following an abbreviated submission
sodium phenylbutyrate granules (Pheburane®) are accepted for use within NHS Scotland.
Indication under review: 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 presentation (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.
Sodium phenylbutyrate granules (Pheburane®) provide an alternative to sodium phenylbutyrate tablets at no additional cost but are more expensive than an existing brand of sodium phenylbutyrate granules.
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Medicine details
- Medicine name:
- sodium phenylbutyrate (Pheburane)
- SMC ID:
- 914/13
- Indication:
- Adjunctive therapy in the chronic management of urea cycle disorders, involving deficiencies of carbamylphosphate synthetase, ornithine transcarbamylase or argininosuccinate synthetase.
- Pharmaceutical company
- Lucane Pharma
- BNF chapter
- Nutrition and blood
- Submission type
- Abbreviated
- Status
- Accepted
- Date advice published
- 11 November 2013