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🏅 FDA Orphan Designation

Carbaglu

CARGLUMIC ACID

Manufacturer: Recordati Rare Diseases

Indicated for:
Hyperammonemia due to N-acetylglutamate synthase deficiency

FDA-Approved Indications (1)

Adjunctive therapy to standard of care for the treatment of acute hyperammonemia due to N-acetylglutamate synthase (NAGS) deficiency

Population: pediatric and adult patients

Indications & Usage

1. INDICATIONS AND USAGE CARBAGLU 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. ( 1.1 ) Maintenance therapy for the treatment of chronic hyperammonemia due to NAGS deficiency. ( 1.1 ) Adjunctive therapy to standard of care for the treatment of acute hyperammonemia due to propionic acidemia (PA) or methylmalonic acidemia (MMA). ( 1.2 ) 1.1 Acute and Chronic Hyperammonemia due to N‑acetylglutamate Synthase (NAGS) Deficiency CARBAGLU is indicated in adult and pediatric patients as: Adjunctive therapy to standard of care for the treatment of acute hyperammonemia due to NAGS deficiency. Maintenance therapy for the treatment of chronic hyperammonemia due to NAGS deficiency. 1.2 Acute Hyperammonemia due to Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA) CARBAGLU is indicated in adult and pediatric patients as adjunctive therapy to standard of care for the treatment of acute hyperammonemia due to PA or MMA.

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Carbaglu
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Medical disclaimer: This information is for educational purposes only. Always consult a qualified healthcare provider before making treatment decisions. Data sourced from FDA and current as of our last update.