A Non-interventional, Multicentre Retrospective Study of the Effectiveness and Safety of Teduglutide (REVESTIVE®) in Short Bowel Syndrome Patients in Canada

The main aim of this study is to assess the effectiveness and side effects of teduglutide (Revestive®) in real-world clinical practice setting in adult participants with intestinal failure due to short bowel syndrome (SBS-IF) in Canada.
This study is about collecting existing data only; participants receive teduglutide (Revestive®) by their doctors according to the clinical practice but not as part of this study. No new information will be collected during this study.
The study will be conducted using data from the Takeda OnePath Patient Support Program (PSP) or Takeda Global Pharmacovigilance.

开始日期2023-09-23

申办/合作机构

Takeda Pharmaceutical Co., Ltd.

NCT03953170

/ Withdrawn临床3期IIT

Pilot Study to Investigate the Effect of Teduglutide on Temporary Ileostomy Function and Complications

The objective of the study is to investigate the natural history of patients with a temporary ileostomy and to assess the effect of Teduglutide in reducing morbidity, hospital readmissions and post reversal complications.

开始日期2022-06-02

申办/合作机构

Allegheny-Singer Research Institute

[+1]

NCT05027308

/ Completed临床3期

A Phase 3, Open-label Safety Study of Teduglutide in Japanese Pediatric Patients With Short Bowel Syndrome Who Are Dependent on Parenteral Support, Aged 4 Months of Corrected Gestational Age or Older, and Requiring the Dosing of 1.25 mg Formulation

The main aims of the study are to check for side effects from teduglutide.
Participants will receive a daily injection of teduglutide just under the skin (subcutaneous) for 24 weeks. Then they are followed up for another 4 weeks. Participants may be able to repeat this treatment if they meet specific criteria. The study doctors will check for side effects from teduglutide until it becomes commercially available. The maximum duration of treatment is approximately 51.3 weeks.

开始日期2022-01-04

申办/合作机构

Takeda Pharmaceutical Co., Ltd.

100 项与替度格鲁肽相关的临床结果

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100 项与替度格鲁肽相关的转化医学

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100 项与替度格鲁肽相关的专利（医药）

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项与替度格鲁肽相关的文献（医药）

2025-04-01·FOOD CHEMISTRY

Encapsulation of hydrophobically ion-paired teduglutide in nanoemulsions: Effect of anionic counterions

Article

作者: Park, Jong-Tae ; Jeon, Youkyung ; Choi, Kyeong-Ok ; Kim, Sung-Gun

This study presents a novel method for encapsulating the bioactive peptide teduglutide to enhance its oral bioavailability using O/W nanoemulsion (NE). Recombinant teduglutide (rTGT), produced in E. coli with 93 % purity, was hydrophobically modified through ion-pairing with phytic acid (PA) and sodium dodecyl sulfate (SDS). This approach increased encapsulation efficiency from 48.5 % to 87.5 % and 88.3 %, respectively. rTGT/SDS was incorporated within the core of lipid particles, whereas rTGT/PA was likely oriented on the surface. rTGT/SDS_NE exhibited smaller particle size, greater stability, and low cytotoxicity across all tested concentrations in HT-29 cells. Additionally, rTGT/SDS_NE achieved the highest upregulation of genes associated with intestinal function (VIL1, SGLT1, and GLUT2), although the differences were not statistically significant. These findings highlight the potential of the hydrophobic ion-pairing of rTGT with SDS and its encapsulation in nanoemulsion for efficient delivery of rTGT, suggesting promise for advancing oral peptide therapeutics.

2025-03-01·NUTRITION

Forty-eight months outcomes of teduglutide treatment in adult stable patients with short bowel syndrome and home parenteral nutrition dependence: A real-world Italian single-center observational cohort study

Article

作者: Lamacchia, Stefania ; Silvestri, Angela ; Regano, Nunzia ; Mazzuoli, Silvia ; Guglielmi, Francesco William

AIM OF THE STUDY:

This real-life study is designed to investigate the short and long-term efficacy and safety of teduglutide (TED) and its effects on the quality of life (QoL) in a cohort of adult, stable patients with short bowel syndrome and chronic intestinal failure receiving long-term parenteral support (PS).

PATIENT AND METHODS:

A prospective, single-center study was conducted for individuals who began to take TED between March 2017 and August 2023.

RESULTS:

Ten patients were included in the analysis, among whom the median duration of TED administration was 48 (range, 12-71) months. Data relative to short-term clinical outcomes demonstrate that 4 of 10 (40%) patients were early responders to TED therapy, defined as a >20% reduction in PS requirement at 3 months. Six of the 10 (60%) were non-responders. Data relative to 48 months long-term clinical outcomes demonstrated the absence of late responder patients and underline that all 4 early TED responder patients continued to maintain a sustained reduction in PS. Indeed, two of the 4 patients (50%) responding to TED discontinued PS while the other 2 patients (50%) reduced the number of weekly PS infusions by approximately 50%. The physical and mental components of the QoL improved significantly (P < 0.05) in responsive patients but did not change in non-responsive ones. Predictability of response to TED therapy in this study seemed to be linked to 1) SBS type 3, which was completely absent in the non-responder patients (P < 0.0001), 2) the residual small bowel length was significantly (P < 0.02) higher in responder (102 ± 18 cm) compared to non-responder patients (67 ± 27 cm), and 3) SBS type 1 and 2, enterostomy, surgical complications are significantly predictive of no response. Crohn's disease is equally present in both responder and non-responder patients. The number of side effects observed in our experience was extremely small both at the 3rd (0.01 n°/d/patients) and at the 48th month (0.001 n°/d/patients). No endoscopic neoplastic lesions were observed up to the 48th month of TED therapy.

