plozasiran

Plozasiran is a quarterly subcutaneous RNAi therapy developed by Arrowhead Pharmaceuticals that silences the APOC3 gene in liver cells to dramatically reduce apolipoprotein C-III (ApoC-III) — a protein that inhibits the triglyceride-clearing enzyme lipoprotein lipase. In familial chylomicronemia syndrome (FCS), biallelic LPL mutations make patients genetically unable to clear dietary fat, producing triglyceride levels that can exceed 1,000 mg/dL and recurrent life-threatening pancreatitis attacks; ApoC-III suppression restores the patient's residual triglyceride clearance capacity. Plozasiran is in a Phase 3 pivotal trial (PALISADE) for FCS, competing directly with Ionis's olezarsen in the same indication.