Efficacy and safety of volanesorsen in patients with multifactorial chylomicronaemia (COMPASS): a multicentre, double-blind, randomised, placebo-controlled, phase 3 trial
RCT (n=114) found volanesoren significantly reduced mean plasma triglyceride concentration vs placebo (-71.2% vs -0.9%, p<0.0001). 5 acute pancreatitis events (a potential consequence of hypertriglyceridaemia) occurred with placebo vs zero for volanesorsen.
Source:
The Lancet Diabetes & Endocrinology
SPS commentary:
A commentary provides context for the research stating that this is the latest study showing volanesorsen is efficacious across a wide range of plasma triglyceride concentrations and particularly in patients with multifactorial chylomicronaemia syndrome. The efficacy of treatment was independent of the type of genetic variants impairing lipoprotein lipase activity, which is a new finding that reinforces the notion that volanesorsen operates via lipoprotein lipase-independent mechanisms—eg, by inhibiting the production of triglyceride-rich lipoproteins.
Another important finding was that volanesorsen was equally efficacious in lowering triglycerides in patients taking and those not taking lipid-lowering treatments such as fibrates, statins, and fish oils. These data support the inclusion of APOC3 antisense therapy in future guidelines for managing patients with severe hypertriglyceridaemia.
Importantly however, COMPASS was not adequately powered to show an effect on the incidence of acute pancreatitis; hence, a large trial is required to test the effect of volanesorsen treatment in patients with multifactorial chylomicronaemia syndrome.