alirocumab

Alirocumab is a human monoclonal antibody that inhibits proprotein convertase subtilisin/kexin type 9 (PCSK9). By binding PCSK9, it prevents degradation of LDL receptors on hepatocytes, promoting recycling of these receptors and increased clearance of low-density lipoprotein cholesterol (LDL-C) from the bloodstream. This mechanism lowers LDL-C and can modestly affect non-HDL cholesterol and apolipoprotein B.

Medical uses and indications vary by jurisdiction, but alirocumab is generally indicated as an adjunct to diet and maximally tolerated statin therapy for adults with heterozygous familial hypercholesterolemia or established atherosclerotic cardiovascular disease to reduce LDL-C. In some regulatory regions, pediatric patients (for example, those aged 12 years and older) with heterozygous familial hypercholesterolemia may also be eligible. It is not a substitute for lifestyle changes and is not typically used as monotherapy.

Administration and dosing typically involve subcutaneous injections every two weeks. Common dosing regimens include 75 mg or 150 mg every two weeks, with the possibility of increasing to 150 mg every two weeks if needed to reach LDL-C targets; some markets offer a 300 mg monthly option for appropriate patients.

Clinical efficacy has shown substantial LDL-C reductions, often around 50–60% when added to statin therapy. Trials such as the ODYSSEY program have reported reductions in major adverse cardiovascular events in high-risk patients on background lipid-lowering therapy.

Safety and tolerability are generally favorable. The most common adverse events are injection-site reactions, upper respiratory infections, and back pain; hypersensitivity and rare neurocognitive adverse events have been reported. Alirocumab is administered by healthcare professionals or patients trained to self-inject, with monitoring for adverse effects as appropriate.