Decentralized Study to Assess Patient Treatment Preference Comparing Their Current Standard-of-care Wilson's Disease (WD) Treatment to a New Trientine (TETA) 4HCl Formulation.

Decentralized study to assess patient reported treatment satisfaction comparing their current standard-of-care Wilson's Disease (WD) treatment with a new once-daily Trientine (TETA) 4HCl formulation.

开始日期2025-07-15

申办/合作机构

Orphalan SA

CTR20244688

/ CompletedN/A

盐酸曲恩汀片在健康受试者中空腹生物等效性试验

主要目的：考察中国健康受试者在空腹状态下单次口服四川科瑞德制药股份有限公司生产的盐酸曲恩汀片与参比制剂体内药代动力学特征，评价两种制剂的生物等效性。次要目的：评价单次口服盐酸曲恩汀片受试制剂及参比制剂在中国健康受试者中的安全性。

开始日期2024-12-28

申办/合作机构

四川科瑞德制药股份有限公司

NCT06128954

/ Completed临床1期

A Phase I, Single Centre, Randomised, Interventional, Open-Label, Cross-Over Study to Evaluate the Pharmacokinetics (PK) and the Safety and Tolerability of a Total Daily Dose of 900mg of TETA 4HCL, Comparing a New Once Daily TETA 4HCL Formulation (300mg) (3x300mg Trientine Base Tablets, OD) With the Current Marketed Cuprior® Formulation (150mg) (3x150mg Trientine Base Tablets, BD) in Adult Healthy Male and Female Participants

A randomised, open-label study evaluating the pharmacokinetics, safety, and tolerability of a new once daily dose of 900mg of TETA 4HCL by comparing it against the current marketed Cuprior® formulation (450mg trientine base, twice daily) in healthy male and female participants.

开始日期2024-01-16

申办/合作机构

Orphalan SA

100 项与四盐酸曲恩汀相关的临床结果

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100 项与四盐酸曲恩汀相关的转化医学

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100 项与四盐酸曲恩汀相关的专利（医药）

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262

项与四盐酸曲恩汀相关的文献（医药）

2026-01-01·INTERNATIONAL IMMUNOPHARMACOLOGY

Recent advances in the study of cuproptosis in gliomas

Review

作者: Wan, Dengfeng ; Zhang, Zhiqiang ; Lin, Yubo

Gliomas, characterized as highly invasive brain tumors, often render traditional therapies ineffective. Cuproptosis, a recently identified mode of cell death, exhibits mechanisms distinctly different from traditional apoptotic pathways. This process involves the disruption of the mitochondrial tricarboxylic acid cycle by excessive copper ions, leading to cell death. This unique cell death pathway has demonstrated significant potential in the treatment of gliomas. Research has shown that genes associated with cuproptosis, such as FDX1 and LIAS, are aberrantly expressed in gliomas, affecting tumor metabolism and closely correlating with prognosis. Copper chelators, such as trientine and disulfiram, can regulate intracellular copper levels and induce glioma cell death, thereby enhancing the efficacy of chemotherapy. However, the specific mechanisms of cuproptosis and its clinical application in glioma treatment require further exploration. This field of study promises to offer novel insights and directions for developing more effective glioma therapies.

2025-12-01·Current Neurology and Neuroscience Reports

Pitfalls in the Diagnosis of Wilson Disease

Review

作者: Chakravarty, Ambar ; Roy, Debasish ; Mukherjee, Angshuman

PURPOSE OF REVIEW:

Wilson disease (WD), an uncommon autosomal recessive (AR) hereditary disorder characterized by abnormal accumulation of copper primarily in the liver and secondarily in other organs like the brain, is caused by a deficiency in the ATP7B transporter gene. The key to successful therapy is early diagnosis.

RECENT FINDINGS:

Mutant genes need to be inherited from both parents for phenotypic expression. The ATP7B gene located on chromosome 13q14.3 comprises 20 introns and 21 exons, encodes a protein of 165 amino acids, and this helps in incorporation of copper into ceruloplasmin, the copper binding protein. So far, more than 800 mutations have been reported, of which 380 have confirmed involvement in the pathogenesis of WD. The most common mutations are H1069Q and R778L in European and Asian populations respectively. Approximately 90%-98% of WD subjects are heterozygous, showing different mutations in each of the alleles encoding the ATP7B. Conversely, the phenotype and the penetrance of WD can be extremely variable. Even patients carrying two disease-causing mutations do not necessarily have a demonstrable alteration of copper metabolism. Considering the possibility of late-onset disease, asymptomatic cases, and phenotypic variability, it is crucial to evaluate previous and future generations of the index case. WD ranges from being asymptomatic in some patients to result in acute liver failure and/or a variety of neuropsychiatric disorders among others. Although WD may be present at any age, is more common between the ages of 5 and 35 years. However, it should be investigated in patients with liver failure of unknown cause and those with liver disease associated with neuropsychiatric symptomatology. Diagnosis requires a combination of clinical signs and symptoms, as well as relevant diagnostic tests such as measurement of serum ceruloplasmin, urinary excretion of copper, liver biopsy or genetic testing. Treatment is lifelong and includes chelating agents (penicillamine and trientine) and inhibitors of copper absorption (zinc salts). Liver transplant is an option for patients with end-stage liver disease. The key to successful therapy is early diagnosis.

