Inborn errors of metabolism:
Indications for: RAVICTI
Chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. Limitations of use: not for treatment of acute hyperammonemia in patients with UCDs. Safety and efficacy for the treatment of N-acetylglutamate synthase (NAGS) deficiency has not been established.
Adults and Children:
Take with food or formula. Administer dose via oral syringe or dosing cup; may give by NG or gastrostomy tube: see full labeling. <2yrs: give in 3 or more equally divided doses, rounded to nearest 0.1mL. ≥2yrs: give in 3 equally divided doses, rounded to nearest 0.5mL. Max 17.5mL (19g) daily. Must be used with dietary protein restriction and, in some cases, dietary supplements (eg, essential amino acids, arginine, citrulline, protein-free calorie supplements). Phenylbutyrate-naïve: 4.5–11.2mL/m2/day (5–12.4g/m2/day); patients with some residual enzyme activity not adequately controlled with protein restrictions: 4.5mL/m2/day. To determine starting dose in treatment-naïve patients: consider the patient’s residual urea synthetic capacity, dietary protein requirements, and diet adherence. Switching from sodium phenylbutyrate: give dose that contains the same amount of phenylbutyric acid; convert as follows: Total daily dose of Ravicti (mL) = total daily dose of sodium phenylbutyrate tablets (g) X 0.86 or total daily dose of sodium phenylbutyrate powder (g) x 0.81. Moderate to severe hepatic impairment: start at the lower end of dosing range. Dose adjustment and monitoring: see full labeling.
Monitor for signs/symptoms of neurotoxicity; reduce dose if occurs. Pancreatic insufficiency or intestinal malabsorption: closely monitor ammonia levels. Hepatic or renal impairment: monitor closely. Pregnancy. Nursing mothers: not recommended.
Concomitant corticosteroids, valproic acid, or haloperidol may increase plasma ammonia levels; monitor closely. May be potentiated by probenecid. Antagonizes midazolam; monitor. May antagonize CYP3A4 substrates with narrow therapeutic index (eg, alfentanil, quinidine, cyclosporine).
Diarrhea, flatulence, headache, nausea, vomiting, decreased appetite, dizziness, fatigue, abdominal pain; hyperammonemia. In children: also rash, neutropenia, constipation, pyrexia, hypophagia, cough, nasal congestion, rhinorrhea, papule.
Enroll women exposed to Ravicti during pregnancy by calling (855) 823-2595.
Generic Drug Availability:
Liq (25mL)—1, 4