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更新于：2026-09-08

Metreleptin

美曲普汀

更新于：2026-09-08

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

LEPR

作用方式

激动剂

作用机制

LEPR激动剂(瘦素受体激动剂)

治疗领域

神经系统疾病遗传病与畸形内分泌与代谢疾病+ [2]

在研适应症

瘦素缺乏先天性全身性脂肪营养不良家族局部性脂质营养不良+ [3]

非在研适应症

糖尿病1型糖尿病2型糖尿病+ [7]

原研机构

The Rockefeller University

在研机构

CHIESI Farmaceutici SpAChiesi Canada Corp.

Shionogi Pharma, Inc.

+ [3]

非在研机构

Amgen, Inc.

Amylin Pharmaceuticals, Inc.

AstraZeneca PLC

权益机构

Medison Pharma Ltd.

Amylin Pharmaceuticals, Inc.

AstraZeneca PLC

+ [6]

最高研发阶段批准上市

首次获批日期

日本 (2013-03-25),

脂肪营养不良

最高研发阶段(中国)-

特殊审评快速通道 (美国)、孤儿药 (美国)、孤儿药 (欧盟)、孤儿药 (日本)、孤儿药 (澳大利亚)

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结构/序列

Sequence Code 59859

来源: *****

关联

项与美曲普汀相关的临床试验

NCT06502990

/ Terminated临床3期

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

This is an 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

开始日期2025-05-23

申办/合作机构

Amryt Pharma Holdings Ltd.

NCT06679270

/ Recruiting临床3期

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

This Phase 3 study is an Open Label Extension of the APG-20 Study To Evaluate the Long-term Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects with Partial Lipodystrophy

开始日期2024-10-14

申办/合作机构

CHIESI Farmaceutici SpA

NCT06484868

/ Recruiting临床4期

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

This is an Open Label, Phase IV, Post Authorisation Study to Evaluate the Efficacy, Safety and Immunogenicity of Daily Subcutaneous Metreleptin Treatment in people with Partial Lipodystrophy

开始日期2024-02-08

申办/合作机构

Amryt Pharma Holdings Ltd.

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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304

项与美曲普汀相关的文献（医药）

2026-07-01·DIABETES CARE

Comment on Korakas et al. 780G Hybrid Closed-Loop Pump as Adjunct to Metreleptin in Lipodystrophy-Associated Diabetes: Three Case Observations

Letter

作者: Lamothe, Sophie ; Christin-Maitre, Sophie ; Vatier, Camille ; Bourcigaux, Nathalie ; Vigouroux, Corinne ; Donadille, Bruno

2026-06-01·Endocrine 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

OBJECTIVE:

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 2 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 glucagon-like peptide-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.

CONCLUSION:

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.

2026-05-04·Expert Opinion On Drug Safety

Adverse drug events associated with metreleptin administration: a real-world pharmacovigilance study from 2014 to 2024 using the FAERS database

Article

作者: Luo, Xuehu ; He, Jinhao ; Zhang, Chenning ; Sun, Mo ; Liu, Jie

BACKGROUND:

This study analyzed adverse drug event (ADE) signals associated with metreleptin using the FDA Adverse Event Reporting System (FAERS) to provide insights for safe clinical use.

RESEARCH DESIGN AND METHODS:

Data from 1 January 2014, to 31 March 2024, were extracted. Signal intensity for adverse events was assessed using reporting odds ratio (ROR), Medicines and Healthcare Products Regulatory Agency (MHRA), Bayesian Confidence Propagation Neural Network (BCPNN), and Multi-item Gamma Poisson Shrinker (MGPS) methods. Time-to-onset (TTO) correlations were evaluated using the Weibull shape parameter (WSP). Logistic regression identified potential hospitalization risk factors.

RESULTS:

4,000 ADE reports were identified, with 1,025 significant events mainly involving females (66.73%) and reported by healthcare professionals (67.80%), primarily from the United States (84.10%). 101 preferred term (PT) signals were confirmed by all methods, with 57 PTs not listed on the drug label. Overall TTO was 547 days (IQR: 113-1325), with gastrointestinal disorders and investigations as hospitalization risk factors.

CONCLUSIONS:

This study provides critical insights into the TTO of ADEs related to metreleptin, informing safe clinical application and emphasizing the importance of monitoring potential side effects.

项与美曲普汀相关的新闻（医药）

2026-08-11

·EINPresswire

USPTO Issues Foundational Patent for Leptin Replacement in Anorexia Nervosa, Exclusively Licensed to Zula Therapeutics

Method-of-use patent gives Zula an exclusive position in leptin replacement for anorexia nervosa We are grateful to the patients, institutions, and funders whose support has allowed us to demonstrate the novelty, symptom improvement, and safety of leptin as a treatment for anorexia thus far.” — Professor Johannes Hebebrand, University of Duisburg-Essen, co-inventor BOSTON, MA, UNITED STATES, August 11, 2026 / EINPresswire.com / -- Zula Therapeutics, Inc. today announced that the United States Patent and Trademark Office issued U.S. Patent No. 12,651,658 , titled "Methods for Treating Anorexia Nervosa and Related Conditions by Administering a Leptin Receptor Agonist." Zula holds exclusive rights under its license with the University of Duisburg-Essen (UDE). The patent is expected to provide exclusive protection for leptin-based treatment of anorexia nervosa through 2045. Protection extends beyond metreleptin, the drug tested clinically to date, to any leptin receptor agonist. "This patent gives Zula something rare for a company at our stage: a clear, exclusive foundation for the medicines we intend to build. It protects the use of leptin replacement in anorexia nervosa well into the next decade. With this foundation in place, we are now equipped to pursue the partnerships required to develop the first FDA approved medicine for anorexia nervosa," said Dawn Bardot, PhD, Chief Executive Officer of Zula Therapeutics. SCIENTIFIC AND CLINICAL FOUNDATION The patented approach reflects the foundational research of Professor Johannes Hebebrand and his co-inventors, who identified leptin deficiency as an active driver of anorexia nervosa rather than a consequence of starvation. According to his construct of weight loss-induced entrapment, weight loss itself triggers anorexia via a loss of adipose tissue mass entailing sub-physiological circulating leptin levels. The therapeutic rationale is actively being tested clinically: Dr. Gabriella Milos of University Hospital Zurich, a co-inventor on the patent, is leading an investigator-initiated randomized controlled trial of metreleptin in patients with anorexia nervosa (ClinicalTrials.gov NCT06305182), initiated in 2024 and scheduled to complete enrollment in 2026. "We are grateful to the patients, institutions, and funders whose support has allowed us to demonstrate the novelty, symptom improvement, and safety of leptin as a treatment for anorexia thus far. Working with Zula by licensing an issued patent gives us, as inventors and clinicians, the opportunity to translate our work into a therapy widely adopted to benefit patients globally," said Professor Johannes Hebebrand, University of Duisburg-Essen, co-inventor. Metreleptin is not approved by the FDA for the treatment of anorexia nervosa. CLINICAL COLLABORATION WITH MOUNT SINAI Zula has entered into a memorandum of understanding with the Center of Excellence in Eating and Weight Disorders at the Icahn School of Medicine at Mount Sinai, led by Thomas Hildebrandt, PsyD, Professor of Psychiatry, to collaborate on the design and execution of clinical studies of leptin replacement in severe anorexia nervosa. The Mount Sinai team is leading a philanthropic fundraising campaign in support of this work, detailed in the Center's Case for Support . "Having reviewed the case-series evidence and considering our research into leptin as a biomarker for recovery, I believe this science must be tested in a rigorous process designed to achieve an FDA regulatory approval. Mount Sinai has a track record of helping to develop breakthrough psychiatric drugs, and we look forward to working with Zula to conduct the first clinical trials designed to support FDA submission here in New York," said Hildebrandt. A PATIENT-ADVOCACY PERSPECTIVE Patient-advocacy leaders have welcomed renewed scientific attention to the biological drivers of eating disorders. "At NEDA, we educate the public on eating disorders and their biological basis. Despite the severity and prevalence of anorexia nervosa specifically, there are still far too few effective treatments. This research represents the kind of scientific breakthrough our community needs, and we hope this newly issued patent helps investors and philanthropists finance the clinical trials required to turn that research into a medicine for our community," said Jessica Scheer, Chief Executive Officer, National Eating Disorders Association (NEDA). ABOUT ZULA THERAPEUTICS Zula Therapeutics is developing the first medicine for anorexia nervosa, the deadliest psychiatric illness and one that still has no approved drug. Headquartered in Boston, Zula is incorporated as a Delaware public benefit corporation. The company maintains a collaborative relationship with Courage Therapeutics, which holds an equity stake in Zula. Additional information is available at www.zulatx.com . Forward-Looking Statements: This press release contains forward-looking statements involving known and unknown risks and uncertainties. Actual results may differ materially from those anticipated. Dawn Bardot Zula Therapeutics, Inc. press@zulatx.com Visit us on social media: LinkedIn Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

