Homozygous familial hypercholesterolemia (FH) is a congenital disorder that makes it difficult for your body to remove LDL cholesterol from your blood. Alirocumab is a biopharmaceutical medication approved as a second-line treatment for high cholesterol in adults whose cholesterol is independent of diet and statin treatment. This study aims to investigate the efficacy and safety of alirocumab for patients with homozygous FH.

This is a double-blind, randomized, parallel-group, placebo-controlled, phase-3 trial that included a total of 69 patients with homozygous FH. The patients were randomly assigned in a 2:1 ratio to alirocumab or placebo. The primary outcome of the study was the reduction in LDL-C levels, along with treatment-related adverse events.

At the beginning of the trial, the mean baseline LDL-C was 295.0 mg/dl in the alirocumab group and 259.6 mg/dl in the placebo group. At week 12, the least-squares mean difference in LDL-C percentage change was -26.9% in the alirocumab group and -8.6% in the placebo group. No treatment-related adverse events, such as treatment discontinuations or deaths, were reported during the study.

The research concluded that in patients with homozygous FH, alirocumab resulted in a significant reduction in LDL-C levels at week 12. Alirocumab was also well-tolerated with no adverse treatment-related events.

Ref: https://www.onlinejacc.org/content/76/2/131