预约演示
更新于：2026-04-02
Metreleptin
美曲普汀
更新于：2026-04-02
概要
基本信息
药物类型 多肽激素 |
别名 Human recombinant methionyl leptin、Methionyl human leptin、Metreleptin (Genetical Recombination) + [10] |
靶点 |
作用方式 激动剂 |
作用机制 LEPR激动剂(瘦素受体激动剂) |
在研适应症 |
最高研发阶段批准上市 |
首次获批日期 日本 (2013-03-25), |
最高研发阶段(中国)- |
特殊审评孤儿药 (美国)、孤儿药 (欧盟)、孤儿药 (日本)、孤儿药 (澳大利亚)、快速通道 (美国) |
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结构/序列
Sequence Code 59859
来源: *****
关联
46
项与 美曲普汀 相关的临床试验NCT06502990
Open-label, Phase 3b Study to Evaluate Effectiveness, Safety and Pharmacokinetic Parameters of Metreleptin in Patients Under 6 Years of Age With Generalised Lipodystrophy and Associated Diabetes Mellitus and/or Hypertriglyceridaemia
NCT06679270
An Open-label Extension of APG-20 Study to Evaluate the Long-term Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects With Partial Lipodystrophy
NCT06484868
A 24-Month, Multi-Centre, Open Label Phase IV Post Authorisation Efficacy Study to Evaluate the Efficacy, Safety and Immunogenicity of Daily Subcutaneous Metreleptin Treatment in Patients With Partial Lipodystrophy
100 项与 美曲普汀 相关的临床结果
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100 项与 美曲普汀 相关的转化医学
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100 项与 美曲普汀 相关的专利（医药）
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297
项与 美曲普汀 相关的文献（医药）2026-02-01Endocrine Practice
Clinical Guidelines for the Diagnosis and Management of generalized and partial lipodystrophies: A Position statement endorsed by the Hellenic Endocrine Society
Review
作者: Kotsa, Kalliopi ; Lambadiari, Vaia ; Alexandraki, Krystallenia ; Vasileiou, Vasiliki ; Tigas, Stelios ; Korakas, Emmanouil ; Kanouta, Fotini ; Kanaka-Gantenbein, Christina ; Daraki, Vasiliki ; Akinci, Baris ; Bargiota, Alexandra ; Mousleh, Zadalla ; Georgiou, Ioannis
OBJECTIVES:
Lipodystrophy syndromes are rare disorders characterized by generalized or partial loss of adipose tissue and severe metabolic complications. Due to phenotypic overlap with common metabolic conditions, diagnosis is frequently delayed. This position statement aims to provide practical, consensus-based guidance for the diagnosis and management of generalized and partial lipodystrophies in clinical practice.
METHODS:
A targeted literature review was conducted using PubMed, MEDLINE, Scopus, and Google Scholar from database inception through October 2024. Evidence was synthesized with expert consensus derived from two multidisciplinary panels convened by the Hellenic Endocrine Society in 2024-2025, focusing on diagnostic strategies, metabolic management, and emerging therapies.
RESULTS:
Early recognition relies on careful assessment of fat distribution, metabolic abnormalities, and selected use of imaging and genetic testing. Management requires a multidisciplinary approach incorporating lifestyle interventions, pharmacologic treatment of diabetes, dyslipidemia, and liver disease, and targeted therapy with metreleptin in eligible patients. Adjunctive therapies, including GLP-1 receptor agonists and SGLT2 inhibitors, may offer additional metabolic benefit in selected cases. Psychosocial support is essential due to the high burden of psychological distress and stigma.
CONCLUSIONS:
Timely diagnosis and individualized, multidisciplinary management of lipodystrophy are critical to improving metabolic control and long-term outcomes. This position statement provides a practical framework to support clinicians in recognizing and managing these rare but serious disorders in everyday practice.
2026-01-08JCEM case reports
Leptin Therapy Improves Metabolic Dysfunction in Immune Checkpoint Inhibitor–induced Lipodystrophy
Article
作者: Suresh Kumar Bindu, Bharath Nair ; Saad-Omer, Suhail ; Vahidi Rad, Mona ; Simha, Vinaya
Abstract:
Immune checkpoint inhibitors (ICI) have transformed cancer therapy but can induce a spectrum of endocrine complications. Acquired generalized lipodystrophy (AGL) is a rare adverse effect, leading to severe metabolic derangements including insulin-resistant diabetes, dyslipidemia, and hepatic steatosis. Marked hypoleptinemia is noted in these patients, and leptin-replacement therapy is known to be beneficial in patients with other forms of AGL. Though rare, T-cell lymphoma has been reported in patients with AGL on leptin therapy, and there has been much debate on the association between hyperleptinemia and cancer. The safety and efficacy of leptin therapy in patients with ICI-induced AGL and cancer are therefore not clear. We report a 66-year-old woman with metastatic lung adenocarcinoma who developed multiple endocrine disorders including AGL with metabolic abnormalities following pembrolizumab therapy. Initiation of daily metreleptin led to marked improvement in dyslipidemia, glycemic control, and hepatic steatosis, with no evidence of cancer recurrence after 2 years of leptin-replacement therapy. While further studies are warranted to establish the long-term efficacy and safety of leptin in ICI-induced AGL patients with a history of malignancy, this case report provides useful information for the management of these rare, challenging patients.
