Search
carglumic acid (Carbaglu)
Indications:
- treatment of hyperammonemia due to N-acetylglutamate synthase deficiency
Dosage:
- initial dose of 100-250 mg/kg/day in 2-4 divided doses
- adjust to maintain normal ammonia plasma levels
- maintenance doses range from 10-100 mg/kg/day
- concomitant use of other ammonia-lowering therapies is recommended during episodes of acute hyperammonemia
Monitor:
- liver function tests
- renal function tests
- complete blood count (CBC)
Adverse effects:
- vomiting
- abdominal pain
- diarrhea
- fever
- tonsillitis
- anemia
- ear infection
- headache
Mechanism of action:
- structural analogue of N-acetylglutamate, the naturally occurring activator of carbamoyl phosphate synthetase, the 1st enzyme of the urea cycle
General
orphan drug
metabolic agent (metabolic modifier)
References
- Yael Waknine
FDA Approves Carglumic Acid Therapy for Rare Genetic Disorder
Medscape Today, March 19, 2010
http://www.medscape.com/viewarticle/718847
- Deprecated Reference