CONCLUSIONS:

This real-world experience allows us to state that, after 48 months of treatment, TED: 1) is a safe therapy with minimal, rare side effects; 2) produces a rapid reduction in PS volumes in 40% of treated patients; 3) consents 20% of enrolled patients to be weaned off PS; 4) maintains a stable nutritional state and, finally, 5) significantly improves the QoL.

2025-02-01·NUTRITION IN CLINICAL PRACTICE

Retrospective review of growth in pediatric intestinal failure after weaning from parenteral nutrition

Article

作者: Rudolph, Jeffrey ; Nucci, Anita M. ; Bashaw, Hillary ; Kirpich, Alexander

Abstract:

Background:

Growth outcomes in children with intestinal failure (IF) after weaning from parenteral nutrition (PN) may be modified by primary diagnosis and interventions aimed at achieving enteral tolerance. We evaluated growth after weaning by diagnosis and intestinal transplant status and during treatment with the glucagon‐like peptide‐2 analog teduglutide.

Methods:

A two‐center retrospective review was conducted on children diagnosed with IF at age <12 months. The z scores for weight and length/height were examined up to 5 years after PN weaning and in children who received teduglutide for >6 months. Data were reported as median and interquartile range (IQR).

Results:

A total of 362 children (58% male and 72% White) were reviewed; 41% (n = 150) weaned from PN at age 1.5 years (IQR = 0.96–3). Weight and length/height data were available for 144 children; 46 received an intestinal transplant. Median weight and length/height z scores at weaning were −1.15 (IQR = −2.09 to −0.39) and −1.89 (IQR = −2.9 to −1.02), respectively. In those not transplanted, z scores remained stable (± 0.5 change). Children with small bowel atresia experienced accelerated linear growth (> +0.5 change) beginning in year 3. Most children transplanted experienced growth acceleration beginning in year 2. Fourteen children received teduglutide (median = 840 [IQR = 425–1530] days), and growth remained stable throughout treatment. Five were weaned from PN within 1 year.

Conclusion:

We observed stable growth with limited catch‐up after PN weaning, with minimal variation by diagnosis, and during teduglutide therapy. Children who received an intestinal transplant experienced acceleration in weight and linear growth after weaning.