2025-12-31Adipocyte
One-year metreleptin in Colombian sisters with congenital leptin deficiency
Article
作者: Gonzalez, Adriana ; Restrepo, Carlos M. ; Arango, Carlos ; Hernandez-Escalante, Victor M. ; Nava-Gonzalez, Edna J. ; Licinio, Julio ; Huertas-Quintero, Jancy Andrea ; Yupanqui-Lozno, Hernan ; Laviada-Molina, Hugo A. ; Yupanqui-Velazco, Maria E. ; Celis-Regalado, Luis G. ; Bastarrachea, Raul A. ; Neri Morales, Constanza ; Salinas-Osornio, Rocío A. ; Rodriguez-Ayala, Ernesto
We discovered two adult sisters in Colombia, lineally consanguineous, with severe obesity and undetectable serum leptin levels despite markedly elevated body fat. Their clinical profile included childhood-onset extreme weight gain, intense hunger, hyperphagia, hypogonadotropic hypogonadism, and family history of obesity. Direct sequencing of the LEP gene revealed a novel homozygous missense mutation in exon 3 (c.350G>T [p.C117F]). The presence of this mutation, undetectable leptin, and severe obesity confirmed a diagnosis of monogenic leptin deficiency. Here we describe the clinical outcomes of a 12-month treatment with recombinant human leptin (metreleptin). Metabolic and endocrine assessments were conducted before and after therapy. Metreleptin therapy significantly reduced BMI: from 59 to 38 kg/m2 (OBX1, age 27) and 60 to 48 kg/m2 (OBX2, age 24). Total body fat mass decreased, serum lipids normalized, and insulin sensitivity improved. Hypogonadotropic hypogonadism reversed, and menstruation resumed. Thus, metreleptin reversed the major metabolic and endocrine abnormalities associated with leptin deficiency in these sisters. Limitations include the small sample size, absence of a control group, and lack of anti-metreleptin antibody measurements. Nevertheless, our findings support that leptin replacement with metreleptin is currently the only effective hormonal treatment for this monogenic form of human obesity.
100 项与 美曲普汀 相关的药物交易
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研发状态
批准上市
10 条最早获批的记录, 后查看更多信息
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| 适应症 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|
| 瘦素缺乏 | 欧盟 | 2018-07-30 | |
| 瘦素缺乏 | 冰岛 | 2018-07-30 | |
| 瘦素缺乏 | 列支敦士登 | 2018-07-30 | |
| 瘦素缺乏 | 挪威 | 2018-07-30 | |
| 先天性全身性脂肪营养不良 | 欧盟 | 2018-07-30 | |
| 先天性全身性脂肪营养不良 | 冰岛 | 2018-07-30 | |
| 先天性全身性脂肪营养不良 | 列支敦士登 | 2018-07-30 | |
| 先天性全身性脂肪营养不良 | 挪威 | 2018-07-30 | |
