CARBAGLU

The pharmacokinetics of carglumic acid in healthy subjects following an intravenous (IV) infusion over 2 hours at 8 mg/kg or an oral administration at 100 mg/kg are summarized in Table 3 .

Table 3Mean (SD) Pharmacokinetic Parameter Values of Carglumic Acid in Healthy Subjects # Median (range); N/A, not applicable PK parameter IV infusion 8 mg/kg (N=10) Oral 100 mg/kg (N=12) C max (ng/mL) 8613 (558) 3284 (321) T max (hr) # 2 (1-2) 3 (2-4) AUC (ng*hr/mL) 24501 (1613) 31426 (2150) T ½ (hr) 31 (3)...

14.1 Acute and Chronic Hyperammonemia due to NAGS Deficiency The efficacy of CARBAGLU in the treatment of acute and chronic hyperammonemia due to NAGS deficiency was evaluated in a retrospective case series of 23 NAGS deficiency patients treated with CARBAGLU over a median duration of 7.9 years (range 0.6 to 20.8 years). For acute treatment, patients received CARBAGLU at 100 mg/kg/day to 250 mg/kg/day orally administered in 2 to 4 divided doses. For maintenance treatment, the dosage was reduced over time based on plasma ammonia level and clinical response. The baseline characteristics of the p...

NAGS deficiency : Most common adverse reactions (≥13%) are vomiting, abdominal pain, pyrexia, tonsillitis, anemia, diarrhea, ear infection, infections, nasopharyngitis, hemoglobin decreased, and headache. ( 6.1 ) PA and MMA : Most common adverse reactions (≥5%) are neutropenia, anemia, vomiting, electrolyte imbalance, decreased appetite, hypoglycemia, lethargy/stupor, encephalopathy and pancreatitis/lipase increased. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Recordati Rare Diseases ...

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