Metreleptin: First Global Approval
Metreleptin: First Global Approval
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DOI：
10.1007/s40265-013-0074-7
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发表时间：
2013-06-01
期刊：
影响因子：
11.5
通讯作者：
Perry, Caroline M.
中科院分区：
文献类型：
--
作者：
Chou, Ken;Perry, Caroline M.
Metreleptin是由Amylin Pharmaceuticals （Bristol-Myers Squibb的子公司）开发的人类激素leptin的类似物，用于包括脂肪营养不良在内的代谢紊乱的皮下治疗。该化合物有望改善对常规治疗无反应的脂肪营养不良患者的胰岛素敏感性、高甘油三酯血症和高血糖。美曲瘦素已在日本被批准作为一种治疗脂肪营养不良的瘦素疗法。Amylin还完成了向美国FDA提交的metreleptin用于治疗罕见形式脂肪营养不良患者的糖尿病和/或高甘油三酯血症的审批。该药的临床开发也正在美国进行，用于治疗1型糖尿病。安进此前正在评估使用美曲瘦素治疗闭经；然而，这一适应症的发展似乎已经停止。本文总结了美曲瘦素首次获批用于治疗脂肪营养不良的发展历程。
Metreleptin is an analogue of the human hormone leptin being developed by Amylin Pharmaceuticals (a subsidiary of Bristol-Myers Squibb) for the subcutaneous treatment of metabolic disorders including lipodystrophy. The compound is expected to improve insulin sensitivity, hypertriglyceridaemia and hyperglycaemia in patients with lipodystrophy who are unresponsive to conventional treatment. Metreleptin has been approved in Japan as a leptin therapy for the treatment of lipodystrophy. Amylin has also completed a submission for regulatory approval to the US FDA for metreleptin in the treatment of diabetes mellitus and/or hypertriglyceridaemia in patients with rare forms of lipodystrophy. Clinical development of the drug is also underway in the USA for the treatment of type 1 diabetes. Amgen was previously assessing the use of metreleptin as a treatment for amenorrhoea; however, it appears that development in this indication has been discontinued. This article summarizes the milestones in the development of metreleptin leading to this first approval for lipodystrophy.