Physicians' Academy for Cardiovascular Education

Patients with heterozygous familial hypercholesterolemia benefit from PCSK9-antibody

Efficacy and safety of the proprotein convertase subtilisin/kexin type 9 monoclonal antibody alirocumab vs placebo with heterozygous familial hypercholesterolemia

Literature - Kastelein JJP, Hovingh GH, Langslet G et al. - J Clin Lipidol, 2017, DOI:


Only approximately 20% of patients with heterozygous familial hypercholesterolemia (HeFH) reach their LDL-C goal of ≤100 mg/dL when treated with statins, ezetimibe, and/or bile acid sequestrants [1,2]. On the other hand, the PCSK9 inhibitor alirocumab resulted in significant LDL-C reductions, both as monotherapy and in combination with statin therapy, in a variety of patient populations in the phase III ODYSSEY program [3-5].

In this analysis of four 78-week placebo-controlled ODYSSEY studies (FH I, FH II, long term and high FH), the LDL-C-lowering efficacy and safety of alirocumab Q2W were evaluated in 1257 HeFH patients on maximally tolerated dose of statins and other lipid-lowering therapies. Patients of the FH I and FH II trials received 75 mg Q2W or 150 mg Q2W and at week 12, some patients increased their dose from 75 to 150 mg Q2W if LDL-c was still ≥70 mg/dL at week 8. Patients of the long term and high FH received 150 mg Q2W from the start.

Main results


In four studies with HeFH patients, alirocumab resulted in significant LDL-C-lowering in most patients who were receiving the maximally tolerated dose of a statin, with or without other lipid-lowering therapies.


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