ApoC[III] You Later: A Review of Plozasiran for the Management of Familial Chylomicronemia Syndrome

OBJECTIVE: The purpose of this review is to evaluate the efficacy and safety of plozasiran, a novel agent recently approved by the US Food and Drug Administration for familial chylomicronemia syndrome (FCS). DATA SOURCES: A comprehensive literature search was conducted in PubMed and the National Institutes of Health Clinical Trials Registry (http://www. CLINICALTRIALS: gov) from April 2014 to April 2026 using the keyword "plozasiran." Trials that were phase 1 trials, review papers, pooled analyses of trials, or ongoing studies without complete results were excluded. STUDY SELECTION AND DATA EXTRACTION: Phase 2 and 3 trials assessing plozasiran's safety and efficacy were included. Initially, 57 articles were yielded, and 3 studies were included after application of inclusion criteria. DATA SYNTHESIS: The findings from this review indicate that plozasiran is overall a safe and effective medication to significantly lower triglycerides and acute pancreatitis in patients with FCS.Relevance to Patient Care and Clinical Practice in Comparison to Existing Drugs:Plozasiran is a novel agent that targets a similar pathway as olezarsen but requires less frequent administration (4 times a year for plozasiran vs 12 times a year for olezarsen). While this may help with patient adherence compared to olezarsen, plozasiran's higher annual cost (approximately $72 000/year) compared to olezarsen ($40 000/year) may limit use for FCS management. CONCLUSIONS: Plozasiran offers an alternative pharmacological agent to olezarsen for management of FCS in the United States. Currently, there are limited data evaluating the long-term outcomes of plozasiran use in patients with FCS.