2025-10-20·Zhonghua gan zang bing za zhi = Zhonghua ganzangbing zazhi = Chinese journal of hepatology

[Recommendations from the European Association for the Study of the Liver and the European Reference Network for Rare Liver Diseases Clinical Practice Guidelines for hepatolenticular degeneration].

Review

作者: Hou, W ; Zheng, S J ; Tang, S

Wilson disease (WD), also known as hepatolenticular degeneration, is an autosomal recessive inherited disorder of copper metabolism that primarily affects the liver, brain, and other organs. The diagnostic criteria include clinical features, biochemical tests (plasma ceruloplasmin, 24-hour urinary copper, liver copper content), and molecular genetic analysis. The Leipzig scoring system, supplemented by the use of exchangeable copper, is recommended for diagnosis. Pharmacotherapy mainly includes chelating agents (such as penicillamine and trientine) and zinc salts. Chelating agent therapy is recommended only for patients with severe liver disease. Patient monitoring is primarily based on clinical symptoms, liver biochemical indices, and copper metabolism parameters (such as 24-hour urinary copper and exchangeable copper) to identify poor adherence as well as over-treatment or under-treatment situations. The diagnosis and treatment of acute liver failure with WD is extremely challenging, as the diagnosis is difficult and medical treatment cannot save life. The role of liver transplantation has been clearly recognized in the treatment of acute liver failure with WD, and it may also be considered in cases with neurological involvement.

项与四盐酸曲恩汀相关的新闻（医药）

2025-12-12

·PRNewswire

Orphalan announces acquisition of Orphelia Pharma

PARIS, Dec. 12, 2025 /PRNewswire/ -- Orphalan, an international pharmaceutical company specializing in the development and commercialization of orphan drugs, has acquired Orphelia Pharma, a company committed to treating rare and severe pediatric diseases, notably in neurology and oncology. This strategic transaction strengthens Orphalan's position as a European leader with a global commercial footprint in rare diseases, expanding its therapeutic portfolio. Key highlights of the acquisition include: Expansion of our capabilities in rare neurological and oncological pediatric diseases Greater reach and improved access to medicines for children and adults with rare conditions A unique platform combining scientific excellence, agility, and global commercialization By bringing together complementary expertise, the companies are now positioned to accelerate innovation and improve access to transformative treatments for patients around the world. As they enter this new stage, their teams remain fully committed to ensuring continuity of care and supporting their partners and patients. Dr. Naseem Amin, Chief Executive Officer of Orphalan said: "The acquisition of Orphelia Pharma reinforces our mission: to provide innovative and accessible treatments to patients living with rare diseases, wherever they are. "Together, we are building a unique platform that brings together scientific excellence, agility, international development and commercial capabilities to advance care for patients of all ages suffering from orphan diseases." Dr. Hugues Bienaymé, Founder and Deputy Chief Executive Officer of Orphelia Pharma, added: "At Orphelia Pharma, we have dedicated ourselves to developing safe and effective pediatric medicines that address major unmet medical needs in rare diseases. "Joining Orphalan will allow us to increase the impact of our treatments and make them available more rapidly to the children who need them most." Gilles Alberici, Chief Executive Officer of Orphelia Pharma, commented: "Bringing together our two complementary companies marks an important milestone, both for our organizations and for the broader community of patients living with rare diseases." About Orphalan Founded in 2011, Orphalan SA is an international pharmaceutical company dedicated to the development and commercialization of orphan drugs. Originating from a collaboration with AGEPS and Hôpital Lariboisière, Orphalan developed Cuprior®, a treatment for Wilson's disease, now authorized in 38 countries and marketed in 28. Orphalan's ambition is to reduce diagnostic delays and make innovative orphan drugs accessible to as many patients as possible through a patient-centered approach and close collaboration with healthcare professionals. Orphalan's shareholders include Bpifrance, Advent, and the management team. About Orphelia Pharma Orphelia Pharma is a pharmaceutical company based in Paris and Lyon, specializing in the development and commercialization of medicines for rare and severe pediatric diseases. With one authorized medicine in the European Union ( Kigabeq®) and a second under regulatory review ( Kizfizo®), Orphelia Pharma has established regional distribution agreements across Europe and other regions and collaborates with academic and industrial partners to advance research in rare diseases. Further information: Bien Commun Advisory M. Benedict Brodrick +33.6.76.14.02.02 [email protected] SOURCE Orphalan 21% more press release views with Request a Demo