项与替度格鲁肽相关的新闻（医药）

2025-03-07

·微信

肽类药物：现状、进展与未来展望

本文将概述肽类治疗药物、递送平台和疫苗的当前市场和临床试验进展。通过文献分析，探讨肽类药物开发的关键研究领域，并着重介绍肽类药物的结构修饰原则，以及筛选、设计和递送技术的最新进展。新型肽类治疗药物，包括肽 - 药物复合物、新型肽类疫苗和创新的肽类诊断试剂的加速发展，有望推动疾病治疗方案迈向精准定制的新时代。一、肽类药物的发展历程与市场现状自 1922 年胰岛素用于治疗糖尿病，肽类药物正式登上医学舞台。发展至今，全球已有近 100 种肽类药物获批，广泛应用于糖尿病、肥胖症、心血管疾病等诸多领域。从市场数据来看（图 8a展示了部分 GLP-1 激动剂的销售情况），肽类药物市场呈现出持续增长的趋势。以司美格鲁肽为例，其不同剂型在 2024 年的销售额相当可观，这充分反映出市场对肽类治疗药物的强劲需求。通过对 2005 - 2024 年文献的分析（图 3），可以发现该领域的研究热度不断攀升，美国和中国在肽类药物研究方面成果突出。其中，癌症研究逐渐成为肽类药物开发的关键领域（图 3b），这也为攻克癌症难题带来了新的希望。二、肽类药物的特性与优势肽类药物具有诸多独特优势。它的特异性极高，能够精准地作用于目标，极大地减少了对正常细胞的不良影响；免疫原性低，引发人体免疫反应的概率较低；生产成本相对较低，在大规模生产上具有一定的经济优势。然而，肽类药物也存在一些局限性，比如在体内稳定性差，容易被快速降解，导致生物利用度较低，这使得许多肽类药物目前只能通过皮下注射给药，给患者带来了不便。三、肽类药物的递送平台创新为了解决肽类药物的局限性，科研人员在递送平台方面进行了大量创新研究。1.肽 - 药物偶联物（PDCs） PDCs 是一种极具潜力的递送平台，它由细胞靶向肽、连接子和细胞毒性有效载荷组成。形象地说，细胞靶向肽就像是 “导航仪”，能引导药物精准地找到肿瘤细胞；连接子起着连接和控制药物释放的作用；细胞毒性有效载荷则是 “战斗武器”，负责对肿瘤细胞进行攻击。目前，已有一些 PDCs 进入临床试验阶段（表 5展示了处于临床试验阶段的多种 PDCs），例如 Lutathera 用于治疗神经内分泌肿瘤，Pepaxto 用于治疗多发性骨髓瘤，不过 Pepaxto 因疗效问题退出了美国市场。此外，像 SNG1005 和 AEZS - 108 等在研的 PDCs，分别在治疗脑转移癌和前列腺癌方面展现出了良好的前景。 2.CPPs - 工程化纳米载体 CPPs（细胞穿透肽）可以帮助药物顺利穿过细胞膜，进入细胞内部发挥作用。虽然目前相关产品尚未获批上市，但科研人员通过将其与靶向肽结合等方式，不断提高药物递送的精准性。在针对心脏疾病和跨越血脑屏障的药物递送研究方面，已经取得了一些令人欣喜的进展。四、肽类药物在各治疗领域的应用进展1.糖尿病治疗在糖尿病治疗领域，肽类药物不断推陈出新。从最初的胰岛素，到如今的 GLP - 1 受体激动剂，治疗效果得到了显著提升。例如司美格鲁肽，经过结构修饰后，不仅实现了口服给药，而且只需每周服用一次，大大提高了患者的用药依从性。还有替尔泊肽，作为双靶点激动剂，在控制血糖和体重方面表现优异（图 8b对部分 GLP - 1 激动剂的相关信息进行了展示）。2.癌症治疗肽类药物在癌症治疗中发挥着重要作用。抗癌肽（ACPs）可以直接杀死肿瘤细胞，还能作为疫苗激活人体的抗肿瘤免疫反应。像天然抗癌肽 Apicidin，能够抑制肿瘤血管生成；合成或修饰的抗癌肽，如 Octreotide、Bortezomib 和 Motixafortide 等，已获批用于癌症治疗，并在临床实践中取得了一定的成效。