Drug Therapy for Hypertriglyceridemia and Familial Chylomicronemia Syndrome: Focus on Volnesorsen

Volanesorsen is a new medication that may soon be used in the treatment of hypertriglyceridemia and familial chylomicronemia syndrome (FCS). Volanesorsen works via binding to Apo C-III mRNA and degrading that mRNA, thus decreasing the synthesis of Apo C-III. This decreased synthesis of Apo C-III will increase the binding of triglycerides to LDL receptors and decrease triglyceride plasma levels. It is important to note that currently there are 3 other medication classes available for the treatment of hypertriglyceridemia, including niacin, fish oil/omega-3-fatty acids, and fibrates. However, there are no Food and Drug Administration-approved medications to treat FCS. Recently, volanesorsen was approved in the European Union for the treatment of FCS, but that indication was denied in the United States by the Food and Drug Administration. This was due to the side effects of the drug. Volanesorsen may cause a decrease in platelet count, renal toxicity, and elevate liver enzymes. The current drug regimen for volanesorsen consists of 285 mg once a week by subcutaneous injection, with a recommendation to immediately stop the medication if any of these side effects are noted, to prevent long-term complications. With these side effects reported, fish oil/omega-3-fatty acids seem likely the best choice when it comes to treating hypertriglyceridemia. If FCS is debilitating or greatly affecting the patient's life, then one could recommend volanesorsen. Otherwise, at this time, the side effects of volanesorsen may be too severe to justify its use for mild episodes of FCS or hypertriglyceridemia.