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Paediatr Drugs. 2024 Jul;26(4):469-474. doi: 10.1007/s40272-024-00637-7.
Alirocumab (Praluent), a proprotein convertase subtilisin kexin type 9 (PCSK9) inhibitor that has been co-developed by Regeneron Pharmaceuticals, Inc. and Sanofi (formerly sanofi-aventis), is approved globally for use in adults with established cardiovascular disease, primary hyperlipidemia [including heterozygous familial hypercholesterolemia (HeFH) or homozygous familial hypercholesterolemia (HoFH)]. In November 2023, based on clinical data in patients aged 8-17 years, alirocumab received its first pediatric approval in the EU as an adjunct to diet alone, or in combination with a statin and/or other low-density lipoprotein cholesterol (LDL-C)-lowering therapies, in pediatric patients aged ≥ 8 years with HeFH. Alirocumab was approved a few months later in the US for use as an adjunct to diet and other LDL-C-lowering therapies in pediatric patients aged ≥ 8 years with HeFH to reduce LDL-C. This article summarizes the milestones in the development of alirocumab leading to this first pediatric approval for HeFH.
阿利西尤单抗（瑞百安），一种由再生元制药公司和赛诺菲（前身为赛诺菲-安万特）共同开发的前蛋白转化酶枯草溶菌素 9（PCSK9）抑制剂，已在全球范围内获得批准，用于治疗已确诊心血管疾病的成年人、原发性高脂血症[包括杂合子家族性高胆固醇血症（HeFH）或纯合子家族性高胆固醇血症（HoFH）]。2023 年 11 月，基于 8-17 岁患者的临床数据，阿利西尤单抗在欧盟首次获得儿科批准，作为饮食辅助疗法，或与他汀类药物和/或其他降低低密度脂蛋白胆固醇（LDL-C）的疗法联合使用，用于 8 岁及以上 HeFH 儿科患者。几个月后，该药在美国获得批准，用于 8 岁及以上 HeFH 儿科患者，与饮食和其他 LDL-C 降低疗法联合使用，以降低 LDL-C。本文总结了阿利西尤单抗研发过程中的重要里程碑，最终使其获得了 HeFH 的首次儿科批准。