此外，肽类在肿瘤诊断方面也有重要应用，例如\([^{68}Ga]Ga - DOTA - TOC\)和 Illuccix® 等放射性药物，能够帮助医生更精准地定位和诊断肿瘤。3.心血管疾病治疗在心血管疾病治疗方面，肽类药物作用于多个靶点。例如作用于 G 蛋白偶联受体的 GLP - 1 受体激动剂，不仅能有效控制血糖，还对心血管疾病具有一定的预防作用；作用于利钠肽受体的 Nesiritide 和 Carperitide 等药物，可用于治疗心力衰竭；作用于血管紧张素转化酶（ACE）的抑制肽，则有助于降低血压。4.抗感染治疗由于抗生素耐药问题日益严峻，抗菌肽（AMPs）成为了研究热点。AMPs 具有抗菌活性高、抗菌谱广、不易产生耐药性等优点。像 Daptomycin、Dalbavancin 和 Rezafungin 等，都是已获批用于治疗细菌感染的抗菌肽药物，还有一些处于研究阶段的药物，也展现出了巨大的潜力。5.抗新冠病毒治疗针对新冠病毒，肽类药物的研发也在积极推进。例如 Paxlovid®，它由 3CL 蛋白酶抑制剂和低剂量 Ritonavir 组成，能够有效降低患者的住院和死亡风险。此外，还有一些针对新冠病毒 S 蛋白和 3CL 蛋白酶的肽类抑制剂正在研究中，有望成为抗击新冠病毒的新利器。6.其他领域治疗肽类药物在消化系统疾病、阿尔茨海默病、罕见病、骨科代谢疾病和偏头痛等领域也有应用和研究进展。比如在消化系统疾病中，部分肽类药物可用于控制胃肠道出血和治疗肠道疾病；在阿尔茨海默病治疗中，科研人员尝试利用肽类干预 β - 淀粉样蛋白的聚集；在罕见病治疗方面，Trofinetide 用于治疗 Rett 综合征，Zilucoplan 用于治疗重症肌无力，Teduglutide 用于治疗短肠综合征，为患者带来了新的希望；在骨科代谢疾病中，一些肽类药物有助于促进骨形成，治疗骨质疏松；在偏头痛治疗中，针对降钙素基因相关肽（CGRP）的药物，如 Ubrogepant 和 Rimegepant 等，能够有效缓解症状。五、肽类疫苗的研究进展在疫苗领域，肽类疫苗具有独特的优势。与传统疫苗相比，它安全性更高，质量更易于控制，还能根据实际需求进行定制。虽然目前尚无肽类疫苗获批上市，但在肿瘤免疫治疗方面的研究进展迅速，许多针对肿瘤特异性抗原的肽类疫苗正在进行临床试验（表 7展示了相关临床试验信息）。六、推动肽类药物发展的关键技术1.展示文库技术展示文库技术是发现新肽类药物的重要手段，包括噬菌体展示技术、细菌展示技术、酵母展示技术、哺乳动物细胞展示技术和无细胞展示技术等。这些技术各自具有优缺点，相互补充，能够快速筛选出与特定靶点结合的肽，为肽类药物的研发提供了有力支持。2.深度学习深度学习在肽类药物研发中也发挥着重要作用。通过对大量数据的分析，它能够辅助设计和优化肽类药物，显著提高研发效率，加速新型肽类药物的发现。七、口服肽类药物的挑战与突破口服肽类药物一直是研究的难点，因为肽类药物在胃肠道中容易被降解，且吸收效果不佳。不过，科研人员通过结构修饰，如采用 D - 氨基酸取代、脂质化、PEG 化和环化等方法，以及改进制药配方，如使用渗透增强剂、开发新型载体等，在口服肽类药物的研究上取得了一定的突破（表 8列举了部分已获批的口服肽类药物及其递送策略）。八、肽类药物面临的挑战与未来展望尽管肽类药物发展前景广阔，但目前仍面临一些挑战，如药物的稳定性、生物利用度、免疫原性等问题有待进一步解决，生产成本也需要降低。不过，随着技术的不断进步，多学科的深度交叉融合，我坚信肽类药物在未来将会取得更大的发展，为更多患者带来更安全、更有效的治疗方案。识别微信二维码，添加生物制品圈小编，符合条件者即可加入生物制品微信群！请注明：姓名+研究方向！版权声明本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观不本站。

