Alirocumab—a novel drug for familial hypercholesterolemia

Familial hypercholesterolemia (FH) is a genetically transmitted condition, wherein abnormally high levels of total cholesterol and low density lipoproteins (LDL) are seen. Occurrence of premature cardiovascular complications is common, resulting in significant morbidities and fatalities. Currently statins are the first-choice drugs to treat FH, given alone, or in combination with other lipid lowering drugs. Alirocumab is a monoclonal antibody directed against PCSK9, a ‘gain-of-function’ mutation of which is thought to be responsible for the defective processing of LDL cholesterol, resulting in lesser uptake of LDL-C into the hepatocytes. It was approved by the US-FDA in July 2015 as a first-in-class drug, “for use in addition to diet and maximally tolerated statin therapy in adult patients with heterozygous familial hypercholesterolemia (HeFH) or patients with clinical atherosclerotic cardiovascular disease such as heart attacks or strokes, who require additional lowering of LDL cholesterol.” It has to be administered subcutaneously every 2 weeks, and the dose is titrated as per need. Injection-site reactions, myalgia, neurocognitive events, pruritus were the major adverse effects observed during clinical trials. Inhibition of PCSK 9 is a new promising target for the treatment of patients with familial hypercholesterolemia.