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Drugs. 2013 Jun;73(9):989-97. doi: 10.1007/s40265-013-0074-7.
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.
美曲普汀是一种人源化瘦素类似物，由 Amylin 制药公司（百时美施贵宝的子公司）研发，用于治疗包括脂肪营养不良在内的代谢紊乱。该药有望改善脂肪营养不良患者对常规治疗无反应者的胰岛素敏感性、高甘油三酯血症和高血糖。美曲普汀已在日本获准作为治疗脂肪营养不良的瘦素疗法。Amylin 还向美国 FDA 提交了 metreleptin 治疗罕见脂肪营养不良患者糖尿病和/或高甘油三酯血症的监管批准申请。该药在美国也在进行治疗 1 型糖尿病的临床开发。安进公司曾评估 metreleptin 治疗闭经的用途，但似乎已停止该适应证的开发。本文总结了美曲普汀的开发里程碑，最终该药获得了脂肪营养不良的首次批准。
