December 22, 2021, the U.S. Food and Drug Administration (FDA) approved Leqvio® (inclisiran) for additional low-density lipoprotein cholesterol (LDL-C) lowering in adult patients with atherosclerotic cardiovascular disease (ASCVD) or heterozygous familial hypercholesterolemia (HeFH) currently being treated with maximally tolerated statin therapy. ASCVD is a group of diseases in which patients have a build-up of cholesterol plaque in their arteries and may lead to heart conditions such as sudden reduced blood flow to the heart, heart attack and stroke. HeFH, which affects approximately 1 in 250 people, is a life-threatening condition characterized by a mutation in the genes responsible for the natural lowering of cholesterol levels, often resulting in significantly higher levels of LDL-C. LDL-C, also referred to as “bad cholesterol”, is a type of protein that carries cholesterol throughout the body and high levels are associated with an increased risk for heart attack, stroke and coronary artery disease. Leqvio® is administered by a health care provider and is given as a 284 mg injection initial dose followed by a second dose three months later, and then every six months thereafter. Leqvio® works by interfering with genetic material in liver cells resulting in decreased levels of a protein called PCSK9. By blocking PCSK9, a patient’s liver can better regulate LDL-C levels, including an increased ability to remove LDL-C from the blood. The efficacy of Leqvio® was studied in three randomized clinical trials enrolling 3,457 adult patients with either HeFH or ASCVD who were currently receiving maximally tolerated statin therapy but had not achieved their targeted LDL-C level. In each trial, the participants were divided into two groups and given four doses of either placebo or 284 mg Leqvio® on day 1, 90, 270 and 450. The results of the three studies all showed positive results with the group receiving Leqvio® having a decreased LDL-C of 51%, 46%, and 40%, whereas the placebo groups had an increased LDL-C of 1%, 4%, and 8%, respectively. Common side effects of Leqvio® include injection site reaction, joint pain, urinary tract infection, diarrhea, pain in legs or arms and shortness of breath. This biannual injection provides a new treatment option for high-risk patients who are not at their recommended LDL-C target and is considered an adjunct therapy with a balanced diet and statin therapy.