Following a limited submission
AWMSG advice |
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| Status: Recommended | |||
Sodium phenylbutyrate (Pheburane®) is recommended as an option for use within NHS Wales as adjunctive therapy in the chronic management of urea-cycle disorders, involving deficiencies of carbamylphosphate synthetase, ornithine transcarbamylase or argininosuccinate synthetase; in all patients with neonatal-onset presentation (complete enzyme deficiencies, presenting within the first 28 days of life) and in patients with late-onset disease (partial enzyme deficiencies, presenting after the first month of life) who have a history of hyperammonaemic encephalopathy. |
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Medicine details |
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| Medicine name | sodium phenylbutyrate (Pheburane®) | ||
| Formulation | 483 mg/g granules | ||
| Reference number | 2227 | ||
| Indication | 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 |
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| Company | Lucane Pharma | ||
| BNF chapter | Nutrition & blood | ||
| Submission type | Limited | ||
| Status | Recommended | ||
| Advice number | 3413 | ||
| NMG meeting date | 23/10/2013 | ||
| AWMSG meeting date | 20/11/2013 | ||
| Ratification by Welsh Government | 06/12/2013 | ||
| Date of issue | 13/12/2013 | ||
| Date of last review | 01/12/2016 | ||