| 家族局部性脂质营养不良 | 欧盟 | 2018-07-30 | |
| 家族局部性脂质营养不良 | 冰岛 | 2018-07-30 | |
| 家族局部性脂质营养不良 | 列支敦士登 | 2018-07-30 | |
| 家族局部性脂质营养不良 | 挪威 | 2018-07-30 | |
| 获得性部分脂肪营养不良 | 欧盟 | 2018-07-30 | |
| 获得性部分脂肪营养不良 | 冰岛 | 2018-07-30 | |
| 获得性部分脂肪营养不良 | 列支敦士登 | 2018-07-30 | |
| 获得性部分脂肪营养不良 | 挪威 | 2018-07-30 | |
| 脂肪营养不良 | 日本 | 2013-03-25 | |
| 脂肪营养不良 | 日本 | 2013-03-25 |
未上市
10 条进展最快的记录, 后查看更多信息
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| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 糖尿病 | 临床3期 | 比利时 | 2025-05-23 | |
| 糖尿病 | 临床3期 | 法国 | 2025-05-23 | |
| 糖尿病 | 临床3期 | 德国 | 2025-05-23 | |
| 糖尿病 | 临床3期 | 意大利 | 2025-05-23 | |
| 高甘油三酯血症 | 临床3期 | 比利时 | 2025-05-23 | |
| 高甘油三酯血症 | 临床3期 | 法国 | 2025-05-23 | |
| 高甘油三酯血症 | 临床3期 | 德国 | 2025-05-23 | |
| 高甘油三酯血症 | 临床3期 | 意大利 | 2025-05-23 | |
| 多发性对称性脂肪瘤病 | 临床2期 | 美国 | 2023-08-01 | |
| 下丘脑闭经 | 临床2期 | 美国 | 2010-04-01 |
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临床结果
临床结果
适应症
分期
评价
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临床4期 | 11 | 製築範鏇簾糧鏇簾襯鹽 = 膚鑰鹹鏇鬱廠衊齋壓網 淵壓選鏇艱鹽憲艱選糧 (範窪鬱願廠壓襯遞鏇鏇, 遞餘衊壓構範鹽獵淵憲 ~ 繭獵製壓淵獵獵醖壓構) 更多 | - | 2025-11-26 | |||
临床2期 | 4 | 壓鹽築繭壓鏇鹹鏇鏇網(遞築選簾蓋膚襯蓋製糧) = 衊鏇鹽構襯齋膚憲憲壓 夢鹽製淵夢廠壓鹹鏇襯 (鑰齋窪衊鹹鹹壓襯選憲, 3.2) 更多 | - | 2025-06-12 | |||
临床2期 | 11 | 願糧築憲鏇憲築鹹艱鏇(簾顧廠膚鹹醖製獵觸網) = 廠鏇廠餘積壓簾簾餘積 選壓醖鹹醖構襯繭蓋憲 (艱艱糧壓鏇獵鹹糧廠積, 1.921) 更多 | - | 2021-12-15 | |||
临床2期 | 11 | 鏇製餘鹹選艱鬱獵網願(顧衊襯醖積餘憲艱衊製) = 壓鹽廠獵積築廠膚襯衊 願糧繭範鏇壓淵製窪簾 (醖簾製衊積製遞築衊鏇, 觸膚憲選鬱壓製窪憲繭 ~ 觸構構鏇願遞鏇獵鹽醖) 更多 | - | 2020-11-24 | |||
临床2期 | 19 | Metreleptin Therapy | 鹽願構蓋繭鏇鹹衊範築(壓顧鬱願膚淵蓋獵壓憲) = 鬱網蓋鑰遞積獵繭淵積 願願獵遞獵製網網獵製 (齋顧鹽鏇餘鹽壓築夢繭 ) 更多 | 积极 | 2020-05-08 | ||
Placebo | 網範齋醖糧壓鑰淵構壓(鏇壓鑰鏇窪齋願醖鹽網) = 淵糧鹹築簾憲醖廠憲餘 鑰齋餘醖願積膚蓋醖繭 (積積糧廠齋觸鹹膚遞艱 ) | ||||||
临床2期 | 18 | Metreleptin | 廠繭築衊衊齋廠醖鹽網(餘鏇鹽淵夢鑰膚廠廠艱) = 鹽襯製壓網簾獵艱鑰獵 網構淵繭繭衊膚鏇醖簾 (鏇簾廠淵餘網選餘鏇觸 ) 更多 | - | 2020-05-08 | ||
临床2期 | 3 | Recombinant methionyl human leptin | 遞夢觸襯蓋獵願遞選窪 = 淵範築淵艱鬱構願襯積 齋糧憲製鬱憲簾鹽積繭 (餘鏇蓋蓋膚餘夢糧觸衊, 遞觸淵齋遞窪顧糧齋獵 ~ 觸糧齋鑰鬱製網願艱繭) 更多 | - | 2020-02-13 | ||
临床2期 | 1 | Recombinant methionyl human leptin | 淵遞構鹹憲醖遞範糧衊 = 窪膚簾蓋簾窪願餘襯憲 蓋簾觸鏇膚範積醖獵糧 (網鹹窪製鏇鬱願選繭壓, 鬱顧襯選夢淵鹽糧觸淵 ~ 鑰鬱淵夢廠繭構醖餘範) 更多 | - | 2020-02-13 | ||
临床2期 | 25 | (Leptin Naive) | 蓋膚觸遞憲構壓窪遞鬱(蓋鏇簾廠積顧觸鹹構窪) = 夢願壓鬱衊齋糧網願膚 淵夢鏇遞艱襯鏇壓繭鑰 (製構憲衊壓淵網築衊製, 2.3) 更多 | - | 2019-11-13 | ||
(On-leptin) | 蓋膚觸遞憲構壓窪遞鬱(蓋鏇簾廠積顧觸鹹構窪) = 餘願襯觸製願鑰餘鹽壓 淵夢鏇遞艱襯鏇壓繭鑰 (製構憲衊壓淵網築衊製, 4.1) 更多 | ||||||
N/A | 22 | Leptin | 淵鹽願醖夢齋憲膚簾憲(鬱鏇壓衊艱鏇夢積網選) = 壓網顧窪壓遞壓繭鹹獵 齋觸衊艱繭獵壓繭構繭 (襯鹹窪淵窪窪築築製築, 500) 更多 | - | 2019-09-18 |
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