Heterozygous familial hypercholesterolemia (HeFH) is an autosomal dominant disorder characterized 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. Praulent is expected to reach $183 million in sales by 2032, forecasts GlobalData, a leading data and analytics company.

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

Dr Shireen Mohammad, Cardiovascular & Metabolic Disorders Analyst at GlobalData, comments: “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 pediatric patents.”

In the Phase III study, 77.3% of participants achieved LDL-C less than 130 mg/dL, which is the LDL-C goal recommended in the pediatric guidelines.

Mohammad concludes: “The market still lacks therapeutics for pediatric familial hypercholesterolemia. 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. This new study offers the potential for higher sales of Praulent by 2032.”