Active Ingredient: Sodium phenylbutyrate
Sodium phenylbutyrate is indicated as adjunctive therapy in the chronic management of urea cycle disorders, involving deficiencies of carbamylphosphate synthetase, ornithine transcarbamylase or argininosuccinate synthetase.
It is indicated in all patients with neonatal-onset disease (complete enzyme deficiencies, presenting within the first 28 days of life). It is also indicated in patients with late-onset disease (partial enzyme deficiencies, presenting after the first month of life) who have a history of hyperammonaemic encephalopathy.
For this indication, competent medicine agencies globally authorize below treatments:
Oral
9.9 - 13 g per m² of body surface area (BSA)
From 3.3 To 4.333 g per m² of body surface area (BSA) 3 time(s) per day every day
The daily dose should be individually adjusted according to the patient’s protein tolerance and the daily dietary protein intake needed to promote growth and development.
The usual total daily dose of sodium phenylbutyrate in clinical experience is:
The safety and efficacy of doses in excess of 20 g/day have not been established.
Plasma levels of ammonia, arginine, essential amino acids (especially branched chain amino acids), carnitine and serum proteins should be maintained within normal limits. Plasma glutamine should be maintained at levels less than 1,000 μmol/L.
Sodium phenylbutyrate must be combined with dietary protein restriction and, in some cases, essential amino acid and carnitine supplementation.
Citrulline or arginine supplementation is required for patients diagnosed with neonatal-onset form of carbamyl phosphate synthetase or ornithine transcarbamylase deficiency at a dose of 0.17 g/kg/day or 3.8 g/m²/day.
Arginine supplementation is required for patients diagnosed with deficiency of argininosuccinate synthetase at a dose of 0.4-0.7 g/kg/day or 8.8-15.4 g/m²/day.
If caloric supplementation is indicated, a protein-free product is recommended.
The total daily dose should be divided into equal amounts and given with each meal or feeding (e.g. 4-6 times per day in small children).
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