疫苗核酸药物

2025-02-27

·医药观澜

17款罕见病新药在中国获批上市！来自罗氏、武田、诺华、诺和诺德等公司

▎药明康德内容团队报道对于罕见病患者，每一款新药的诞生都是一束光！过去一年，在中国又有一批罕见病新药获批上市，为无数患者和家庭带来希望。在罕见病日到来的时节，让我们回顾这些进展，致敬科研与坚持的力量，期待更多生命被治愈。根据中国国家药监局（NMPA）官方批件信息以及各公司公开资料，2024年全年有至少17款罕见病新药获批上市，覆盖Wilson病、阵发性睡眠性血红蛋白尿症、血友病、“渐冻症”、杜氏肌营养不良等十多种罕见病类型。（本文盘点范围为2024年首次获NMPA批准上市的1类、3.1类和5.1类新药；罕见病参考中国《第一批罕见病目录》及《第二批罕见病目录》）图片来源：123RF Orphalan公司：盐酸曲恩汀片作用机制：铜离子络合剂适应症：Wilson病 2024年1月，Orphalan公司的四盐酸曲恩汀薄膜衣片获NMPA批准上市，用于治疗不耐受D-青霉胺治疗的成人、青少年和≥5岁儿童的Wilson病（威尔逊病，又称肝豆状核变性）。在Wilson病患者中，遗传突变使这种胆汁排泄途径失效，导致过量的铜在肝细胞中累积，并最终超过身体的安全储存能力，引起细胞损伤。曲恩汀（trientine）是一款铜离子络合剂，它的作用类似青霉胺，可以促使铜通过肾脏排泄出体外。罗氏：可伐利单抗注射液作用机制：抗C5循环单克隆抗体适应症：PNH 2024年2月，罗氏（Roche）的可伐利单抗获NMPA批准上市，用于未接受过补体抑制剂治疗的阵发性睡眠性血红蛋白尿症（PNH）成人和青少年（≥12岁）患者。可伐利单抗是一种新型抗C5循环单克隆抗体，患者可进行每四周一次皮下注射。PNH是一种罕见且危及生命的综合征，患者容易出现乏力、骨髓衰竭、血红蛋白尿、贫血、血栓等症状。诺贝仁医药：醋酸锌片作用机制：铜吸收抑制剂适应症：Wilson病（肝豆状核变性） 2024年2月，由诺贝仁制药（Nobelpharma）申报的5.1类新药醋酸锌片获NMPA批准上市，用于治疗Wilson病。醋酸锌片是一种铜吸收抑制剂。研究表明，锌制剂既能阻止铜在肠道的吸收，也能清除沉积于组织中的铜，可作为该疾病的治疗方式之一。武田：注射用替度格鲁肽作用机制：GLP-2类似物适应症：短肠综合征 2024年2月，武田（Takeda）研发的注射用替度格鲁肽获NMPA批准上市，用于治疗短肠综合征（SBS）成人和1岁及以上儿童患者。替度格鲁肽为一款人胰高血糖素样肽2（GLP-2）类似物。短肠综合征是指因各种原因引起广泛小肠切除后，肠道有效吸收面积减少，残存功能性肠管不能维持患者的营养或生长需求，出现以腹泻、酸碱/水/电解质紊乱、以及营养吸收和代谢功能障碍为主的症候群。研究证实，替度格鲁肽可以增加绒毛高度及隐窝深度，加强肠道上皮屏障，从而减轻局部炎症并改善肠道通透性，促进肠适应。武田：注射用舒索凝血素α 作用机制：重组猪FⅧ（rpFⅧ）药物适应症：获得性血友病A 2024年2月，武田注射用舒索凝血素α（susoctocog alfa）在中国获批上市，用于获得性血友病A成人患者按需治疗和出血事件的控制。舒索凝血素α是是一款重组猪FⅧ（rpFⅧ）药物，它与人序列凝血因子Ⅷ结构相似，序列同源，而且不容易受到抗人FⅧ自身抗体的灭活，可以替代人FⅧ发挥明显的止血作用。血友病（hemophilia）是一种X染色体连锁的隐性遗传性出血性疾病，可分为血友病A和血友病B。前者为凝血因子Ⅷ（FⅧ）缺乏，后者为凝血因子Ⅸ（FⅨ）缺乏，均由相应的凝血因子基因突变引起。诺华：盐酸伊普可泮胶囊作用机制：补体B因子口服抑制剂适应症：PNH 2024年4月，诺华（Novartis）创新药物盐酸伊普可泮胶囊获得NMPA批准上市，用于治疗既往未接受过补体抑制剂治疗的阵发性睡眠性血红蛋白尿症（PNH）成人患者。盐酸伊普可泮胶囊是一款特异性补体B因子口服抑制剂，作用于免疫系统的补体旁路途径中的近端通路，可通过控制血管内溶血和血管外溶血，弥补抗C5抗体治疗的缺陷，为PNH提供了一种新型的口服单药靶向治疗选择。爱施健：醋酸氟氢可的松片作用机制：肾上腺皮质激素类产品适应症：失盐型先天性肾上腺皮质增生症和失盐型原发性慢性肾上腺皮质功能减退症 2024年5月，爱施健（aspen）的醋酸氟氢可的松片上市申请已获得NMPA批准上市，用于治疗失盐型先天性肾上腺皮质增生症（CAH）和失盐型原发性慢性肾上腺皮质功能减退症（Addison病，艾迪生病）。其中，21-羟化酶缺乏症（21-OHD）是先天性肾上腺皮质增生症中最常见的类型，是由于编码21-羟化酶的基因缺陷导致肾上腺皮质类固醇激素合成障碍的一种先天性疾病，患者需要每天甚至终身服药。醋酸氟氢可的松片为肾上腺皮质激素类产品，能抑制结缔组织增生，降低毛细血管和细胞膜的通透性，减少炎性渗出，抑制组胺及其他炎症介质的形成和释放。诺和诺德：注射用培妥罗凝血素α 作用机制：长效重组凝血因子VIII 适应症：血友病A 2024年7月，诺和诺德（Novo Nordisk）注射用培妥罗凝血素α获NMPA批准上市，用于12岁及以上成人和儿童血友病A患者按需治疗及控制出血事件、围手术期管理、常规预防治疗以防止或减少出血事件的发生。诺和诺德新闻稿指出，这是中国获批的首个长效重组凝血因子VIII（FVIII），开启了血友病长效治疗的新时代。注射用培图罗凝血素α（N8-GP）经创结构修饰后可将半衰期延长1.6倍，以长效治疗帮助血友病患者减少输液次数，减轻治疗负担。元羿生物/Marinus公司：加那索龙口服混悬剂作用机制：靶向GABAA受体的阳性别构调节剂适应症：“希舞综合征” 2024年7月，元羿生物引进自Marinus公司的的加那索龙口服混悬剂在中国获批上市，用于治疗2岁及以上细胞周期蛋白依赖性激酶5（CDKL5）缺乏症患者癫痫发作。CDKL5缺乏症（又称为“希舞综合征”）是由位于X染色体上的CDKL5基因突变引起，具有难以控制的癫痫发作和重度神经发育障碍等特征。