Data from Pharmawand - Curated by EPG Health - Date added 19 November 2018
The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a change to the terms of the marketing authorisation for the medicinal product Ravicti (glycerol phenylbutyrate) from is Horizon Pharma Ireland Limited.
The CHMP adopted a change to the existing indication as follows: “Ravicti is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) including deficiencies of carbamoyl phosphate synthetase I (CPS), ornithine carbamoyltransferase (OTC), argininosuccinate synthetase (ASS), argininosuccinate lyase (ASL), arginase I (ARG) and ornithine translocase deficiency hyper ornithinaemia-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).”
Comment: Urea Cycle Disorders are metabolic diseases that affect a specific enzyme or transporter of the urea cycle, causing heightened levels of ammonia in the blood stream. Symptoms of the disorder can begin at any age, with more severe defects beginning early in life. UCD patients may experience episodes, called hyperammonemic crises, when ammonia levels in the blood become excessively high, which can result in irreversible brain damage, coma or death.