RCT: Olezarsen effectively reduces triglyceride levels and pancreatitis episodes in familial chylomicronemia Syndrome

This phase 3, double-blind, placebo-controlled trial evaluated olezarsen in 66 patients with familial chylomicronemia syndrome. Participants received 80 mg or 50 mg of olezarsen or a placebo subcutaneously every 4 weeks for 49 weeks. The study found significant reductions in fasting triglyceride levels at the 6-month mark in the 80 mg olezarsen group compared to the placebo (-43.5 percentage points), with a similar trend observed in apolipoprotein C-III levels. Furthermore, the incidence of acute pancreatitis was significantly lower in both olezarsen groups compared to placebo by the 53-week endpoint. The findings suggest olezarsen as a potent therapy for reducing triglyceride levels and preventing pancreatitis in this population.

Reference (link to abstract – $ for full-text):

Erik S.G. Stroes et al. (2024). Olezarsen, Acute Pancreatitis, and Familial Chylomicronemia Syndrome. New England Journal of Medicine, Volume (Issue), Pages. DOI: 10.1056/NEJMoa2400201