Summary

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• This summary is based on the review of one randomized controlled trial. ^[1]
• In patients with genetically identified familial chylomicronemia syndrome (FCS), the 80 mg olezarsen group showed a significant reduction in fasting triglyceride levels at 6 months compared to placebo by -43.5 percentage points (95% Confidence Interval [CI]: -69.1 to -17.9; P < 0.001), while the 50 mg group showed a non-significant reduction of -22.4 percentage points (95% CI: -47.2 to 2.5; P = 0.08).
• The mean percent change in apolipoprotein C-III levels from baseline to 6 months compared to placebo was -73.7 percentage points (95% CI: -94.6 to -52.8) in the 80 mg group and -65.5 percentage points (95% CI: -82.6 to -48.3) in the 50 mg group.
• There were 11 episodes of acute pancreatitis in the placebo group and 1 episode in each olezarsen group, with a pooled rate ratio of 0.12 (95% CI: 0.02 to 0.66) for olezarsen versus placebo.
• Adverse events (AEs) of moderate severity considered related to the trial drug or placebo occurred in 4 patients in the 80 mg olezarsen group. No specific AEs or safety concerns were reported for the 50 mg olezarsen group or the placebo group.
• In the overall population of patients with genetically confirmed FCS (mean baseline triglyceride level 2630 ± 1315 mg/dL; 71% with prior acute pancreatitis within the past 10 years), treatment with 80 mg olezarsen resulted in significant reductions in triglyceride levels (-43.5 percentage points; 95% CI: -69.1 to -17.9; P < 0.001), apolipoprotein C-III levels (-73.7 percentage points; 95% CI: -94.6 to -52.8), and episodes of acute pancreatitis (rate ratio vs. placebo: 0.12; 95% CI: 0.02 to 0.66).