Alirocumab Evaluated for Treatment of Homozygous Familial Hypercholesterolemia


Alirocumab significantly reduced low-density lipoprotein cholesterol in adults with homozygous familial hypercholesterolemia, according to phase 3 data. 


Alirocumab significantly reduced low-density lipoprotein (LDL) cholesterol in adults with homozygous familial hypercholesterolemia (HoFH), according to data from a phase 3 clinical trial presented at the American College of Cardiology’s Annual Scientific Session Together with World Congress of Cardiology (ACC.20/WCC).

Alirocumab, a PCSK9 inhibitor, is FDA-approved for the treatment of high LDL cholesterol levels in adults alone or in combination with other lipid-lowering therapies. Although alirocumab has been evaluated for use in a wide range of patients with high cholesterol, this is the first study to assess the efficacy and safety of the drug in patients with HoFH.

“This is a worthwhile potential treatment that helps to lower LDL cholesterol, though a lot of patients still require additional therapy because the LDL cholesterol is so high at baseline,” lead study author Dirk Blom, MD, PhD, head of the Division of Lipidology in the Department of Medicine at the University of Cape Town in Cape Town, South Africa, said in a press release. “Alirocumab is a potential new therapy that should be considered in appropriate patients with HoFH.”

For the study, 69 patients with HoFH were enrolled. Of these patients, 45 were randomly assigned to receive alirocumab via subcutaneous injection every 2 weeks for 12 weeks. The rest received placebo injections. All patients in the study maintained their regular cholesterol medications and treatments, such as statins, ezetimibe, lomitapide, and apheresis, but did not take any other proprotein convertase subtilisin/kexin type 9 (PSCK9) inhibitors.

Overall, average LDL cholesterol levels among those who received alirocumab were 26.9% lower than at the beginning of the trial, according to the results. Among those who received a placebo, LDL cholesterol levels rose by 8.6%, resulting in an average reduction of 35.6% among those receiving alirocumab.

The average absolute reduction in LDL cholesterol in the patients receiving alirocumab was 62.8 mg/dL, according to the study.

Additionally, compared with placebo, alirocumab was also found to substantially reduce other harmful lipids that are typically elevated in individuals with FH: apolipoprotein B dropped by 29.8%, lipoprotein(a) dropped by 28.4%, non-high-density cholesterol dropped by 32.9%, and total cholesterol dropped by 26.5%.

Alirocumab was generally well-tolerated, with no treatment-relate serious adverse events that occurred in the trial.

The study was funded by Regeneron Pharmaceuticals.



1. Alirocumab Substantially Reduces Cholesterol in Adult Patents with HoFH [news release]. American College of Cardiology’s website. Accessed March 31, 2020.

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