2026-07-24

·中草药杂志社

严重高三酰甘油血症新型治疗药物的研究进展

高三酰甘油血症是临床常见的血脂紊乱类型，不仅与肥胖、糖尿病等代谢性疾病相关，而且还会严重引起心、肝、肾、胰腺等脏器功能紊乱[1]。轻中度高三酰甘油血症易引起动脉粥样硬化性心血管疾病，严重高三酰甘油血症与急性胰腺炎发病风险升高密切相关[2]。依据国内外指南，严重高三酰甘油血症以三酰甘油（TG）≥5.65 mmol/L（或500 mg/dL）为标准[1-3]。严重高三酰甘油血症发病机制涉及遗传易感性、继发性因素[2]，根据其遗传特征和发病机制可主要分为3大亚型：家族性乳糜微粒血症综合征、多因素乳糜微粒血症综合征和家族性部分脂肪营养不良[4]。当前严重高三酰甘油血症的治疗面临诸多挑战，极低脂肪饮食依从性差，传统药物的疗效有限，虽然血浆置换、胰岛素和肝素应用等特殊干预措施能快速降低血清TG，但却无法降低急性胰腺炎的发生风险、重症患者死亡率或缩短器官衰竭持续时间。此外，不同亚型严重高三酰甘油血症致病机制的差异较大，治疗方案大相径庭。基于传统疗法疗效有限这一背景，许多新型治疗药物应运而生。载脂蛋白C3（APOC3）抑制剂、血管生成素样蛋白家族（ANGPTL）抑制剂、成纤维细胞生长因子21（FGF21）类似物、胰高血糖素样肽-1受体激动剂（GLP-1RA）、微粒体三酰甘油转移蛋白（MTP）抑制剂、人瘦素类似物、法尼醇X受体（FXR）激动剂在临床上广泛用于严重高三酰甘油血症的治疗。本文总结了严重高三酰甘油血症新型治疗药物的研究进展，为临床个体化治疗方案提供参考依据。 1 APOC3抑制剂 APOC3主要在肝脏和肠道中合成，与富含三酰甘油脂蛋白（TRL）具有高亲和力，可通过依赖脂蛋白脂肪酶（LPL）介导途径和非LPL介导途径来抑制血清TG清除[5]。APOC3一方面促进肝脏合成和分泌极低密度脂蛋白胆固醇（VLDL-C），另一方面抑制LPL活性，干扰TRL分解和清除，使TG升高。目前针对APOC3研制出多种药物，如反义寡核苷酸（ASO）药物（沃兰奈生、奥莱扎尔森等）、小干扰RNA药物（普乐司兰钠等）、单克隆抗体等。 1.1 沃兰奈生（volanesorsen）沃兰奈生是第2代ASO，通过抑制APOC3表达来降低TG水平，需每周或每2周ip 1次。针对3例LPL完全缺陷的家族性乳糜微粒血症综合征患者的Ⅱ期临床试验结果发现，3例受试者APOC3降低了71%～90%、TG降低56%～86%，而LPL活性未见明显变化，这表明沃兰奈生可通过非LPL依赖途径发挥作用[6]。APPROACH试验是针对66例家族性乳糜微粒血症综合征患者的Ⅲ期临床研究，治疗3个月时，沃兰奈生组APOC3降低84%，TG降低77%，低密度脂蛋白胆固醇（LDL-C）升高136%，非高密度脂蛋白胆固醇（HDL-C）降低45%，且急性胰腺炎发生率亦显著下降，这表明沃兰奈生可能降低急性胰腺炎发生风险[7]。COMPASS试验是针对114例多因素乳糜微粒血症综合征患者的Ⅲ期临床研究，治疗3个月时，沃兰奈生组APOC3降低76.1%，TG降低71.2%，LDL-C升高95.5%，非HDL-C降低27.3%，且在本试验中亦发现沃兰奈生可降低急性胰腺炎发生率[8]。BROADEN试验是针对家族性部分脂肪营养不良患者的Ⅱ/Ⅲ期临床研究，治疗3个月时，沃兰奈生组TG降低88%，治疗12个月时，肝脏脂肪分数（HFF）显著下降了53%，LDL-C升高95%，非HDL-C降低27%[9]。综上所述，沃兰奈生能显著降低不同亚型严重高三酰甘油血症患者血清TG水平，且家族性部分脂肪营养不良患者疗效最显著，这表明沃兰奈生可能同时作用于依赖LPL介导途径和非LPL介导途径发挥降TG效应。有趣的是，在应用沃兰奈生治疗时，不同亚型严重高三酰甘油血症患者均出现LDL-C升高，非HDL-C降低，这可能是由于沃兰奈生抑制APOC3表达，使TRL分解为IDL-C，进而转化为LDL-C，故表现为LDL-C升高；非HDL-C被认为是更全面的心血管风险指标。非HDL-C降低表明其不仅降低了TRL水平，还降低了整体心血管风险，因此，在应用沃兰奈生治疗时，应优先关注非HDL-C，若LDL-C持续升高，可联用他汀类药物[8]。沃兰奈生主要不良反应为血小板减少和注射部位反应。通过对其他2'-O-甲氧基乙基ASO类药物研究发现，血小板减少是沃兰奈生特有的不良反应，并非ASO药物的共性风险。值得注意的是，血小板减少在停药后可能是可逆的，且呈剂量相关[7]。因此，在应用沃兰奈生时，需严格筛选患者，并密切监测血小板水平和出血倾向。沃兰奈生在2019年获得欧洲药品管理局（EMA）的批准，用于基因确诊的、急性胰腺炎高风险的且饮食降TG疗效不佳的家族性乳糜微粒血症综合征成年患者的饮食辅助药物[10]，而美国食品药品监督管理局（FDA）因其血小板减少风险暂未批准。在《2025 ESC/EAS血脂异常管理指南（更新版）》中，建议基因确诊的家族性乳糜微粒血症综合征患者TG＞750 mg/dL（或8.5 mmol/L）时可考虑ip沃兰奈生300 mg/周，以降低TG、急性胰腺炎发生率[11]。未来尚需开展大规模长期试验、真实世界研究分析其长期疗效、剂量需求、不良反应预防措施等，为临床应用提供精准指导。 1.2 奥莱扎尔森（olezarsen）奥莱扎尔森是第3代N-乙酰半乳糖胺（GalNAc）偶联的ASO，通过结合肝细胞表面去唾液酸糖蛋白受体增强其靶向性，从而减少药物使用剂量，仅需每月皮下注射1次。BALANCE试验是针对66例家族性乳糜微粒血症综合征患者的Ⅲ期临床研究，奥莱扎尔森不同剂量均表现出显著疗效，治疗6个月时，奥莱扎尔森80 mg组APOC3降低73.7%，TG降低43.5%，且急性胰腺炎发生率显著降低88%[12]。为了探索奥莱扎尔森长期疗效和安全性，针对既往接受沃兰奈生治疗的家族性乳糜微粒血症综合征患者进行了Ⅲ期临床研究，在至少6周洗脱期后给予奥莱扎尔森80 mg，结果发现，奥莱扎尔森治疗期间未出现严重不良事件，如肝功能衰竭、肾功能损伤、严重出血和血小板小于50 000个/μL等[13]。由此可见，奥莱扎尔森具有更优的疗效、安全性和潜在高依从性。为了验证奥莱扎尔森在更大人群中的长期疗效，两项针对严重高三酰甘油血症患者在不同地区的Ⅲ期试验CORE-TIMI 72a和CORE2-TIMI 72b为奥莱扎尔森应用提供了更充分的证据[14]。