glycerol phenylbutyrate (Ravicti)

following a full submission:

glycerol phenylbutyrate (Ravicti®) is accepted for use within NHSScotland.

Indication under review: For use as adjunctive therapy for chronic management of adult and paediatric patients ≥2 months of age with urea cycle disorders (UCDs) 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. Glycerol phenylbutyrate must be used with dietary protein restriction and, in some cases, dietary supplements (e.g. essential amino acids, arginine, citrulline, protein-free calorie supplements).

Glycerol phenylbutyrate is non-inferior to sodium phenylbutyrate for control of blood ammonia levels in patients with UCDs.

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of glycerol phenylbutyrate. This advice is contingent upon the continuing availability of the PAS in NHSScotland or a list price that is equivalent or lower.