• Pierre Sabouret, Michel Farnier, and Etienne Puymirat.
• Pitié-Salpétrière Hospital and ACTION-Group, Heart Institute, Cardiology Department, 47-83, boulevard de l'Hôpital, 75013 Paris, France.
• Presse Med. 2019 Mar 1; 48 (3 Pt 1): 227-237.

AbstractPCSK9 protein is a key regulator of LDL receptor activity. Gain-of-function mutations in PCSK9 are one of the genetic causes of familial hypercholesterolemia. Conversely, loss-of-function mutations are associated with lower levels of LDL cholesterol and reduced coronary heart disease. Monoclonal antibodies targeting PCSK9 are highly efficacious in lowering LDL-C levels, with a good tolerability and safety profile. Two PCSK9 inhibitors, alirocumab and evolocumab, have demonstrated a cardiovascular benefit in addition to statin therapy in patients with established cardiovascular disease. A recent European consensus has defined the candidates for PCSK9 inhibitors, e.g., patients with established cardiovascular disease and patients with familial hypercholesterolemia in primary prevention, with substantially elevated LDL-C levels despite maximally tolerated statin with or without ezetimibe therapy.Copyright © 2019 Elsevier Masson SAS. All rights reserved.

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