Buphenyl

Adults

Dosage Forms and Advantages

Tablet 500 mg

Oral Suspension 250 g

Urea Cycle Disorders

Indicated as an adjunct to dietary protein restriction and essential amino acid supplementation for the long-term management of patients with urea cycle disorders, including arginase deficiency (Argininemia), carbamoyl phosphate synthetase deficiency (CPS) 1) Yes and Ornithine Carbamyltransferase Deficiency (OTC) Deficiency

All patients with neonatal deficiency (complete enzyme deficiency, appearing within the first 28 days of life) and late-onset disease (partial enzyme deficiency, appearing in the first month of life) demonstrate a history of hyperammonemia

≥20kg (tablet or suspension): 9.9-13 g/m Oral divided by Q4-8 hours Oral; not to exceed 20 g/day

<20 kg (suspension): 450-600 mg/kg/day divided by Q4-6 hours Oral (solution only)

Dosage Precautions

Take with food in equal amounts with meals (i.e. 3-6 times/day)

Mix the powder with solid or liquid food, but not with acidic beverages (such as orange juice).

Sickling DisordersSickle cell anemia (orphan disease)

Orphan drug designation for the treatment of disorders including sickle hemoglobinopathy anemia SS, SC and S-thalassemia

Malignant glioma (orphan disease)

Orphan drug designation Indicated for the treatment of surgery, radiation and chemotherapy for primary or recurrent malignant glioma

Spinal Muscular Atrophy (Orphan Disease)

Orphan Indicated for the treatment of Spinal Muscular Atrophy

Dosage Precautions< /p>

Must be combined with dietary protein restriction and, in some cases, essential amino acids must be supplemented

Monitor plasma amino acids, ammonia levels

Administration

Take equal amounts with meals Food (i.e. 3-6 times/day)

Mix the powder with solid or liquid food but not acidic drinks (e.g. orange juice)

Pediatric

Dosage Forms and Advantages

Tablets 500 mg

Oral Suspension 250 mg

Urea Cycle Disorders

Indicated as an adjunct to dietary protein restriction and essential amino acid supplementation for the long-term management of patients with urea cycle disorders, including argininase deficiency (Argininemia), carbamoyl phosphate synthetase deficiency (CPS) 1) Yes and Ornithine Carbamyltransferase Deficiency (OTC) Deficiency

All patients with neonatal deficiency (complete enzyme deficiency, appearing within the first 28 days of life) and late-onset disease (partial enzyme deficiency, appearing in the first month of life) demonstrate a history of hyperammonemia

≥20kg (tablet or suspension): 9.9-13 g /m²PO divided q4-8hr PO; not to exceed 20 g/day

<20 kg (suspension): 450-600 mg/kg/day divided q4 -6 hours PO (solution only)

Administration

Administer food in equal divided doses as meals or feedings (i.e. 3-6 times/day)

Mix powder with solid or liquid food but not acidic drinks (e.g. orange juice)

Adverse reactions

> 10%

Amenorrhea/dysmenorrhea (23%)

Hypoalbuminemia (11%)

Metabolic acidosis (14%)

1-10%

Anemia (9%)< /p>

Anorexia (4%)

Hypophosphatemia (2%)

Hypernatremia (1%)

Hyperuricemia (2%)

Hypokalemia (1%)

Leukopenia (4%)

Strong body odor (3%)

Thrombocytopenia (3%)

Warnings/Contraindications

1. Allergies

2. Acute hyperammonemia

Precautions

1. Renal insufficiency, liver insufficiency, CHF

2. Patients with restricted sodium intake (including 125 mg Na/g)

3. Powder should not be mixed with acidic liquids

4. May cause sodium and liquid retention; Use caution in patients with heart failure and poor fluid retention

5. Hyperammonemia and hyperammonemic encephalopathy may occur during treatment (treat hyperammonemia as a medical emergency)

6. Will not reverse neurological damage from existing hyperammonemia

7. Recommend low-protein diet to replace possible amino acid intake