Core Insights - Novartis announced that the FDA has approved the expanded indication for its siRNA therapy Leqvio (inclisiran), allowing it to be used as a monotherapy alongside diet and exercise to lower LDL-C levels in adults with hypercholesterolemia [1][2] - The FDA requested the label update based on positive data from the PCSK9-targeting therapy, removing the requirement for Leqvio to be used in conjunction with statins [1] - The label update also specifies the term "hypercholesterolemia" instead of "primary hyperlipidemia," emphasizing Leqvio's role in lowering LDL-C levels more accurately [1] Company Insights - Leqvio is a "first-in-class" siRNA therapy targeting PCSK9 mRNA, initially approved by the FDA in December 2021 as an adjunct to diet and statin therapy for adults with primary hyperlipidemia, including heterozygous familial hypercholesterolemia (HeFH) patients [2] - The mechanism of action involves reducing PCSK9 protein levels, which allows more LDL receptors to return to the liver cell surface, enhancing the clearance of LDL from the bloodstream [2] - Leqvio is noted as the first siRNA therapy capable of lowering LDL-C levels [2]