Inborn errors of metabolism:
Indications for CARBAGLU:
Adjunctive therapy to standard of care for the treatment of acute hyperammonemia due to N-acetylglutamate synthase (NAGS) deficiency, propionic acidemia (PA) or methylmalonic acidemia (MMA). Maintenance therapy for the treatment of chronic hyperammonemia due to NAGS deficiency.
Adults and Children:
Do not swallow whole or crush tabs. Dissolve each tab with at least 2.5mL of water; stir and mix. Swallow mixture orally or via an oral syringe immediately before meals; may administer via NG tube or G-tube. NAGS deficiency (acute hyperammonemia): 100–250mg/kg/day; divided into 2–4 doses rounded to the nearest 100mg; administer with other ammonia lowering therapies (eg, alternate pathway meds, hemodialysis, protein restriction); (chronic hyperammonemia): 10–100mg/kg/day; divided into 2–4 doses rounded to the nearest 100mg; may need other ammonia lowering therapies and protein restriction based on plasma ammonia levels. PA or MMA (acute hyperammonemia): (≤15kg): 150mg/kg/day; (>15kg): 3.3g/m2/day; divided into 2 equal doses (given 12hrs apart) rounded up to the next multiple of 50mg; continue until ammonia level <50micromol/L and max duration of 7 days; administer with other ammonia lowering therapies (eg, IV glucose, insulin, L-carnitine, protein restriction, dialysis).
Monitor plasma ammonia levels closely. Pregnancy. Nursing mothers.
Carbamoyl phosphate synthetase 1 (CPS 1) activator.
Vomiting, anemia; NAGS: also abdominal pain, pyrexia, tonsillitis, diarrhea, ear infection, infections, nasopharyngitis, hemoglobin decreased, headache; PA and MMA: also neutropenia, electrolyte imbalance, decreased appetite, hypoglycemia, lethargy/stupor, encephalopathy, pancreatitis/lipase increased.
Generic Drug Availability: