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.