Pheburane — Blue Cross Blue Shield of Alabama

Initial criteria

• Diagnosis of hyperammonemia AND ALL of the following:
• - Elevated plasma ammonia levels according to age [Neonate: ≥150 micromol/L; Older child or adult: >100 micromol/L]
• - Normal anion gap
• - Normal blood glucose level
• - Diagnosis of one of the following urea cycle disorders confirmed by enzyme analysis OR genetic testing: carbamoyl phosphate synthetase I deficiency (CPSID), ornithine transcarbamylase deficiency (OTCD), argininosuccinic acid synthetase deficiency (ASSD), argininosuccinic acid lyase deficiency (ASLD), arginase deficiency (ARG1D)
• - Requested agent will NOT be used as treatment of acute hyperammonemia
• - Patient unable to maintain plasma ammonia within normal range despite protein restricted diet and, when appropriate, essential amino acid supplementation
• - Requested agent will be used as adjunctive therapy to dietary protein restriction
• AND ONE of the following brand-specific conditions:
• If the requested agent is Buphenyl or Olpruva: patient has intolerance or hypersensitivity to generic sodium phenylbutyrate OR FDA-labeled contraindication to generic sodium phenylbutyrate OR support for use of brand over generic sodium phenylbutyrate
• If the requested agent is Ravicti: patient has tried and had inadequate response to generic sodium phenylbutyrate AND Pheburane OR intolerance/hypersensitivity to both OR FDA-labeled contraindication to both OR support for use of brand over both
• - Prescriber is a specialist in metabolic disorders or has consulted with one
• - Patient has no FDA-labeled contraindications to the requested agent
• - Requested dose within FDA labeled dosing for indication

Reauthorization criteria

• Previously approved through plan’s PA process
• Documented clinical benefit (e.g., plasma ammonia levels within normal range)
• Agent not used for acute hyperammonemia treatment
• Used as adjunctive therapy to dietary protein restriction

Approval duration

12 months