加那索龙（ganaxolone）是一款靶向GABAA受体的阳性别构调节剂，具有静脉注射和口服两种给药方式。它作用于神经元突触和突触外GABAA受体，从而达到抗癫痫和抗焦虑活性的效用。药华医药：罗培干扰素α-2b注射液作用机制：超长效干扰素适应症：真性红细胞增多症 2024年7月，药华医药的罗培干扰素获得NMPA批准上市，用于治疗真性红细胞增多症（PV），这是一种造血干细胞恶性肿瘤。罗培干扰素是单一异构体超长效干扰素，可2~4周注射1次。经多项临床研究显示，该产品可快速有效控制血细胞的过度增殖、并显著降低致病基因的突变负荷，从而有效降低血栓栓塞并发症及其所导致的死亡风险。武田：注射用伏尼凝血素α 作用机制：重组血管性血友病因子适应症：血管性血友病 2024年8月，武田的注射用vonicog alfa（注射用伏尼凝血素α）在中国获批上市，用于血管性血友病成人患者（18岁及以上）的按需治疗和出血事件控制，以及围手术期出血管理。血管性血友病是常见的遗传性出血性疾病之一，主要是由于血管性血友病因子（VWF）基因突变引起血浆中的血管性血友病因子数量减少或质量异常。根据不同的类型，VWD患者的出血严重程度差异大。其中血管性血友病Ⅲ型已在中国被纳入《第二批罕见病目录》。注射用伏尼凝血素α是一款重组血管性血友病因子。它包含完整的VWF多聚体，含有超大多聚体，半衰期长，可有效实现血管性血友病的替代治疗。兴科蓉药业/奥克特珐玛：注射用重组人凝血因子Ⅷ 作用机制：重组人凝血因子Ⅷ 适应症：血友病A 2024年8月，奥克特珐玛（Octapharma）和兴科蓉药业共同申报的重组人凝血因子Ⅷ获NMPA批准上市。重组人凝血因子Ⅷ是一种用于血友病A替代治疗的产品。渤健：托夫生注射液作用机制：反义寡核苷酸（ASO）药物适应症：SOD1基因突变的ALS成人患者 2024年10月，由渤健（Biogen）和Ionis Pharmaceuticals联合开发的托夫生注射液在中国获批上市，用于治疗携带超氧化物歧化酶1（SOD1）基因突变的肌萎缩侧索硬化（ALS）成人患者。ALS是一种进行性神经退行性疾病，也被称为“渐冻症”。许多基因被认为与此疾病有关联，其中具有SOD1突变的患者（SOD1-ALS）约占2%。且患者中大部分疾病进展迅速。托夫生注射液是一种反义寡核苷酸（ASO）药物，可通过减少SOD1蛋白合成，减少毒性SOD1蛋白的蓄积，从而减轻运动神经元的损伤，减缓疾病进展。安进：阿伐可泮胶囊作用机制：口服选择性补体5a（C5a）受体抑制剂适应症：ANCA相关血管炎 2024年11月，安进（Amgen）公司的阿伐可泮胶囊获NMPA批准上市，适用于成人严重、活动性抗中性粒细胞胞浆抗体（ANCA）相关性血管炎（肉芽肿性多血管炎[GPA]或显微镜下多血管炎[MPA]）在含糖皮质激素的标准治疗下的辅助治疗。ANCA相关血管炎（AAV）是一种罕见的血管炎症性疾病，其疾病机制是由于C5a补体途径的过度激活，进一步激活中性粒细胞，导致全身炎症反应和小血管破坏，进而可能导致肾脏、肺和其他器官的严重损伤和功能障碍。阿伐可泮是一款口服选择性补体5a（C5a）受体抑制剂，它通过精确阻断位于中性粒细胞等炎症细胞上的C5a受体，阻止这些细胞受到C5a的激活，降低炎症损伤。华东医药：注射用利纳西普作用机制：IL-1抑制剂适应症：CAPS、复发性心包炎 2024年11月，华东医药引进的注射用利纳西普获NMPA批准上市，用于治疗成人和12岁及以上青少年冷吡啉相关周期性综合征（CAPS），包括家族性寒冷型自身炎症综合征（FCAS）、Muckle-Wells综合征（MWS）。同年12月，该产品获批罕见病新适应症，用于治疗成人和12岁及以上青少年复发性心包炎（RP）以及降低复发风险。注射用利纳西普是一款每周进行皮下注射给药的重组二聚体融合蛋白，可阻断白细胞介素-1α（IL-1α）和IL-1β的信号传导。 CAPS是与NLRP3基因缺陷有关的一组罕见的炎症性疾病。NLRP3基因突变的患者会不断过度产生IL-1β分子，从而引起炎症。该病的临床特征是反复发作的多系统炎症，累及皮肤、肌肉、骨骼、关节、眼、耳以及中枢神经系统等。复发性心包炎是一种罕见的自身炎症性的心血管疾病，患者会有反复的胸痛发作，且伴有危及生命的并发症。益普生：奥德昔巴特胶囊作用机制：IBAT抑制剂适应症：进行性家族性肝内胆汁淤积症 2024年12月，益普生（Ipsen）公司奥德昔巴特胶囊（odevixibat）获NMPA批准上市，用于治疗≥6月龄的进行性家族性肝内胆汁淤积症（PFIC）患者的瘙痒。PFIC是一种罕见的儿童疾病，会导致进行性、危及生命的肝病。在许多情况下，PFIC在患者出生后10年内会发生肝硬化和肝衰竭。PFIC最突出的持续症状是剧烈瘙痒，患者的生活质量严重下降。奥德昔巴特胶囊是一种强效、非体循环回肠胆汁酸转运蛋白（IBAT）抑制剂，可通过抑制回肠远端胆汁酸的再摄取，增加胆汁酸通过结肠的清除，降低血清中胆汁酸浓度，减轻患者瘙痒症状。曙方医药：伐莫洛龙口服混悬液作用机制：新型解离性皮质类固醇适应症：DMD 2024年12月，曙方医药引进自Santhera Pharmaceuticals的伐莫洛龙口服混悬液（vamorolone）在中国获批上市，用于治疗4岁及以上杜氏肌营养不良（DMD）患者。DMD是一种致命的罕见X连锁退行性神经肌肉疾病。伐莫洛龙的作用机制与糖皮质激素结合的受体相同，但改变了其下游活性，且不会造成局部组织扩增和局部组织中皮质类固醇相关毒性。让罕见病不再“罕治”！我们期待在全球各界的共同携手努力下，罕见病治疗领域可以涌现更多创新成果，为更多患者照亮未来。本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权或其他合作需求，请联系wuxi_media@wuxiapptec.com。免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。

