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Drugs. 2015 Sep;75(13):1567-73. doi: 10.1007/s40265-015-0460-4.
Evolocumab (Repatha™) is a fully human monoclonal antibody developed by Amgen that has been approved as a treatment for hypercholesterolaemia in the EU, and is awaiting approval in the USA and Japan. It specifically binds proprotein convertase subtilisin/kexin type 9 (PCSK9)-a negative regulator of low-density lipoprotein (LDL)-receptors-thereby improving the ability of the liver to bind LDL-cholesterol (LDL-C), leading to reduced LDL-C blood levels. The drug reduces LDL-C levels in patients with hypercholesterolaemia when used as monotherapy or in conjunction with a statin. This article summarizes the milestones in the development of evolocumab leading to this approval for the treatment of adults with primary hypercholesterolaemia (heterozygous familial and non-familial) or mixed dyslipidaemia as an adjunct to diet with or without a statin and/or other lipid lowering therapies, and in adults and adolescents aged ≥12 years with homozygous familial hypercholesterolaemia in combination with other lipid lowering therapies.
依洛尤单抗（瑞百安）是安进公司开发的一种全人源单克隆抗体，已在欧盟获批用于治疗高胆固醇血症，目前正在美国和日本等待批准。它特异性结合前蛋白转化酶枯草溶菌素/克那霉 9（PCSK9），PCSK9 是一种低密度脂蛋白（LDL）受体的负调节剂，从而提高肝脏结合 LDL-胆固醇（LDL-C）的能力，导致 LDL-C 血药浓度降低。该药作为单药治疗或与他汀类药物联合使用时，可降低高胆固醇血症患者的 LDL-C 水平。本文总结了依洛尤单抗开发过程中的重要里程碑，该药获批用于治疗原发性高胆固醇血症（杂合子家族性和非家族性）或混合血脂异常的成年患者，联合或不联合饮食、他汀类药物和/或其他降脂疗法，以及联合其他降脂疗法治疗年龄≥12 岁的杂合子家族性高胆固醇血症的成年和青少年患者。
