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glycerol phenylbutyrate (Ravicti®)


Reference No. 2127


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: In progress
Advice No: Not available
Ratification by Welsh Government: Not available

Current Progress


Submission
received
NMG
meeting
AWMSG
meeting
Ratification by
Welsh Government

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)
AWMSG Secretariat Appraisal Report (ASAR)
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Preliminary Appraisal Recommendation (PAR)
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Company Response to the Preliminary Appraisal Recommendation (CR PAR)
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