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    Dojolvi™

    Drug - Dojolvi™ (triheptanoin) [Ultragenyx Pharmaceutical Inc.]

    March 2021

    Therapeutic area - Fatty acid oxidation disorder

    Initial approval criteria

    • Patient must have a diagnosis of a long-chain fatty acid oxidation disorder (LC-FAOD) confirmed by the following:
      • 1 or more definitive pathogenic mutations in any LC-FAOD gene OR
      • Two of the following:
        • 1 or more non-definitive pathogenic mutations in any LC-FAOD gene OR
        • Low enzyme activity in cultured fibroblasts OR
        • Disease specific elevations of acylcarnitines on a newborn blood spot or in plasma AND
    • Patient must have severe LC-FAOD confirmed by a history of ≥ 1 of the following despite therapy:
      • Chronic elevated creatine kinase ([CK] ≥ 2 times the upper limit of normal) with ≥ 2 major clinical events (e.g., hospitalizations, hypoglycemia, cardiomyopathy, and rhabdomyolysis) OR
      • Episodic elevated CK with reported muscle dysfunction (e.g., frequent muscle fatigue, exercise intolerance, limitation of exercise) OR
      • Highly elevated CK (≥ 4 times the upper limit of normal) OR
      • Frequent (≥ 3 within a year or ≥ 5 within 2 years) severe major clinical events (e.g., hospitalizations, hypoglycemia, cardiomyopathy, and rhabdomyolysis) OR
      • Severe susceptibility to hypoglycemia after short periods of fasting (≥ 2 events within a year that require ongoing prophylactic management or recurrent symptomatic hypoglycemia requiring intervention ≥ 2 times per week) OR
      • Evidence of functional cardiomyopathy (echocardiogram documenting poor ejection fraction) AND
    • Patient is being followed by a clinical specialist knowledgeable in appropriate disease-related dietary management (e.g., medical geneticist, genetic metabolic disorders, or a physician with a board certification in nutrition) AND
    • Patient is NOT taking a pancreatic lipase inhibitor (e.g., orlistat) AND
    • Patient will NOT receive an additional medium chain triglyceride while taking triheptanoi

    Renewal criteria

    • Patient must continue to meet the above criteria AND
    • Patient must demonstrate disease improvement and/or stabilization (e.g., cardiac function, exercise tolerance, reduction in major clinical events, including hospitalization) AND
    • Patient does NOT experience serious treatment-related adverse effects

    Quantity limits

    • Requested quantity (in mL) and days supplied must be clearly stated on the prior authorization request form; and they must be supported by the following information:
      • Patient’s total prescribed daily caloric intake (DCI)
      • Patient’s target % dose of DCI

    Questions?

    MHCP Provider Call Center 651-431-2700 or 800-366-5411

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