Ravicti is a medicine used to manage urea cycle disorders in adults and children, when the diseases cannot be managed by changes in diet alone. Patients with urea cycle disorders are not able to get rid of waste nitrogen from the body because they lack some liver enzymes. In the body, waste nitrogen is turned into ammonia, which is harmful when it accumulates. Ravicti is used in patients who lack one or more of the following enzymes: carbamoyl phosphate synthase-I, ornithine carbamoyltransferase, argininosuccinate synthetase, argininosuccinate lyase, arginase I and ornithine translocase. Ravicti contains the active substance glycerol phenylbutyrate. Urea cycle disorders are rare, and Ravicti was designated an ‘orphan medicine’ (a medicine used in rare diseases) for several forms of the disease on 10 June 2010.
Therapeutic Indication
### Therapeutic indication Ravicti is indicated for use as adjunctive therapy for chronic management of patients 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. 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).
Therapeutic Area (MeSH)
ATC Code
A16AX09
ATC Item
glycerol phenylbutyrate
Pharmacotherapeutic Group
Other alimentary tract and metabolism products
Active Substance (Summary)
INN / Common Names
| Substance | CAS | Monograph |
|---|---|---|
| glycerol phenylbutyrate | N/A | 苯丁酸甘油酯 |
EMA Name
Ravicti
Medicine Name
Ravicti
Aliases
N/A
