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glycerol phenylbutyrate (Ravicti®)


Reference No. 2127

Publication date:
13/12/2019


Appraisal information

glycerol phenylbutyrate (Ravicti®) 1.1 g/ml oral formulation


Company: Immedica Pharma
BNF category: Nutrition and blood
NMG meeting date: 06/11/2019
AWMSG meeting date: 11/12/2019
   
   
Submission Type: Full Submission
Status: Recommended
Advice No: 1819
Ratification by Welsh Government: 12/12/2019

Current Progress


Submission
received
NMG
meeting
AWMSG
meeting
Ratification by
Welsh Government

AWMSG advice

Glycerol phenylbutyrate (Ravicti®) is recommended as an option for use within NHS Wales for use as an adjunctive therapy for chronic management of patients with urea cycle disorders including deficiencies of: carbamoyl phosphate synthetase I (CPS); ornithine carbamoyltransferase (OTC); argininosuccinate synthetase (ASS); argininosuccinate lyase (ASL); arginase I (ARG) and ornithine translocase deficiency hyperornithinaemia-hyperammonaemia homocitrullinuria syndrome (HHH) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. Ravicti® must be used with dietary protein restriction and, in some cases, dietary supplements (for example, essential amino acids, arginine, citrulline, protein-free calorie supplements). This recommendation applies only in circumstances where the approved Wales Patient Access Scheme (WPAS) is utilised or where the list/contract price is equivalent or lower than the PAS/WPAS price.
Final Appraisal Recommendation (FAR)
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AWMSG Secretariat Appraisal Report (ASAR)
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