Plozasiran: a small interfering RNA therapy for hypertriglyceridemia and familial chylomicronemia syndrome

Plozasiran (Redemplo Plozasiran (Redemplo®) is a small interfering RNA that reduces hepatic production of apolipoprotein C-III and circulating triglycerides. Across completed clinical studies, plozasiran demonstrated a generally favorable safety profile, with a low incidence of thrombocytopenia and adverse events primarily limited to abdominal pain, nasopharyngitis, headache, and nausea. Compared with earlier ApoC-III–targeted antisense oligonucleotides, plozasiran offers the advantages of less frequent dosing, sustained pharmacodynamic effects, and potentially a more favorable tolerability profile based on indirect evidence, positioning it as a promising therapeutic option for patients with familial chylomicronemia syndrome and other severe hypertriglyceridemia disorders inadequately managed with existing therapies.