urea. Hyperammonemia is the major cause of mortality and morbidity in many UCDs, including NAGS deficiency.

The release states that Carbaglu (carglumic acid, Orphan Europe) acts as a replacement for the co-factor NAG, which is important for the activation of carbamoyl phosphate synthetase, the first urea cycle enzyme. Carbaglu stimulates CPS1, triggering the urea cycle and normalizing blood ammonia concentration.

A Notice of Compliance was issued for carglumic acid after a priority review by Health Canada was conducted. It will be distributed across Canada by Recordati Rare Diseases Inc. c/o GMD Distribution Inc., Ontario, according to the release.

Carglumic acid is currently marketed in the U.S. by Recordati Rare Diseases for pediatric and adult patients as an adjunctive therapy for acute hyperammonemia and as maintenance therapy for chronic hyperammonemia due to NAGS deficiency.

Carglumic acid is also currently marketed in Europe for the treatment of hyperammonemia due to NAGS deficiency and in the treatment of hyperammonemia due to propionic academia, methylmalonic academia and isovaleric academia in pediatric and adult patients, according to the release.

Editor's note: This article was updated with correct reference to the co-factor NAG.