Dojolvi (triheptanoin oral liquid) — Medica

Initial criteria

• Patient has a molecularly confirmed diagnosis of a long-chain fatty acid oxidation disorder based on at least TWO of the following (disease-specific elevations of acylcarnitines on a newborn blood spot or in plasma; enzyme activity assay below the lower limit of normal; or genetic testing demonstrating a pathogenic variant in a gene associated with LC-FAODs)
• Patient will not use any other medium-chain triglyceride products concomitantly with Dojolvi
• Patient meets at least ONE of the following: (i) inadequate efficacy or significant intolerance to an over-the-counter medium-chain triglyceride product; OR (ii) history of at least one severe or recurrent manifestation of LC-FAODs such as cardiomyopathy, rhabdomyolysis, or hypoglycemia; OR (iii) currently receiving Dojolvi
• Medication is prescribed by or in consultation with a metabolic disease specialist or a physician who specializes in the management of long-chain fatty acid oxidation disorders

Approval duration

1 year