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    Ravicti®

    Drug - Ravicti® (glycerol phenylbutyrate) [Horizon Therapeutics USA, Inc.]

    March 2025

    Therapeutic Area - Urea cycle disorders (UCD)

    Initial approval criteria

    • The patient has a diagnosis of hyperammonemia AND ALL of the following:
      • The patient has elevated ammonia levels according to the patient’s age [Neonate:plasma ammonia level 150 micromol/L (greater than 260 micrograms/dL) or higher; Older child or adult: plasma ammonia level greater than 100 micromol/L (175 micrograms/dL)] AND
      • The patient has a normal anion gap AND
      • The patient has a normal blood glucose level AND
    • The patient has a 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] AND
    • The requested agent will NOT be used as treatment of acute hyperammonemia AND
    • The patient is unable to maintain a plasma ammonia level within the normal range with the use of a protein restricted diet and, when clinically appropriate, essential amino acid supplementation AND
    • The patient will be using the requested agent as adjunctive therapy to dietary protein restriction AND
    • ONE of the following:
      • The patient has tried and had an inadequate response to generic sodium phenylbutyrate AND Pheburane OR
      • The patient has an intolerance or hypersensitivity to generic sodium phenylbutyrate AND Pheburane OR
      • The patient has an FDA labeled contraindication to generic sodium phenylbutyrate AND Pheburane OR
      • There is support for the use of the requested brand agent over generic sodium phenylbutyrate AND Pheburane AND
    • The prescriber is a specialist in the area of the patient’s diagnosis (e.g., metabolic disorders) or the prescriber has consulted with a specialist in the area of the patient’s diagnosis AND
    • The patient does NOT have any FDA labeled contraindications to the requested agent 

    Renewal criteria

    • The patient has been previously approved for the requested agent through the plan’s Prior Authorization process [Note: patients not previously approved for the requested agent will require initial evaluation review] AND
    • The patient has had clinical benefit with the requested agent (e.g., plasma ammonia level within the normal range) AND
    • The requested agent will NOT be used as treatment of acute hyperammonemia AND
    • The patient will be using the requested agent as adjunctive therapy to dietary protein restriction AND
    • ONE of the following:
      • The patient has tried and had an inadequate response to generic sodium phenylbutyrate AND Pheburane OR
      • The patient has an intolerance or hypersensitivity to generic sodium phenylbutyrate AND Pheburane OR
      • The patient has an FDA labeled contraindication to generic sodium phenylbutyrate AND Pheburane OR
      • There is support for the use of the requested brand agent over generic sodium phenylbutyrate AND Pheburane AND
    • The prescriber is a specialist in the area of the patient’s diagnosis (e.g., metabolic disorders) or the prescriber has consulted with a specialist in the area of the patient’s diagnosis AND
    • The patient does NOT have any FDA labeled contraindications to the requested agent

    Quantity limits

    • The requested quantity (dose) is within FDA labeled dosing for the requested indication.
    • Patient’s body surface area (in m2) and the corresponding requested number of 25-mL bottles must be submitted at time of request

    Background

    Ravicti is a nitrogen-binding agent indicated for chronic management of patients with urea cycle disorders (UCDs) 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.  Ravicti is not indicated for treatment of acute hyperammonemia in patients with UCDs because more rapidly acting interventions are essential to reduce plasma ammonia level.  The safety and efficacy of Ravicti for the treatment of N-acetylglutamate synthase (NAGS) deficiency has not been established.

    Questions

    Provider Call Center (844) 575-7887

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