Methods for treating hybrid familial hypercholesterolemia (heFH) patients

By combining PCSK9 inhibitors with statins, the problem of existing treatments being unable to effectively control heterozygous familial hypercholesterolemia has been solved, resulting in a significant reduction in LDL-C and improvement in serum levels of various lipid components, thereby reducing the risk of cardiovascular disease.

CN122124232APending Publication Date: 2026-06-02SANOFI BIOTECH SAS +1
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Patent Information

Application Number
CN202610219438.1
Authority / Receiving Office
CN · China
Patent Type
Applications(China)
Current Assignee / Owner
Priority Date
2015-03-23
Filing Date
2015-07-16
Publication Date
2026-06-02

AI Technical Summary

Technical Problem

Current maximum tolerated dose statin therapy is ineffective in controlling low-density lipoprotein cholesterol levels in patients with heterozygous familial hypercholesterolemia, leading to an increased risk of cardiovascular disease.

Method used

PCSK9 inhibitors are used in combination with the maximum tolerated dose of statins, including antibodies that specifically bind to PCSK9 or their antigen-binding fragments, to treat patients with heterozygous familial hypercholesterolemia. This involves administering one or more doses of PCSK9 inhibitors to lower LDL cholesterol and improve serum levels of other lipid components.

Benefits of technology

Significantly reduces low-density lipoprotein cholesterol (LDL-C) by at least 40%, apolipoprotein B (ApoB) by at least 30%, non-high-density lipoprotein cholesterol (non-HDL-C) by at least 40%, total cholesterol by at least 20%, triglycerides by at least 5%, lipoprotein a (Lp(a)) by at least 20%, and increases high-density lipoprotein cholesterol (HDL-C) by at least 3% and apolipoprotein A1 by at least 1%.

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Abstract

The present invention relates to a method for treating a patient with heterozygous familial hypercholesterolemia (heFH), comprising administering to a patient with heterozygous familial hypercholesterolemia a pharmaceutical composition comprising a PCSK9 inhibitor. In some embodiments, the PCSK9 inhibitor is an anti-PCSK9 antibody, such as an exemplary antibody referred to herein as mAb316P. The methods of the invention are useful for treating patients suffering from heterozygous familial hypercholesterolemia that are insufficiently controlled with maximum tolerated dose statin treatment with or without other lipid lowering treatments.
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