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PCSK9 inhibitors for treating familial hypercholesterolaemia

Nature Reviews Endocrinology volume 11, pages 8–9 (2015)Cite this article

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Familial hypercholesterolaemia is caused by mutations in genes that code for proteins involved in cholesterol metabolism. Patients heterozygous for mutations in LDLR respond to statin treatment, whereas individuals with homozygous LDLR mutations do not. PCSK9 inhibitors have been developed for treating familial hypercholesterolaemia, and results are promising for patients with either heterozygous or homozygous familial hypercholesterolaemia.

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Figure 1: Coadministration of PCSK9 inhibitors with statins lowers plasma levels of LDL cholesterol through enhanced expression of LDLR protein on the cell surface.

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Authors and Affiliations

  1. Department of Lipidology, Kanazawa University Graduate School of Medical Science, Takara-machi 13-1, Kanazawa, 920-8640, Japan

    Hiroshi Mabuchi & Atsushi Nohara

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  1. Hiroshi Mabuchi
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  2. Atsushi Nohara
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Correspondence to Hiroshi Mabuchi.

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Competing interests

H.M. and A.N. have received research grant funding from Aegerion, Astellas Pharma, AstraZeneca, Biopharm of Japan, Kowa, MSD, Sanofi, Shionogi and Takeda. H.M. has received an honoraria from AstraZeneca for an invited talk. A.N. has provided consulting to and is an advisory board member for Sanofi.

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Mabuchi, H., Nohara, A. PCSK9 inhibitors for treating familial hypercholesterolaemia. Nat Rev Endocrinol 11, 8–9 (2015). https://doi.org/10.1038/nrendo.2014.205

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