Heterozygous familial hypercholesterolemia (HeFH) is an autosomal dominant disorder characterised by elevated levels of low-density lipoprotein cholesterol (LDL-C), leading to an increased risk of cardiovascular disease. Praluent (alirocumab) is a monoclonal antibody against proprotein convertase subtilisin kexin type 9 (PCSK9), an enzyme that promotes degradation of the low-density lipoprotein (LDL) receptor, which is responsible for clearing excess LDL-C from the blood.

A Phase III study reported in JAMA Pediatrics demonstrated that alirocumab significantly reduced LDL-C and other lipid parameters in paediatric patients aged eight to 17 years with HeFH after 24 weeks of treatment. LDL-C reduction was observed in 43.3% of patients dosed every two weeks and 33.8% in patients dosed every four weeks.

HeFH is an inherited disorder, hence early treatment to control LDL-C is important. This study offers hope to start treatment in patients as young as eight years old with familial hypercholesterolemia. While statins are the standard of care for dyslipidemia, some pediatric patients are unable to achieve their LDL-C targets and lipid-lowering therapy is used in combination with statins for those patients where statin treatment alone is not sufficient. Therefore, alirocumab could be used as an adjunct option to lipid-lowering therapy in paediatric patients. In this study, 77.3% of participants achieved LDL-C less than 130mg/dL, which is the LDL-C goal recommended in the pediatric guidelines.

Addressing these unmet needs in pediatric familial hypercholesterolemia is important for improving the overall care and outcomes of affected pediatric patients, reducing the risk of cardiovascular events, and promoting a better quality of life. Praulent is expected to reach $183m in sales by 2032, however, this new study offers the potential for higher sales.