01 Mar 2024
For: General public
Deferred
The drug Ravicti (Glycerol Phenylbutyrate) was authorized to be used in hospitals in the indication: “as adjuvant therapy for the chronic treatment of patients with urea cycle diseases (UCD) including deficiencies of carbamylphosphate synthetase I (CPS), carbamoyl ornithine transferase (OTC), arginine succinate synthetase (ASS), arginine succinate ligase (ASL), arginase I (ARG) and ornithine translocase (hyperornithinemia-hyperammonemiahomocitrulinemia syndrome [HHH]) that cannot be treated solely through dietary protein restriction and/or amino acid supplementation. Ravicti must be used in conjunction with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free caloric supplements).”
The public report of this evaluation is available on Infomed, on the medicine page, as well as on the Infarmed page dedicated to financing decisions.
Tags: Ravicti Glycerol Phenylbutyrate Public financing evaluation report Infomed Approval
 Public financing evaluation report available on Infomed – Approval){kind=link}