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glycerol phenylbutyrate (Ravicti®)

 

Following a full submission

AWMSG advice

Status: Recommended

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 Recommendation: Glycerol phenylbutyrate (Ravicti) 2127 (PDF, 265Kb)
 Appraisal Report: Glycerol phenylbutyrate (Ravicti) 2127 (PDF, 305Kb)

Medicine details

Medicine name glycerol phenylbutyrate (Ravicti®)
Formulation 1.1 g/ml oral formulation
Reference number 2127
Indication

Adjunctive therapy for chronic management of patients with urea cycle disorders including deficiencies of

  • carbamoyl phosphate-synthase-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 (e.g. essential amino acids, arginine, citrulline, protein-free calorie supplements)

Company Immedica Pharma
BNF chapter Nutrition & blood
Submission type Full
Status Recommended
Advice number 1819
NMG meeting date 06/11/2019
AWMSG meeting date 11/12/2019
Date of issue 12/12/2019
Commercial arrangement WPAS