奥莱扎尔森已于2024年12月被FDA批准用于无需基因确诊的成人家族性乳糜微粒血症综合征的饮食辅助手段[15]。 1.3 普乐司兰钠（plozasiran sodium）普乐司兰钠是首个靶向APOC3的siRNA药物，在肝细胞中通过与RNA诱导沉默复合体结合来抑制APOC3表达，仅需每3个月sc 1次。SHASTA-2试验是针对严重高三酰甘油血症患者的Ⅱb期临床研究，治疗24周时，普乐司兰钠50 mg组APOC3降低77%、TG降低57%，其中90.6%患者TG水平可降至急性胰腺炎发生阈值（5.65 mmol/L）以下，急性胰腺炎发生风险显著降低[16]。普乐司兰钠未见明显的血小板减少不良反应，其常见的不良事件为血糖控制不佳（仅基线合并糖尿病患者）、腹泻、尿路感染、头痛等。值得注意的是，1例65岁男性在使用普乐司兰钠治疗后TG仍偏高，经检测发现该患者合并甘油激酶缺乏症，其游离甘油高达40.24 mg/dL，表现出假性高三酰甘油血症。在排除游离甘油干扰后发现该患者仅为轻度高三酰甘油血症，且普乐司兰钠使其TG水平显著降低71%，但对游离的甘油无显著影响[17]。案例提示普乐司兰钠治疗无效的患者需及时检测游离甘油以排除甘油激酶缺乏症，避免误诊和无效治疗。 PALISADE试验是针对持续性乳糜微粒血症患者的Ⅲ期临床试验，治疗10个月时，普乐司兰钠 25、50 mg组APOC3降低93%～96%、TG降低80%～78%，且治疗12个月时，观察到普乐司兰钠合并组急性胰腺炎风险显著降低83%。经研究人员进行持续性乳糜微粒血症患者亚组分析，发现无论是否基因确诊为家族性乳糜微粒血症综合征，普乐司兰钠的疗效均无显著差异[18]。由此可见，普乐司兰钠是首个被证实可降低持续性乳糜微粒患者急性胰腺炎发生风险的药物，且其避免了沃兰奈生的血小板减少风险，具有高效安全的优势。普乐司兰钠已于2025年11月获得FDA批准作为饮食的辅助治疗手段，用于治疗家族性乳糜微粒血症综合征成人患者，成为首个治疗家族性乳糜微粒血症综合征的siRNA药物。2026年1月5日国家药品监督管理局正式批准普乐司兰钠注射液用于家族性乳糜微粒血症综合征成人患者，这一重要进展为我国家族性乳糜微粒血症综合征患者提供有效管理措施，改善了患者预后和生活质量。普乐司兰钠未来仍需开展更多临床研究来观察其长期疗效和安全性（NCT06822790），并在不同地区、不同亚型严重高三酰甘油血症人群中观察疗效，以扩大其适用人群。 1.4 RN0361 RN0361是一种靶向APOC3的siRNA，仅需每6个月sc 1次。2025年11月大睿生物公司在美国心脏协会年会上口头报道，Ⅰ期临床试验结果示，RN0361能显著降低血清APOC3、TG水平最高可分别达93%、69%，且疗效维持时间可长达6个月，与此同时，RN0361显著降低了非HDL-C、VLDL-C、残余胆固醇等指标，且其空腹血糖、糖化血红蛋白（HbA1c）未出现明显升高，结果表明RN0361可能比普乐司兰钠更安全、更高效。针对高三酰甘油血症和家族性乳糜微粒血症综合征患者的Ⅱ期临床试验（NCT06471543）正在进行，将为RN0361的疗效和安全性提供更充分的证据。 2 ANGPTL抑制剂 ANGPTL3、ANGPTL4和ANGPTL8在脂代谢过程中发挥着重要作用。ANGPTL3主要在肝脏表达，其分泌过程不受进食状态影响，可通过抑制LPL和内皮脂肪酶（EL）活性使血清TG水平升高。值得注意的是，单独存在的ANGPTL3对LPL的抑制作用较弱，但与ANGPTL8结合后可显著增强其LPL抑制活性，增幅超过100倍。ANGPTL4主要在白色脂肪组织和肝脏中表达，在禁食状态下大量分泌。与ANGPTL3不同，单独存在的ANGPT4具有强效的LPL抑制活性，但与ANGPTL8结合后可显著降低其LPL抑制活性，降幅超100倍。ANGPTL8主要在肝脏、脂肪组织中表达，在进餐后分泌显著增多，其自身无法直接抑制LPL活性，主要与ANGPTL3或ANGPTL4结合，从而在不同营养状态下调控LPL活性的动态平衡。由此可见，通过抑制ANGPTL3、ANGPTL8、ANGPTL4可恢复LPL活性，从而降低TG。目前研究较多的主要是ANGPTL3抑制剂，主要有ASO、单克隆抗体、siRNA等形式。 2.1 武帕诺森（vupanorsen）武帕诺森是一种靶向ANGPTL3的GalNAc偶联的ASO，需每周皮下注射1次。针对3例家族性乳糜微粒血症综合征患者的Ⅱ期临床试验结果显示，治疗3个月时，受试者ANGPTL3降低51.3%，TG降低32.8%，治疗期间未出现严重不良事件和血小板减少事件[19]。针对4例家族性部分脂肪营养不良患者的Ⅱ期临床试验结果显示，治疗27周时，受试者ANGPTL3降低55%，TG降低59.9%，且APOC3降低51%，但HFF降低仅1.14%[20]。在随后在接受他汀类药物治疗的非HDL-C和TG升高的人群中进行武帕诺森剂量范围研究，结果发现武帕诺森治疗24周后TG降低41.3%～56.8%，ANGPTL3降低69.9%～95.2%，但同时出现了HFF剂量相关增加高达76%，肝酶显著升高（＞3×ULN）、注射部位反应在较高剂量下发生率更高[21]。通过ANGPTL3基因敲除小鼠验证发现并无HFF升高现象，说明武帕诺森导致HFF升高的原因可能是药物靶向肝细胞过程中出现脱靶现象，而非抑制ANGPTL3所导致。这启示未来研发严重高三酰甘油血症治疗药物应优先开发作用机制更精准、脱靶风险更低的药物，并且在后续临床试验中严密监测肝脏脂质沉积和肝酶。最终，武帕诺森因其肝毒性于2022年终止开发。 2.2 依维苏单抗（evinacumab）依维苏单抗是一种全人源单克隆抗体，通过抑制ANGPTL3恢复LPL和EL活性来降低血清TG和LDL-C，需每4周静脉注射1次。针对既往患急性胰腺炎的严重高三酰甘油血症患者的Ⅱ期临床试验，根据基因型分为3个队列，分别为LPL双等位基因功能缺失突变的家族性乳糜微粒血症综合征患者、LPL通路杂合缺失突变的多因素乳糜微粒血症综合征患者、无LPL通路基因突变的多因素乳糜微粒血症综合征患者，给药12周后，3个队列的TG分别显著下降27.7%、64.8%、81.7%，非HDL-C均显著降低，LDL-C升高，但无统计学意义，且未观察到肝酶显著升高和HFF升高的药物不良反应；研究期间急性胰腺炎主要发生在TG＞1 000 mg/dL患者中，且80%以上发生在末次给药超过4周后或停药后[22]。