Carglumic acid

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Information about Carglumic acid

Carglumic acid is an orphan drug and a derivative of N-acetylglutamate that activates the first enzyme in the urea cycle that is responsible for removal and detoxification of ammonia, making this drug a valuable agent for therapy of hyperammonemia caused by rare forms of urea cycle defects. Clinical experience with carglumic acid is limited, but it has not been linked to significant serum enzyme elevations during therapy or to instances of clinically apparent acute liver injury.

Mechanism of action of Carglumic acid

Carglumic (kar gloo' mik) acid is a small molecule that resembles N-acetylglutamate, a normal occurring metabolite that activates the enzyme carbamoyl phosphate synthetase I (CPS-1), the first step in the urea cycle which is responsible for removal and detoxification of ammonia. Carglumic acid is used to treat the severe hyperammonemia that occurs in the urea cycle disorder caused by deficiency of hepatic N-acetylglutamate synthase (NAGS) which normally produces N-acetylglutamate. Carglumic acid was shown to decrease ammonia levels during acute exacerbation of NAGS deficiency as well as decreasing levels chronically between acute episodes.

FDA approval information for Carglumic acid

Carglumic acid was approved as oral therapy of NAGS deficiency in the United States in 2010. It has been used experimentally and off-label to treat other genetic as well as acquired causes of severe hyperammonemia including idiopathic cases due to valproate and cancer chemotherapy and secondary genetic cases due to organic acidurias such as isovaleric, methylmalonic and propionic aciduria. Carglumic acid is available in tablets of 200 mg under the brand name Carbaglu.

Dosage and administration for Carglumic acid

The initial dose for acute hyperammonemia is 100 to 250 mg/kg daily given in 2 to 4 divided doses. The typical dose for maintenance therapy is 100 mg/kg daily. The tablets are not swallowed whole, but rather dispersed in small amounts of water just before oral administration.

Side effects of Carglumic acid

Side effects are generally dose related and can include nausea, vomiting, abdominal pain, diarrhea, and fever.

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cystic fibrosis agents

enzyme replacement therapy

glucosylceramide synthase inhibitors (substrate restriction therapy)

lysosomal acid lipase deficiency agents


homocystinuria agents

Huntington disease agents

Monoclonal Antibodies

Tyrosinemia Agents

Urea Cycle Disorder Agents

Hematologic Agents

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