上市批准

2025-02-03

·jaguarhealth.gcs-web.com

Jaguar Health Announces First Patient Dosed in Short Bowel Syndrome with Intestinal Failure in Investigator-Initiated Trial of Crofelemer; Proof-of-Concept Data Potentially Available H1 2025

Trial taking place at Sheikh Khalifa Medical City, a flagship United Arab Emirates hospital and largest teaching medical center in Abu Dhabi Crofelemer, Jaguar's novel plant-based prescription drug, has been granted Orphan Drug Designation by the FDA and the European Medicines Agency for both SBS-IF and MVID Proof-of-concept (POC) data generated from this study could potentially lead to reimbursed early patient access in certain European countries for crofelemer for these rare/orphan diseases SAN FRANCISCO, CA / ACCESS Newswire / February 3, 2025 / Jaguar Health, Inc. (NASDAQ:JAGX) (Jaguar) family companies Napo Pharmaceuticals (Napo) and Napo Therapeutics today announced that the first short bowel syndrome with intestinal failure (SBS-IF) patient has been dosed in the independent investigator-initiated pediatric POC trial (IIT) of crofelemer, Jaguar's novel plant-based antidiarrheal prescription drug. The first infant with microvillus inclusion disease (MVID) was dosed as part of the same IIT two weeks ago. POC data generated from this study potentially in the first half of 2025 could lead to reimbursed early patient access to crofelemer for these indications in certain European countries. The trial is being conducted by Dr. Mohamad Miqdady, a recognized leader in pediatric gastroenterology who is the Division Chief of the Pediatric Gastroenterology, Hepatology & Nutrition Division at Sheikh Khalifa Medical City (SKMC), a flagship tertiary hospital in the UAE and the largest teaching medical center in Abu Dhabi . "Dosing of the SBS-IF first patient in this unblinded study is another important milestone in clinical development efforts for crofelemer for SBS-IF and MVID - both of which are rare and severe diseases requiring intensive parenteral nutrition and support," said Lisa Conte , Jaguar's founder, president, and CEO. "This study is one of five clinical efforts in rare diseases - three POC IIT studies and two Phase 2 studies - for crofelemer for the orphan disease indications of MVID and/or SBS-IF in the United States , European Union , and/or Middle East and North Africa (MENA) regions," Conte said. "The Company's Phase 2 study to evaluate the efficacy of crofelemer for MVID in pediatric patients has been initiated, as has the independent IIT in the U.S. to evaluate crofelemer for SBS-IF in adults. The two additional studies are expected to initiate in early Q1 2025, with the availability of the first POC IIT result potentially in H1 2025, and with additional POC IIT results expected throughout 2025. In accordance with the guidelines of specific EU countries, published data from clinical investigations in MVID and SBS-IF could support reimbursed early patient access to crofelemer for these debilitating conditions." SBS-IF and MVID, rare and severe diseases requiring intensive parenteral nutrition and support, have severe morbidity and mortality implications and impact the quality of life of both patients and their caregivers. SBS affects approximately 10,000 to 20,000 people in the U.S. , according to the Crohn's & Colitis Foundation , and it is estimated that the population of SBS patients in Europe is approximately the same size. Pediatric SBS is a malabsorptive condition caused by surgical intestinal resection due to congenital abnormalities, vascular insufficiency or severe inflammatory intestinal disease. The incidence of pediatric SBS varies between 0.02% to 1.2% of live births.1 Based on an estimate of 3.6 million live births in the U.S. in 2023,2 the maximal incidence of pediatric SBS is estimated to be approximately 43,000. Pediatric SBS-IF is associated with significant morbidity and mortality; and high medical expenses, and the patients also have severe chronic diarrhea. The associated sequelae from chronic diarrhea include significant dehydration, metabolic acidosis or alkalosis, and malnutrition. MVID is a pediatric disease with an estimated prevalence of a couple of hundred patients globally. It is characterized by severe diarrhea and malabsorption, requiring intensive parenteral support for nutritional and fluid management. Each MVID patient is a unique patient; their journey requires very careful management of their nutritional needs, and there are currently no approved drug treatments for MVID. The trial is taking place at SKMC. Autosomal recessive disorders and congenital anomalies such as MVID are more prevalent in the MENA region. Crofelemer has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency for SBS and MVID . Dr. Miqdady is an Adjunct Professor at Khalifa University's medical school in Abu Dhabi , and serves as a member of Napo's Scientific Advisory Board . He completed his Fellowship in Pediatric Gastroenterology at Baylor College of Medicine and Texas Children's Hospital in Houston . About Crofelemer Crofelemer is a novel, oral plant-based prescription medicine purified from the red bark sap, also referred to as "dragon's blood," of the Croton lechleri tree in the Amazon Rainforest. Napo has established a sustainable harvesting program, under fair trade practices, for crofelemer to ensure a high degree of quality, ecological integrity, and support for indigenous communities. About the Jaguar Health Family of Companies Jaguar Health, Inc. (Jaguar) is a commercial stage pharmaceuticals company focused on developing novel proprietary prescription medicines sustainably derived from plants from rainforest areas for people and animals with gastrointestinal distress, specifically associated with overactive bowel, which includes symptoms such as chronic debilitating diarrhea, urgency, bowel incontinence, and cramping pain. Jaguar family company Napo Pharmaceuticals (Napo) focuses on developing and commercializing human prescription pharmaceuticals for essential supportive care and management of neglected gastrointestinal symptoms across multiple complicated disease states. Napo's crofelemer is FDA-approved under the brand name Mytesi® for the symptomatic relief of noninfectious diarrhea in adults with HIV/AIDS on antiretroviral therapy. Jaguar family company Napo Therapeutics is an Italian corporation Jaguar established in Milan, Italy in 2021 focused on expanding crofelemer access in Europe and specifically for orphan and/or rare diseases. Jaguar Animal Health is a Jaguar tradename. Magdalena Biosciences, a joint venture formed by Jaguar and Filament Health Corp. that emerged from Jaguar's Entheogen Therapeutics Initiative (ETI), is focused on developing novel prescription medicines derived from plants for mental health indications. For more information about: Jaguar Health , visit https://jaguar.health Napo Pharmaceuticals , visit www.napopharma.com Napo Therapeutics, visit napotherapeutics.com Magdalena Biosciences, visit magdalenabiosciences.com Visit the Make Cancer Less Shitty patient advocacy program on Bluesky, X, Facebook & Instagram Forward-Looking Statements Certain statements in this press release constitute "forward-looking statements." These include statements regarding Jaguar's expectation that another IIT and another Phase 2 study of crofelemer will initiate in early Q1 2025, Jaguar's expectation that POC data from the IIT in Abu Dhabi may be available in H1 2025, Jaguar's expectation that additional POC IIT results for crofelemer may be available throughout 2025, and Jaguar's expectation that, in accordance with the guidelines of specific EU countries, published data from clinical investigations could support early patient access to crofelemer for SBS-IF or MVID in these countries. In some cases, you can identify forward-looking statements by terms such as "may," "will," "should," "expect," "plan," "aim," "anticipate," "could," "intend," "target," "project," "contemplate," "believe," "estimate," "predict," "potential" or "continue" or the negative of these terms or other similar expressions. The forward-looking statements in this release are only predictions. Jaguar has based these forward-looking statements largely on its current expectations and projections about future events. These forward-looking statements speak only as of the date of this release and are subject to several risks, uncertainties, and assumptions, some of which cannot be predicted or quantified and some of which are beyond Jaguar's control. Except as required by applicable law, Jaguar does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. 1 Beth A. Carter , MD, et al. Outcomes from a 12-Week, Open-Label, Multicenter Clinical Trial of Teduglutide in Pediatric Short Bowel Syndrome. The Journal of Pediatrics , 2017; 181:102-11 2 https://www.ncbi.nlm.nih.gov/books/NBK607756/ Contact: hello@jaguar.health Jaguar-JAGX SOURCE: Jaguar Health, Inc.

