Table 4.

Lipid-lowering agents for the treatment of FH in children/adolescents.

Lipid Lowering Agent	Mechanism of Action	HeFH Children	HoFH Children
statins	competitive inhibition of 3-hydroxy-3-methylglutaryl coenzyme A (HMG-CoA) reductase	from 10 years (8 years for pravastatin)	in association with ezetimibe/other lipid lowering drugs
bile acid sequastrants	inhibition of intestinal cholesterol absorption	from 8 years (fat soluble vitamins supplementation)	in association with statins/other lipid lowering drugs
ezetimibe	selective inhibition of intestinal cholesterol absorption (inhibition of the sterol transporter Niemann-Pick C1L1)	from 10 years	in association with statins/other lipid lowering drugs
lomitapide	inhibits microsomal triglyceride transfer protein (MTP)	approved for the treatment of HoFH in adults (EMA-AIFA)	in clinical trials for HoFH (NCT04681170)
mipomersen	antisense oligonucleotide against the coding region of apolipoprotein B mRNA	approved for the treatment of HoFH in adults (FDA-not EMA)	not approved
PCSK9 inhibitors	prevent the breackdown of LDL receptors	approved for the treatment of FH in adults (EMA-AIFA)	in clinical trials for HoFH (NCT03510715) and HeFH (NCT03510884)