This study evaluated the efficacy and safety of alirocumab in pediatric patients with heterozygous familial hypercholesterolemia (HeFH) who were inadequately controlled with statins. The randomized, double-blind, placebo-controlled trial included 153 patients aged 8 to 17 years with LDL-C levels of 130 mg/dL or higher. Patients were randomized 2:1 to receive either alirocumab or placebo, with dosing options of every 2 weeks (Q2W) or every 4 weeks (Q4W). The primary endpoint was the percent change in LDL-C from baseline to week 24. Results showed that alirocumab significantly reduced LDL-C by -43.3% (Q2W) and -33.8% (Q4W) compared to placebo, with improvements maintained through week 24 and week 104. Alirocumab was well tolerated, with no significant differences in adverse event incidence between treatment groups. The study concluded that alirocumab is a suitable adjunct therapy for pediatric patients with HeFH, providing significant LDL-C reduction and other lipid parameter improvements.This study evaluated the efficacy and safety of alirocumab in pediatric patients with heterozygous familial hypercholesterolemia (HeFH) who were inadequately controlled with statins. The randomized, double-blind, placebo-controlled trial included 153 patients aged 8 to 17 years with LDL-C levels of 130 mg/dL or higher. Patients were randomized 2:1 to receive either alirocumab or placebo, with dosing options of every 2 weeks (Q2W) or every 4 weeks (Q4W). The primary endpoint was the percent change in LDL-C from baseline to week 24. Results showed that alirocumab significantly reduced LDL-C by -43.3% (Q2W) and -33.8% (Q4W) compared to placebo, with improvements maintained through week 24 and week 104. Alirocumab was well tolerated, with no significant differences in adverse event incidence between treatment groups. The study concluded that alirocumab is a suitable adjunct therapy for pediatric patients with HeFH, providing significant LDL-C reduction and other lipid parameter improvements.