这个研究结果表明，依维苏单抗可能对LPL双等位基因功能缺失突变的家族性乳糜微粒血症综合征患者无效，对LPL通路基因杂合缺失突变、无缺失突变的多因素乳糜微粒血症综合征患者均有显著降TG疗效，且肝脏安全性良好，但在使用期间应规律给药，并严密监测TG。为了研究依维苏单抗能否预防严重高三酰甘油血症相关急性胰腺炎，针对排除LPL双等位基因功能缺失突变的家族性乳糜微粒血症综合征患者的既往患急性胰腺炎的严重高三酰甘油血症患者进行了Ⅱb期临床试验，虽因研究招募缓慢致样本量过小提前终止而无法确定依维苏单抗能否预防急性胰腺炎，但仍观察到依维苏单抗能快速有效降低TG水平[23]。这一研究期间急性胰腺炎主要发生在依维苏单抗药物浓度低于治疗阈值、TG＞500 mg/dL时，表明依维苏单抗需要维持足够的需要浓度才能发挥起降TG疗效，而其预防急性胰腺炎的疗效仍需扩大样本量、长期给药延、长随访时间进一步研究。依维苏单抗在欧盟（≥6月龄）、美国（≥5岁）、加拿大（≥5岁）、英国（≥12岁）和日本（所有年龄段）已被批准用于纯合子家族性高胆固醇血症（HoFH）的治疗。此外，再生元制药公司在Clinical Trials.gov提交了针对严重高三酰甘油血症、小儿HoFH、异常β脂蛋白血症患者的依维苏单抗同情用药试验（NCT06500598），这一研究将为依维苏单抗在严重高三酰甘油血症人群应用中的疗效和安全性提供更多证据。综上可见，依维苏单抗可能通过抑制ANGPTL3恢复LPL活性来发挥其降TG的疗效，在临床应用时应规律给药，需维持一定的血药浓度，并密切监测TG，必要时可联用其他降脂药物，避免因药物浓度低于阈值或TG水平过高诱发急性胰腺炎。 2.3 佐达西兰（zodasiran）、索尔宾西兰（solbinsiran）目前靶向ANGPTL3的siRNA药物（如佐达西兰、索尔宾西兰）尚无严重高三酰甘油血症相关研究，但其临床前研究或其他类型的血脂异常研究中仍展现出降TG疗效。ARCHES-2试验是针对混合性高脂血症的Ⅱb期临床研究，结果显示，佐达西兰（每3个月皮下注射1次）治疗24周后，TG呈剂量相关降低，200 mg组可高达63%，且肝脏安全性良好[24]。索尔宾西兰目前主要是在混合性高脂血症患者中进行研究，结果显示，索尔宾西兰400 mg（每3个月皮下注射1次）具有持久降脂疗效和良好安全性，其中TG下降50.3%、HFF显著下降27.6%[25]。综上所述，靶向ANGPTL3的siRNA药物虽无针对严重高三酰甘油血症患者的临床研究，但仍具有降TG疗效，未来可针对不同亚型的严重高三酰甘油血症患者开展临床试验进一步观察其疗效。 3 FGF21类似物 FGF21属于成纤维细胞生长因子家族FGF19成员，通过激活腺苷酸活化蛋白激酶（AMPK）通路促进脂质代谢，降低TG水平。pegozafermin是一种FGF21类似物，目前研究证实其在严重高三酰甘油血症和代谢相关脂肪性肝病人群中有显著疗效。ENTRIGUE试验是针对严重高三酰甘油血症患者的Ⅱ期临床研究，结果发现，治疗8周后，pegozafermin组TG降低43.7%，其中79.7%的患者血清TG下降至急性胰腺炎发生阈值以下，且HFF降低42.2%[26]。这表明pegozafermin不仅能有效降低TG，还可能降低急性胰腺炎发病率。未来仍需针对不同严重高三酰甘油血症患者亚型开展更大规模的临床试验、更长的随访期进一步验证其疗效和长期安全性。 4 GLP-1RA GLP-1RA可通过与全身多器官或组织的GLP-1R结合来发挥其降糖、调脂、减轻体质量、心血管保护等作用。 4.1 司美格鲁肽司美格鲁肽是一种长效GLP-1RA，需每周皮下注射1次。针对家族性部分脂肪营养不良患者的临床研究发现，司美格鲁肽可使TG显著降低16.7%，HbA1c显著下降0.5%，发生急性胰腺炎的2例受试者既往均有急性胰腺炎发作病史[27]。基于法国全国罕见病参考网络的针对家族性部分脂肪营养不良患者的真实世界回顾性观察研究结果发现，司美格鲁肽治疗12个月后，TG显著降低14.7%，HbA1c显著下降1.0%，身体质量指数（BMI）下降0.44 kg/m2[28]。由此可知，司美格鲁肽对家族性部分脂肪营养不良患者具有一定疗效，但证据尚不充分，对于合并有急性胰腺炎病史的严重高三酰甘油血症患者须谨慎使用。 4.2 替尔泊肽替尔泊肽是一种抑胃肽/胰高血糖素样肽-1受体（GIP/GLP-1R）双激动剂，需每周皮下注射1次。在脂肪营养不良患者中应用替尔泊肽的观察性队列研究，结果发现，在随访结束时，家族性部分脂肪营养不良患者TG、BMI、HbA1c均显著降低。此外，研究人员通过对比替尔泊肽、美曲普汀、GLP-1RA在部分脂肪营养不良患者中的疗效，结果发现替尔泊肽对脂肪营养不良人群的疗效相对更强。值得注意的是，这一研究中1例患者因供应链短缺停用替尔泊肽导致急性胰腺炎复发，恢复给药后TG显著降低，这提示对于合并严重高三酰甘油血症的脂肪营养不良患者，在应用替尔泊肽时应逐步减量，避免引起急性胰腺炎复发[29]。以上两项研究均属于观察性研究，参与者异质性较大，故替尔泊肽在家族性部分脂肪营养不良患者中的疗效仍需进一步在大型随机对照试验中进行验证。此外，1例42岁因LPL基因杂合变异（c.710G＞Ap.Gly237 Asp）所致复发性高三酰甘油血症性急性胰腺炎的白人女性患者，在应用GLP-1RA治疗19个月后，其TG从3 565 mg/dL降至404 mg/dL，且期间未出现急性胰腺炎复发[30]，这表明GLP-1RA在严重高三酰甘油血症合并急性胰腺炎患者中具有长期疗效和安全性。DR10624是一种GLP-1R/胰高血糖素受体（GCGR）/FGF21R三激动剂，目前针对严重高三酰甘油血症患者的Ⅱ期临床试验正在进行（NCT06555640），其结果将为DR10624的应用提供重要证据。综上所述，GLP-1RA在合并急性胰腺炎的多因素乳糜微粒血症综合征患者、家族性部分脂肪营养不良患者中具有降低TG疗效作用。值得注意的是，在GLP-1RA应用过程中应避免停药，否则可能诱发急性胰腺炎。但GLP-1RA、多重靶点激动剂在严重高三酰甘油血症人群中的疗效证据均不够充足，未来需要针对不同严重高三酰甘油血症亚型患者开展大型的随机对照临床试验进行临床疗效和安全性的验证。 5 MTP抑制剂 MTP主要在肝脏和小肠表达，负责转运TG合成脂蛋白胆固醇（VLDL）和乳糜微粒，在脂质代谢中发挥重要作用。洛美他派（lomitapide）是一种MTP抑制剂，通过减少VLDL和乳糜微粒的合成进而从源头降低TG水平，而不依赖于LPL发挥作用。目前洛美他派的临床研究主要聚焦于HoFH患者，但值得注意的是，目前有多项研究报道其在家族性乳糜微粒血症综合征患者中亦有显著疗效。 LOCHNES试验是首个洛美他派针对18例基因确诊的家族性乳糜微粒血症综合征患者的Ⅲ期临床研究，结果发现，治疗26周后，TG较基线降低70.5%，其中55.6%的受试者TG下降至急性胰腺炎发生阈值以下，且在治疗期间无急性胰腺炎发作。然而，在LOCHNES试验中观察到洛美他派治疗期间有4例患者出现肝酶显著升高（＞3×ULN），HFF也较基线升高20.5%，但肝硬度、纤维化评分无明显变化[31]。针对乳糜微粒血症患者的意大利LIPIGEN注册库真实世界数据分析进一步验证了洛美他派临床治疗价值，结果显示，在接受洛美他派治疗的家族性乳糜微粒血症综合征患者中，治疗12个月后，TG较基线降低62%，且90%患者未发生急性胰腺炎[32]。此外，另有长期随访的病例报道弥补了上述研究的短期试验结果的局限性。1例LPL基因纯合突变的44岁家族性乳糜微粒血症综合征华裔女性患者在经同情用药批准启动洛美他派治疗13年后，其TG降低87%，且慢性腹痛、急性胰腺炎发生率得到有效控制。但值得注意的是，其脂肪肝进展为脂肪性肝炎、3～4期肝纤维化[33]。该病例首次证实洛美他派对家族性乳糜微粒血症综合征长期疗效，同时发现其潜在长期肝毒性风险。另一例46岁家族性乳糜微粒血症综合征男性患者经超说明书同情用药批准后启动洛美他派治疗26个月后，TG较基线降低67%，且治疗期间未再发生慢性腹痛、急性胰腺炎，但肝脂肪变性进展为显著弥漫性大泡性脂肪变，且伴轻度静脉周围、窦周纤维化[34]。基于既往研究，洛美他派肝损伤风险应予以重视，但或许并非不可逆转。1例71岁既往曾接受沃兰奈生治疗不耐受的家族性乳糜微粒血症综合征患者，在LOCHNES试验中接受洛美他派治疗，其TG显著降低90.8%。在试验结束后，经同情用药批准继续应用洛美他派治疗约4年，其TG维持在3.64 mmol/L左右，且未再发生急性胰腺炎。在治疗期间，该患者也出现了肝酶显著升高、进行性肝脂肪变性、肝硬度增加等不良反应，但在停药70 d后脂肪肝完全消退，且肝硬度、肝酶均有所改善。然而，在停药后其TG亦显著回升至2 000 mg/dL，并伴有胰腺损伤[35]。综上所述，洛美他派在降TG、降低急性胰腺炎和慢性腹痛发生率方面具有显著疗效。然而，其肝毒性不容忽视，虽在停药后或可自行消退，但治疗中断所伴随的TG迅速回升可能诱发急性胰腺炎，在临床中应予以高度重视。 6 人瘦素类似物瘦素主要由脂肪组织合成和分泌，在维持内分泌代谢稳态、能量平衡方面发挥重要作用。美曲普汀（metreleptin）是人瘦素类似物，通过结合并激活人瘦素受体来降TG，需每日皮下注射1次。在针对LMNA、PPARG基因突变的家族性部分脂肪营养不良患者的研究中，美曲普汀显著降低其TG，且在合并严重高三酰甘油血症的家族性部分脂肪营养不良患者中疗效更显著，71% LMNA突变患者和所有的PPARG突变患者TG降低高达30%以上[36]。目前，美曲普汀已在美国、欧盟、日本获批用于治疗家族性部分脂肪营养不良。 7 FXR激动剂奥贝胆酸是一种选择性FXR激动剂，针对FPLD2型女性患者的安慰剂对照交叉试验结果显示，奥贝胆酸将肝脏脂肪含量显著降低39.6%，但对血清TG水平、肝酶等指标无显著改善作用。奥贝胆酸应用期间安全性尚可，主要不良反应为皮肤瘙痒、LDL-C升高[37]。这一研究为FPLD2型女性患者提供了安全有效的治疗方案，但未观察到其具有降TG疗效，且样本量较少。 8 结语严重高三酰甘油血症已经进入了个性化精准治疗的新时代，以奥莱扎尔森和普乐司兰钠为代表的APOC3抑制剂实现了强效、持久且安全性良好的降TG疗效，并可预防急性胰腺炎的发作，这意味着严重高三酰甘油血症治疗实现了重要突破。随着研究的深入，针对ANGPTL3、FGF21、GLP-1R等靶点研发的药物也为不同亚型严重高三酰甘油血症患者提供了更多选择。不同严重高三酰甘油血症亚型治疗方案的选择需基于其核心病理生理机制。家族性乳糜微粒血症综合征因LPL功能基本丧失，治疗有赖于非LPL途径的药物，如APOC3抑制剂（普乐司兰钠为首选）和MTP抑制剂。多因素乳糜微粒血症综合征因存在部分LPL活性和继发性因素，治疗需综合管理，可选择APOC3抑制剂、ANGPTL3抑制剂、GLP-1RA以及传统药物，并积极控制继发性因素。家族性部分脂肪营养不良作为全身性代谢疾病，治疗需全面纠正代谢异常，瘦素类似物（如美曲普汀）和GLP-1RA/多靶点激动剂是核心，可联合传统降脂药物共同管理。尽管新型药物取得了里程碑式进展，但严重高三酰甘油血症的精准管理仍面临挑战。一方面，目前严重高三酰甘油血症分型诊断较困难，临床多依据临床表现和基因检测，亟需开发更便捷的诊断工具；另一方面，既往临床研究存在规模小、随访时间短、种族和地区局限性等不足，未来需开展大型、长期新型靶向药物随机对照临床试验研究和真实世界研究，为明确新型靶向药物的长期疗效和安全性以及停药后的血清TG水平、急性胰腺炎复发情况等提供更有力的证据。此外，严重高三酰甘油血症患者病因复杂多样，单一药物疗效可能捉襟见肘，未来可探索多药联合治疗方案，发挥降低血清TG水平和胰腺炎发生风险的协同作用，并减少药物不良反应的发生。最后，基于现有研究，应继续探索TG合成和代谢的新机制以开发新靶点、改进药物研发技术以提高药物靶向的精准度，为严重高三酰甘油血症治疗提供新的证据和方法。利益冲突所有作者均声明不存在利益冲突参考文献（略）来源：刘苗苗，李哲，李映，马瑜瑾，姜宏卫．严重高三酰甘油血症新型治疗药物的研究进展 [J]. 现代药物与临床, 2026, 41(6): 1830-1837

临床2期临床3期寡核苷酸临床结果

2026-06-18

·GlobeNewswire-Health Care

Chiesi Global Rare Diseases Highlights Advancements in Lipodystrophy Research at ENDO 2026

-- Presentations highlight new clinical, real-world, and patient-centered insights on metreleptin across lipodystrophy populations --BOSTON, June 18, 2026 (GLOBE NEWSWIRE) -- Chiesi Global Rare Diseases, a business unit of Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, today announced multiple presentations at the Endocrine Society’s Annual Meeting (ENDO), held June 13 – 16, 2026, in Chicago, Illinois. The five clinical abstracts focus on the metreleptin clinical program in partial lipodystrophy, the safety/tolerability profile of metreleptin in generalized lipodystrophy, the combination of metreleptin with GLP-1 receptor agonists in adults with lipodystrophy, and the impact of mental health challenges and care gaps in lipodystrophy. Collectively, these data expand the clinical understanding of metreleptin, the overall disease burden that people living with lipodystrophy face, and reflect Chiesi’s ongoing commitment to advancing care for people living with lipodystrophy. “Continued research across both clinical and real-world settings is essential to advancing our understanding of treatment approaches in rare endocrine and metabolic disorders,” said Rachele Berria, MD, PhD, Senior Vice President, Head of Global Medical Affairs, Chiesi Global Rare Diseases. “We are proud to share these findings at ENDO 2026, which reflect our commitment to generating meaningful data while integrating the experiences of people living with lipodystrophy. By combining scientific rigor with patient-centered insight, we aim to help inform more comprehensive and effective models of care.” Data from a survey of patients and caregivers highlighted that the mental health burden in lipodystrophy is multifaceted, driven by factors such as altered body image and diagnostic uncertainty. Despite this, more than half of the respondents lacked mental health support, highlighting a critical gap. Proactive screening, especially at diagnosis, can help providers move beyond metabolic control to wholistic patient care. “For people living with lipodystrophy, the burden of disease can extend beyond what can be measured clinically,” said Stuart Siedman, Vice President, Patient Advocacy, Chiesi Global Rare Diseases. “By advancing research that captures both the biological and lived experience, we aim to help ensure care reflects the full reality of patients’ needs.” ENDO 2026 marks an important moment of scientific advancement for the endocrine community and reflects Chiesi’s ongoing commitment to advancing research in rare endocrine and metabolic diseases through research, collaboration, and holistic care. Details of Chiesi-sponsored presentations are as follows: Title: A 36-Month, Multicenter, Open Label Phase 4 Study to Evaluate the Immunogenicity of Daily Metreleptin Treatment in Patients with Generalized LipodystrophyFormat: Poster PresentationPresenter: Dr. Elif A. OralDate: Saturday, June 13 Title: Characterizing Mental Health Challenges And Care Gaps In Patients With Lipodystrophy And Their Care PartnersFormat: Poster PresentationPresenter: Kate StrattonDate: Saturday, June 13 Title: A 12-Month Randomized, Multicenter, Double-Blind, Placebo-Controlled Phase 3 Study to Evaluate the Safety and Efficacy of Metreleptin in Subjects with Partial Lipodystrophy (METRE-PL Study)Format: Poster PresentationPresenter: Dr. Elif A. OralDate: Sunday, June 14 Title: Effectiveness and Safety of Metreleptin Therapy in Patients with Partial Lipodystrophy: A Systemic ReviewFormat: Poster Presentation Presenter: Dr. Baris AkinciDate: Sunday, June 14 Title: Real-World Prescribing Patterns of Combined Metreleptin and GLP-1 Receptor Agonist Therapy in Adults with LipodystrophyFormat: Poster PresentationPresenter: Dr. Lindsay T. FourmanDate: Monday, June 15 IMPORTANT SAFETY INFORMATION INDICATION: Myalept® (metreleptin) for injection is a leptin analog indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy. LIMITATIONS OF USE: The safety and effectiveness of Myalept for the treatment of complications of partial lipodystrophy or for the treatment of liver disease, including nonalcoholic steatohepatitis (NASH), have not been established. Myalept is not indicated for use in patients with HIV-related lipodystrophy. Myalept is not indicated for use in patients with metabolic disease, including diabetes mellitus and hypertriglyceridemia, without concurrent evidence of generalized lipodystrophy. WARNING: RISK OF ANTI-METRELEPTIN ANTIBODIES WITH NEUTRALIZING ACTIVITY AND RISK OF LYMPHOMA Anti-metreleptin antibodies with neutralizing activity have been identified in patients treated with Myalept. The consequences of these neutralizing antibodies are not well characterized but could include inhibition of endogenous leptin action and/or loss of Myalept efficacy. Severe infection and/or worsening metabolic control have been reported. Test for anti-metreleptin antibodies with neutralizing activity in patients who develop severe infections or show signs suspicious for loss of Myalept efficacy during treatment. Contact Chiesi Farmaceutici S.p.A. at 1-866-216-1526 for neutralizing antibody testing of clinical samples. T-cell lymphoma has been reported in patients with acquired generalized lipodystrophy, both treated and not treated with Myalept. Carefully consider the benefits and risks of treatment with Myalept in patients with significant hematologic abnormalities and/or acquired generalized lipodystrophy. Because of these risks associated with the development of anti-metreleptin antibodies that neutralize endogenous leptin and/or Myalept and the risk for lymphoma, Myalept is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Myalept REMS PROGRAM. CONTRAINDICATIONS: Myalept is contraindicated in general obesity not associated with congenital leptin deficiency. Myalept has not been shown to be effective in treating general obesity. The development of anti-metreleptin neutralizing antibodies have been reported in obese patients treated with Myalept. Myalept is contraindicated in patients with prior severe hypersensitivity reactions to metreleptin or to any of its components. WARNINGS AND PRECAUTIONS: A dose adjustment, including possible large reductions, of insulin or insulin secretagogue may be necessary in some patients to minimize risk of hypoglycemia. Closely monitor blood glucose in patients on concomitant insulin, especially those on high doses, or insulin secretagogue. Cases of progression of autoimmune hepatitis and membranoproliferative glomerulonephritis (associated with massive proteinuria and renal failure) were observed in some patients with acquired generalized lipodystrophy treated with Myalept. A causal relationship between Myalept and the development and/or progression of autoimmune disease has not been established. Carefully consider the benefits and risks of Myalept treatment in patients with autoimmune disease. Hypersensitivity reactions (eg, anaphylaxis, urticaria or generalized rash) have been reported. Patient should promptly seek medical advice about discontinuation of Myalept if a hypersensitivity reaction occurs. Myalept contains benzyl alcohol when reconstituted with Bacteriostatic Water for Injection. The preservative benzyl alcohol has been associated with serious adverse events and death in pediatric patients, particularly in neonates and premature infants. Preservative-free Water for Injection is recommended for use in neonates and infants. ADVERSE REACTIONS: Most common adverse reactions (≥10%) in clinical trials were headache, hypoglycemia, decreased weight, and abdominal pain. Please see Full Prescribing Information, including Boxed Warning. About LipodystrophyLipodystrophy syndromes are a heterogeneous group of rare, potentially life-threatening disorders that affect how the body accumulates and stores fat. These syndromes are categorized into two main forms: generalized lipodystrophy, characterized by the near-complete absence or progressive loss of fat (adipose) tissue, and partial lipodystrophy, where the tissue loss is more limited, typically impacting areas like limbs or upper body. Lipodystrophy is also categorized by etiology with inherited and acquired forms.1 About Chiesi GroupChiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The Company’s mission is to improve people’s quality of life and act responsibly towards both the community and the environment. By changing its legal status to a Benefit Corporation in Italy, the US, France and Colombia, Chiesi’s commitment to creating shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, Chiesi is part of a global community of businesses that meet high standards of social and environmental impact. The Company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035. With 90 years of experience, Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,500 employees. The Group’s research and development center in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden. For more information visit www.chiesi.com About Chiesi Global Rare DiseasesChiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have therapy for all diseases and acts as a force for good, for society and the planet. The goal of the Global Rare Diseases unit is to ensure equal access so as many people as possible can experience their most fulfilling life. The unit collaborates with the rare disease community around the globe to bring voice to underserved people in the health care system. For more information visit www.chiesirarediseases.com. Follow @ChiesiGlobalRareDiseases on LinkedIn, Facebook, Instagram, X and YouTube. Chiesi Global Rare Diseases Media ContactSky StriarLifeSci CommunicationsEmail: sstriar@lifescicomms.com References 1) Garg A. Acquired and inherited lipodystrophies. N Engl J Med. 2004 Mar 18;350(12):1220-34. Doi: 10.1056/NEJMra025261. PMID: 15028826. PP-RA-02187 V1.0

临床3期

100 项与美曲普汀相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D05014	美曲普汀	A16AA07	DB09046

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
瘦素缺乏	欧盟	CHIESI Farmaceutici SpA	2018-07-30
瘦素缺乏	冰岛	CHIESI Farmaceutici SpA	2018-07-30
瘦素缺乏	列支敦士登	CHIESI Farmaceutici SpA	2018-07-30
瘦素缺乏	挪威	CHIESI Farmaceutici SpA	2018-07-30
先天性全身性脂肪营养不良	欧盟	CHIESI Farmaceutici SpA	2018-07-30
先天性全身性脂肪营养不良	冰岛	CHIESI Farmaceutici SpA	2018-07-30
先天性全身性脂肪营养不良	列支敦士登	CHIESI Farmaceutici SpA	2018-07-30
先天性全身性脂肪营养不良	挪威	CHIESI Farmaceutici SpA	2018-07-30
家族局部性脂质营养不良	欧盟	CHIESI Farmaceutici SpA	2018-07-30
家族局部性脂质营养不良	冰岛	CHIESI Farmaceutici SpA	2018-07-30
家族局部性脂质营养不良	列支敦士登	CHIESI Farmaceutici SpA	2018-07-30
家族局部性脂质营养不良	挪威	CHIESI Farmaceutici SpA	2018-07-30
获得性部分脂肪营养不良	欧盟	CHIESI Farmaceutici SpA	2018-07-30
获得性部分脂肪营养不良	冰岛	CHIESI Farmaceutici SpA	2018-07-30
获得性部分脂肪营养不良	列支敦士登	CHIESI Farmaceutici SpA	2018-07-30
获得性部分脂肪营养不良	挪威	CHIESI Farmaceutici SpA	2018-07-30
脂肪营养不良	日本	Shionogi Pharma, Inc.	2013-03-25
脂肪营养不良	日本	Chiesi Ltd.	2013-03-25

未上市

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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	Amryt Pharma Holdings Ltd.	2025-05-23
糖尿病	临床3期	法国	Amryt Pharma Holdings Ltd.	2025-05-23
糖尿病	临床3期	德国	Amryt Pharma Holdings Ltd.	2025-05-23
糖尿病	临床3期	意大利	Amryt Pharma Holdings Ltd.	2025-05-23
高甘油三酯血症	临床3期	比利时	Amryt Pharma Holdings Ltd.	2025-05-23
高甘油三酯血症	临床3期	法国	Amryt Pharma Holdings Ltd.	2025-05-23
高甘油三酯血症	临床3期	德国	Amryt Pharma Holdings Ltd.	2025-05-23
高甘油三酯血症	临床3期	意大利	Amryt Pharma Holdings Ltd.	2025-05-23
多发性对称性脂肪瘤病	临床2期	美国	Amryt Pharma Holdings Ltd.	2023-08-01
下丘脑闭经	临床2期	美国	Amgen, Inc.	2010-04-01

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分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04026178 (CTgov)	临床4期	先天性全身性脂肪营养不良	11	Metreleptin	窪範簾築願艱繭襯遞鬱 = 壓憲鹽範襯淵齋獵廠簾鹹選糧窪窪醖窪蓋鬱壓 (製憲醖憲鬱鏇獵糧廠襯, 遞淵鹹膚壓願選憲餘餘 ~ 鏇獵構醖壓簾製構糧憲) 更多	-	2025-11-26
NCT05351164 (CTgov)	临床2期	多发性对称性脂肪瘤病	4	Metreleptin	鹹蓋鏇齋鏇廠醖襯繭顧(夢範積鏇襯積夢願窪製) = 築簾選淵選簾餘鬱選憲獵願選壓鹹夢製鬱遞願 (鑰積衊糧獵遞積積積蓋, 3.2) 更多	-	2025-06-12
NCT00085982 (CTgov)	临床2期	胰岛素抵抗	11	Metreleptin	鬱夢壓齋簾構鏇艱艱鬱(鑰遞願範願觸簾蓋顧獵) = 夢齋築範淵鏇膚餘積鏇憲餘繭醖夢範鹹遞製蓋 (糧製製艱淵製鏇顧觸壓, 1.921) 更多	-	2021-12-15
NCT02654977 (CTgov)	临床2期	家族局部性脂质营养不良 \| 代谢功能障碍相关脂肪性肝炎	11	Metreleptin	餘壓鏇鑰衊窪繭蓋網鬱(餘夢鹹獵壓鹹鬱觸選鏇) = 鹽鏇網鬱鏇觸衊艱築襯襯淵範窪製願廠夢醖顧 (遞淵範餘遞製鏇醖繭遞, 艱鬱鹽糧積襯鏇遞獵鑰 ~ 鹽廠製艱鹹繭製窪選憲) 更多	-	2020-11-24
NCT01679197 (SUN-607, ENDO2020)	临床2期	脂肪营养不良 ferritin \| hepcidin \| iron ... 更多	19	Metreleptin Therapy	觸遞艱憲鏇願蓋獵製蓋(簾襯餘淵膚壓襯壓獵窪) = 壓繭夢鹹積繭衊壓網憲醖膚齋衊鏇齋衊選選選 (艱積艱糧糧積窪醖廠觸 ) 更多	积极	2020-05-08
NCT01679197 (SUN-607, ENDO2020)	临床2期	脂肪营养不良 ferritin \| hepcidin \| iron ... 更多	19	Placebo	觸窪餘襯淵廠夢網網壓(淵夢簾顧鑰鬱顧製觸憲) = 鹹艱醖壓鹹衊繭鑰顧繭願鏇製糧積襯襯鏇顧遞 (網遞獵觸壓衊鬱網夢積 )	积极	2020-05-08
NCT01679197 (OR17-02, ENDO2020)	临床2期	脂肪营养不良 leptin \| IGF-1 \| apo B ... 更多	18	Metreleptin	襯膚夢憲鑰鹽範蓋繭鬱(窪鏇網憲壓廠蓋齋艱糧) = 壓憲範築選鏇獵襯遞構蓋淵壓範蓋蓋淵餘遞夢 (簾衊簾窪繭範遞窪鏇簾 ) 更多	-	2020-05-08
NCT00657605 (CTgov)	临床2期	代谢综合征 \| 糖尿病 \| 肥胖	3	Recombinant methionyl human leptin	範鹽鏇廠觸鹽齋築選觸 = 選齋鏇醖願壓廠獵憲網襯網齋觸艱醖齋膚艱夢 (顧製衊鏇艱網繭簾廠壓, 窪遞餘餘廠遞憲鏇餘夢 ~ 鏇鹽範餘築積餘窪鬱淵) 更多	-	2020-02-13
NCT00659828 (CTgov)	临床2期	代谢综合征 \| 糖尿病 \| 肥胖	1	Recombinant methionyl human leptin	糧鬱簾襯築糧襯膚簾築 = 網繭壓蓋築積選醖襯淵衊遞艱鏇淵襯簾憲衊遞 (積顧鑰鑰夢鹹膚夢淵襯, 積鹽網築廠襯醖選觸遞 ~ 選窪鬱蓋膚憲糧糧鬱觸) 更多	-	2020-02-13
NCT01778556 (CTgov)	临床2期	脂肪营养不良	25	Metreleptin (Leptin Naive)	襯構糧膚範壓蓋構醖願(夢艱鏇積鹽餘蓋繭醖鹽) = 簾觸築顧夢顧齋鏇壓糧積鏇簾醖製糧積艱蓋艱 (遞鬱淵願構積簾窪齋窪, 2.3) 更多	-	2019-11-13
NCT01778556 (CTgov)	临床2期	脂肪营养不良	25	Metreleptin (On-leptin)	襯構糧膚範壓蓋構醖願(夢艱鏇積鹽餘蓋繭醖鹽) = 網鹽築糧範淵鑰網壓製積鏇簾醖製糧積艱蓋艱 (遞鬱淵願構積簾窪齋窪, 4.1) 更多	-	2019-11-13
NCT00265980 (CTgov)	N/A	体重下降 \| 肥胖	22	Leptin	獵衊餘獵範鑰構壓鹽選(繭觸構餘醖膚襯鏇鹹艱) = 鬱遞糧鹽襯鬱築糧衊壓壓糧淵觸積繭廠窪簾構 (蓋積窪憲夢繭憲鏇製獵, 500) 更多	-	2019-09-18