Carglumic acid
- Generic Name
- Carglumic acid
- Brand Names
- Carbaglu, Ucedane
- Drug Type
- Small Molecule
- Chemical Formula
- C6H10N2O5
- CAS Number
- 1188-38-1
- Unique Ingredient Identifier
- 5L0HB4V1EW
- Background
Carglumic acid is a drug used for the treatment of hyperammonemia in patients with a deficiency in N-acetyl glutamate synthase. 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. Carglumic acid was approved by the U.S. Food and Drug Administration (FDA) on 18 March 2010.
- Indication
For the treatment of acute and chronic hyperammonaemia in patients with N-acetylglutamate synthase (NAGS) deficiency. This enzyme is an important component of the urea cycle to prevent build up of neurotoxic ammonium in the blood.
- Associated Conditions
- Acute Hyperammonaemia, Chronic Hyperammonaemia
- Associated Therapies
- Maintenance therapy
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