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Pemafibrate is a novel, highly selective peroxisome proliferator–activated receptor alpha (PPARα) modulator, classified as a selective PPARα modulator (SPPARMα). It acts by binding to PPARα and regulating the expression of genes involved in lipid metabolism, resulting in decreased plasma triglyceride levels and increased high-density lipoprotein cholesterol. Pemafibrate is significantly more potent and selective for PPARα than conventional fibrates such as fenofibrate. Its unique Y-shaped structure allows for complete activation of the PPARα receptor, conferring distinct pharmacokinetic and pharmacodynamic properties compared to older fibrates. Developed by Kowa Pharmaceutical, it was first approved in Japan in 2017 for hyperlipidaemia (including familial hyperlipidaemia), with subsequent approvals or development ongoing globally for dyslipidaemias, hypertriglyceridaemia, diabetic retinopathy, non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH), and primary biliary cirrhosis[1][3][4][5][6][7].
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