孤儿药临床2期上市批准

100 项与替度格鲁肽相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	替度格鲁肽	A16AX08	DB08900

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
短肠综合征	欧盟	Takeda Pharmaceuticals International AG	2012-08-30
短肠综合征	冰岛	Takeda Pharmaceuticals International AG	2012-08-30
短肠综合征	列支敦士登	Takeda Pharmaceuticals International AG	2012-08-30
短肠综合征	挪威	Takeda Pharmaceuticals International AG	2012-08-30

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
术后并发症	临床3期	美国	Shire Plc	2022-06-02
瘘	临床2期	美国	Takeda Pharmaceutical Co., Ltd.	2019-01-31
克罗恩病	临床2期	美国	Shire Plc	2003-11-12
克罗恩病	临床2期	加拿大	Shire Plc	2003-11-12

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临床结果

适应症

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


UEGW2024	N/A	肠衰竭	-	TEDUGLUTIDE	鬱鹹壓鑰觸鹹鑰觸醖顧(製觸鑰齋繭壓鬱鑰齋獵) = 夢齋憲憲窪繭鏇構糧膚繭願選網窪襯構鏇齋範 (襯簾構鹽衊壓選窪網襯 ) 更多	-	2024-10-13
				Placebo	鬱鹹壓鑰觸鹹鑰觸醖顧(製觸鑰齋繭壓鬱鑰齋獵) = 鑰醖觸齋顧範憲觸窪構繭願選網窪襯構鏇齋範 (襯簾構鹽衊壓選窪網襯 ) 更多
NCT05027308 (CTgov)	临床3期	短肠综合征	3	Teduglutide	顧積餘顧鬱願積選網膚 = 鬱艱窪鏇選積觸蓋膚鹹觸餘廠窪窪構繭膚憲艱 (鏇顧願觸蓋夢選鏇範築, 願鹹壓蓋艱網窪壓壓製 ~ 簾膚觸壓網憲窪壓糧繭) 更多	-	2024-05-16
NCT03268811 \| NCT03571516 \| NCT02980666 (Pubmed)	临床3期	短肠综合征	-	Teduglutide	夢憲醖製繭鬱鬱觸積蓋(廠遞醖選廠構醖壓齋築) = All adverse events were in line with known impacts of SBS-IF and adverse reactions to teduglutide. Only one serious adverse event (abdominal pain) was considered related to teduglutide. 繭繭網夢鏇襯網窪廠願 (獵醖鹹齋範憲鏇襯淵餘 )	积极	2023-06-27
				Teduglutide (Standard of care)
NCT02431325 (CTgov)	临床2期	动脉炎 \| HIV感染	32	Teduglutide (Teduglutide)	簾夢構襯鬱餘顧糧獵積(糧憲範壓範選鑰壓範積) = 鑰鹽糧鏇壓膚衊餘夢夢鹽窪網構簾選構淵願憲 (繭齋築築顧願遞淵積艱, 0.29) 更多	-	2023-06-12
				Placebo (Placebo)	簾夢構襯鬱餘顧糧獵積(糧憲範壓範選鑰壓範積) = 餘齋齋鑰蓋鬱窪選蓋鏇鹽窪網構簾選構淵願憲 (繭齋築築顧願遞淵積艱, 0.35) 更多
NCT03562130 (Pubmed \| Am J Clin Nutr)	临床4期	短肠综合征	25	Teduglutide	網壓積築鬱積糧顧簾獵(艱窪淵淵製艱鏇廠蓋繭) = Mild abdominal pain at the early phase of treatment, stoma changes, and redness at the injection site were commonly reported 廠製夢壓顧製壓鹽鏇鏇 (遞獵遞觸糧襯憲鏇艱遞 )	-	2023-06-01
NCT03596164 (CTgov)	临床3期	短肠综合征	11	SHP633-306+Teduglutide (SHP633-306: Teduglutide 0.05 mg/kg)	艱簾襯糧鑰構願蓋夢範 = 窪範憲獵構窪網艱鏇網壓憲憲憲構壓網壓膚糧 (蓋鑰壓製築範餘築膚糧, 繭遞鑰製襯獵獵衊齋壓 ~ 齋襯廠鬱鹹醖選艱糧範) 更多	-	2023-03-15
				TED-C14-004+Teduglutide (TED-C14-004: Teduglutide 0.05 mg/kg)	艱簾襯糧鑰構願蓋夢範 = 夢製餘鹹窪醖構選遞顧壓憲憲憲構壓網壓膚糧 (蓋鑰壓製築範餘築膚糧, 網構襯繭鹹築窪糧鏇蓋 ~ 獵觸襯窪鏇鹽齋鹹顧夢) 更多
NCT03268811 (CTgov)	临床3期	短肠综合征	9	teduglutide (Total Children (Aged: 1 to 15 Years))	餘網鏇夢製衊網廠遞鬱 = 簾範鹹鹹窪壓淵積顧蓋願獵築膚鹽構遞艱繭鏇 (夢範夢積淵憲襯餘醖襯, 鑰憲遞簾鏇鏇餘鹹窪餘 ~ 選膚壓製簾觸淵鏇鹽築) 更多	-	2022-05-24
				teduglutide (Infants (Corrected Gestational Age: 4 to < 12 Months))	餘網鏇夢製衊網廠遞鬱 = 餘夢網鑰壓襯積蓋壓壓願獵築膚鹽構遞艱繭鏇 (夢範夢積淵憲襯餘醖襯, 製衊壓壓鏇鑰窪選網糧 ~ 艱構淵襯範積選顧齋蓋) 更多
NCT02954458 (CTgov)	临床3期	短肠综合征	61	NTT+teduglutide (Non-teduglutide/Non-teduglutide Treatment (NTT/NTT))	廠獵窪憲襯鑰觸鑰鬱鑰 = 艱製築範積夢鹽構壓範選夢蓋蓋築餘願餘齋襯 (鑰膚製鑰壓鑰願衊觸蓋, 窪齋襯遞憲鬱製糧觸製 ~ 餘齋艱鹽膚顧遞鬱選廠) 更多	-	2021-10-13
				NTT+Teduglutide (Non-teduglutide/Teduglutide Treatment (NTT/TED))	廠獵窪憲襯鑰觸鑰鬱鑰 = 餘築餘鹹觸蓋網選醖襯選夢蓋蓋築餘願餘齋襯 (鑰膚製鑰壓鑰願衊觸蓋, 廠獵艱選繭衊製餘窪蓋 ~ 窪網淵衊簾範觸網艱窪) 更多
NCT02949362 (CTgov)	临床3期	短肠综合征	29	teduglutide (Retrospective TED/NTT Group)	憲壓願鹹淵鹽鏇衊觸廠 = 範衊衊繭鬱壓蓋淵鏇獵積膚窪廠獵憲繭鹽顧鑰 (淵襯壓壓鹹積鏇構艱積, 窪齋窪衊鹽鑰醖襯鏇獵 ~ 簾獵夢糧構製範鬱糧繭) 更多	-	2021-06-14
				teduglutide (Retrospective TED/TED Group)	憲壓願鹹淵鹽鏇衊觸廠 = 廠築窪鏇廠壓願製獵獵積膚窪廠獵憲繭鹽顧鑰 (淵襯壓壓鹹積鏇構艱積, 選廠網餘膚觸製醖鏇襯 ~ 壓獵壓構網鏇繭鬱糧鏇) 更多
NCT03571516 (CTgov)	临床3期	短肠综合征	10	Syringe	積淵鬱顧廠鏇築齋鑰憲 = 願鑰糧蓋鏇網糧膚顧鑰襯構鬱顧簾遞夢醖憲齋 (觸鹹顧憲築憲齋觸選餘, 齋製襯繭淵鑰製願顧簾 ~ 艱膚醖蓋製廠遞選獵壓) 更多	-	2021-04-08