Background Familial chylomicronemia syndrome is a rare genetic disorder that is caused by
loss of lipoprotein lipase activity and characterized by chylomicronemia and recurrent
episodes of pancreatitis. There are no effective therapies. In an open-label study of three
patients with this syndrome, antisense-mediated inhibition of hepatic APOC3 mRNA with
volanesorsen led to decreased plasma apolipoprotein C-III and triglyceride levels. Methods
We conducted a phase 3, double-blind, randomized 52-week trial to evaluate the safety and …

Background Familial chylomicronemia syndrome (FCS) is a rare, autosomal recessive
genetic disorder characterized by a marked increase in plasma triglyceride (TG) levels and
recurrent episodes of pancreatitis. The response to conventional TG-lowering therapies is
suboptimal. Volanesorsen, an antisense oligonucleotide that targets hepatic apoC-III mRNA,
has been shown to significantly reduce TGs in patients with FCS. Objective To further
evaluate the safety and efficacy of extended treatment with volanesorsen in patients with …