并购上市批准

2025-12-04

·百度百家

生物医药领域新动态：创新药物临床试验受理与进展

生物制药行业动态一览：创新药物临床试验申请受理情况。聚焦生物制药领域，我们为您追踪最新动态，了解企业资讯。聚焦生物制药领域，我们为您提供一个交流与分享的平台，让您随时了解生物企业的最新信息。通过分析6篇原创内容，深入剖析生物制药行业动态。 ❒ 创新药物临床试验受理聚焦生物制药领域，我们为您追踪行业动态，深入剖析热点话题，并提供一个交流与分享的平台，让您随时了解生物企业最新信息。 ❒ 盐酸曲恩汀片进展 Orphalan研发的盐酸曲恩汀片在2022年9月21日已顺利通过国家药品监督管理局药品审评中心（CDE）的上市许可申请受理环节。作为一种螯合剂，曲恩汀专为结合和清除体内铜而设计，是肝豆状核变性的有效治疗药物。自1982年获得FDA批准以来，该药已成为那些无法耐受青霉胺治疗患者的优选，其副作用也相对较轻。肝豆状核变性，又称威尔逊氏病，是一种常染色体隐性遗传病，由ATP7B基因突变引起体内铜离子转运障碍，导致铜在肝脏、神经系统等多处蓄积，引发一系列临床症状。发病者年龄跨度大，从婴儿到老年人都有报道。 ❒ OMTX705研究进展 OMTX705在临床前研究中显示出对多种癌症的显著疗效，包括肺癌、乳腺癌和肝癌等。其独特的作用机制和良好的安全性，使得OMTX705有望成为未来癌症治疗的新选择。 ❒ TQB2102药物特性 TQB2102注射用溶液，作为一款备受瞩目的候选药物，目前正处于临床研究的关键阶段。TQB2102是一种创新的氘代HER2双抗ADC，其独特之处在于能够特异性结合HER2表达细胞上的HER2的ECD4表位和ECD2表位。通过第一抗原结合片段（scFv）与第二抗原结合片段（Fab）的巧妙设计，TQB2102展现出了对HER2的高亲和力。此外，正大天晴药业集团通过采用氘代技术，成功规避了原研药的专利问题，为患者带来了更多的治疗选择。 ❒ LEO 152020临床试验据悉，LEO 152020是一款口服H4R拮抗剂，专为治疗特应性皮炎而设计。该药物的研究权在2018年8月由JW Pharmaceutical授权给LEO Pharma。多项研究显示，在健康成人中进行的单剂量和重复剂量研究均观察到良好的安全性和耐受性。目前，LEO 152020在美国和欧盟均处于II期临床阶段，正在进行一项随机、三盲、安慰剂对照、多中心的临床试验，旨在评估其治疗中度至重度AD成人的疗效。试验的主要终点是观察湿疹面积和严重程度指数（EASI）从基线到第16周的变化；同时，还将评估患者报告的症状，如瘙痒和失眠。 ❒ 南京征祥药物研究 ZX-4081是一种高选择性的PI3Kγ抑制剂，以其独特的抗肿瘤机理脱颖而出。它通过激活肿瘤微环境中的CD8+T细胞并促进巨噬细胞向M1（抗肿瘤）表型转化，从而有效抑制肿瘤的生长和转移。值得一提的是，PI3Kγ的抑制还能帮助克服免疫检查点抑制剂的耐药性。因此，当ZX-4081与PD-1/L1、CTLA4等免疫检查点抑制剂联合使用时，有望为那些对PD1和CTLA4类抑制剂产生耐药性的患者带来新的治疗希望。目前，ZX-4081在美国已进入临床1期，成为全球第二个进入此阶段的PI3Kγ抑制剂。 ❒ 重庆精准生物细胞制剂 C-13-60是一种旨在突破实体瘤微环境的CAR-T细胞制剂，其推测靶点为CEA。该制剂在官网数据中显示，其适应症广泛，涵盖结直肠癌、肾癌及食管癌。近期研究显示，CEA-CAR-T技术有望通过高压递送机制提升渗透性，进而增强抗肿瘤活性。 ❒ 标新生物GT919开发 GT919是一款靶向IKZF1/3的分子胶降解剂，专为恶性血液肿瘤治疗而设计。标新生物，这家成立于2020年2月的生物医药公司，专注于研发口服蛋白降解小分子药物。自2021年3月正式运营以来，公司已建立起包括人工智能虚拟筛选、体外药效筛选、药代动力学、蛋白质组学以及肿瘤动物药效模型在内的完备药物研发体系。其独特的分子胶降解剂（GLUE）和双机制降解剂（GLUETAC）开发平台，为公司带来了差异化的技术路线和发展战略。

