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