Familial hypercholesterolemia (FH ) is one of the most common fatal genetic diseases, affecting over 25,000 Belgians. it is responsible for very high cholesterol levels (> 300mg/dL) from birth, along with an increased risk of early vascular, cardiac, and cerebral complications, such as myocardial infarction and stroke, from the age of 30 years onwards in men and 40 in women. Cardiovascular complications may, however, be prevented by means of early diagnosis and proper treatment, ideally started in childhood.
The 2003 discovery of a new protein called PCSK9 (proprotein convertase subtilisin/kexin type 9), involved in LDL particle metabolism, resulted in the development of a new class of highly potent cholesterol-lowering agents. Given this context, several monthly or bimonthly subcutaneously administered monoclonal antibodies that inhibit PCSK9 (evolocumab, Amgen; alirocumab, Sanofi/Regeneron; bococizumab, Pfizer) were shown to reduce LDL cholesterol by 45-75%, with a very good tolerance and safety profile. This paved the way for a new therapeutic strategy aimed at helping patients achieve their LDL-cholesterol targets while compensating the statins' limitations of use.