It is a second-generation[3] 2'-O-methoxyethyl (2'-MOE) chimeric antisense therapeutic oligonucleotide (ASO)[4] that targets the messenger RNA for apolipoprotein C3 (apo-CIII).

The most common side effects include reduced platelet levels and reactions at the site of the injection such as pain, swelling, itching, or bruising.

[5] Fat accumulation can also cause repeated bouts of pancreatitis (inflammation of the pancreas) and xanthomas (formation of yellow fatty deposits just under the skin, generally around joints)[5] Volanesorsen is indicated as an adjunct to diet in adults with genetically confirmed familial chylomicronemia syndrome (FCS) and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate.

Volanesorsen was designated an orphan drug by the European Medicines Agency (EMA) in February 2014, for phosphorothioate oligonucleotide targeted to apolipoprotein C-III for treatment of familial chylomicronaemia syndrome.

[5] Volanesorsen was effective in reducing triglycerides in the blood in a study of 67 participants with familial chylomicronemia syndrome (FCS).