INDICATIONS AND USAGE
Carbaglu® (carglumic acid) tablets for oral suspension 200mg is a carbamoyl phosphate synthetase 1 (CPS 1) activator indicated in pediatric and adult patients as:
- Adjunctive therapy to standard of care for the treatment of acute hyperammonemia due to N-acetylglutamate synthase (NAGS) deficiency.
- Maintenance therapy for the treatment of chronic hyperammonemia due to N-acetylglutamate synthase (NAGS) deficiency.
- Adjunctive therapy to standard of care for the treatment of acute hyperammonemia due to propionic acidemia (PA) or methylmalonic acidemia (MMA).
IMPORTANT SAFETY INFORMATION
- Contraindications: None.
- NAGS deficiency: Most common adverse reactions (≥13%) are: vomiting, abdominal pain, pyrexia, tonsillitis, anemia, diarrhea, ear infection, infections, nasopharyngitis, hemoglobin decreased, and headache.
- PA and MMA: Most common adverse reactions (≥5%) are neutropenia, anemia, vomiting, electrolyte imbalance, decreased appetite, hypoglycemia, lethargy/stupor, encephalopathy and pancreatitis/lipase increased.
- To report SUSPECTED ADVERSE REACTIONS, contact Recordati Rare Diseases Inc. at 1-888-575-8344, or FDA at 1‑800-FDA-1088 or www.fda.gov/medwatch.
- If CARBAGLU is administered during pregnancy to women with NAGS deficiency, health care providers should report CARBAGLU exposure to the pregnancy pharmacovigilance program by calling 1-888-575-8344.