Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome

mRNA with volanesorsen led to decreased plasma apolipoprotein C-III and triglyceride levels.

We conducted a phase 3, double-blind, randomized 52-week trial to evaluate the safety and effectiveness of volanesorsen in 66 patients with familial chylomicronemia syndrome. Patients were randomly assigned, in a 1:1 ratio, to receive volanesorsen or placebo. The primary end point was the percentage change in fasting triglyceride levels from baseline to 3 months.