2025-08-20

·ETPharma

Govt mulls tweaks in PRIP to boost rare disease therapies: Pharma Secy

New Delhi: In a bid to address the steep cost of imported rare disease therapies , the government is considering modifications to its Promotion of Research and Innovation in Pharma MedTech Sector ( PRIP ) scheme to support domestic development, said Amit Agrawal, Secretary, Department of Pharmaceuticals. Delivering the special guest address at the FICCI Rare Disease Conference 2025, Agrawal said, “Rare diseases in India are as significant as any other public health issue. While we are known as the pharmacy of the world, the need of the hour is to invest in research and development." “Under this vision we launched PRIP and we are at very final stages of making some tweaks to make it better and one of the key focus is the inclusion of rare diseases and orphan drugs,” he said adding that the government is considering rare diseases as a strategic focus with a higher policy support under the scheme.” Launched in 2023 with an allocation of Rs 5,000 crore, PRIP is a Research-Linked Incentive (RLI) initiative aimed at driving innovation in pharmaceuticals and MedTech. Of the scheme’s total outlay Rs 700 crore is earmarked under component ‘A’ for establishing 7 CoEs at NIPER and the rest Rs 4,250 crore is under component ‘B’ for promoting drug development in areas like New Chemical Entities, Complex generics including biosimilars, medical devices, stem cell therapy, orphan drugs, Anti-microbial resistance etc.” Agrawal also highlighted progress under the Production-Linked Incentive (PLI) scheme, noting that eight rare disease drugs—including Trientine (Wilson’s disease), Nitisinone (Type 1 Tyrosinemia), Eliglustat and Miglustat (Gaucher’s), Hydroxyurea (sickle cell anemia), Sapropterin (Phenylketonuria), Sodium Phenylbutyrate, and Carglumic Acid (Hyperammonemia) are now produced domestically. Stating that PLI scheme has brought down treatment costs significantly, he noted, “the annual cost of therapy for Gaucher’s disease has dropped from around Rs 3.6 crore to Rs 3 to 6 lakhs." The production of these drugs began in 2023 following the addition of pharmaceuticals in the PLI scheme with a financial outlay of ₹15,000 crore for the production period of fiscal year 2022-2023 to 2028-29. However, patient groups argue that the therapies currently covered are largely off-patent small molecules, whereas the the greater hurdle for patients is posed by advanced therapies such as large molecules, biologics, and gene therapies , that are still under patent protection. For instance, a disease-modifying therapy Risdiplam marketed by Swiss drugmaker Roche under the brand name Evrysdi for spinal muscular atrophy (SMA)–a neuromuscular disease–costs Rs 60–80 lakh annually, while Novartis’ one-time gene therapy Zolgensma (onasemnogene abeparvovec) entails Rs 14–16 crore. India’s National Policy for Rare Diseases (NPRD), introduced in 2021, has established 13 CoEs—including the latest at AIIMS Patna—to provide treatment and counselling for 63 listed rare diseases. According to the Ministry of Health and Family Welfare, as of August 2024, over 1,100 patients have benefited. Yet advocacy groups stress that the Rs 50 lakh financial support cap is inadequate for advanced therapies , and access to centers remains limited. By Abhijeet Singh ,

基因疗法

100 项与四盐酸曲恩汀相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
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研发状态

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
肝豆状核变性	欧盟	Orphalan SA	2017-09-05
肝豆状核变性	冰岛	Orphalan SA	2017-09-05
肝豆状核变性	列支敦士登	Orphalan SA	2017-09-05
肝豆状核变性	挪威	Orphalan SA	2017-09-05

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临床结果

适应症

分期

评价

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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


NCT06128954 (CTgov)	临床1期	肝豆状核变性	26	900mg TETA 4HCl Once Daily Formulation (Once Daily Dose Formulation (Treatment A))	鏇構積齋遞顧壓淵鑰觸(夢鑰觸襯廠齋廠齋遞憲) = 範壓觸醖構夢憲簾鹽窪鏇築淵餘鑰襯糧膚醖膚 (壓觸襯簾膚壓遞願醖醖, 顧築糧繭構築鏇醖選鑰 ~ 淵製積醖網範窪鬱膚築) 更多	-	2025-08-15
				900mg TETA 4HCl Cuprior® (Cuprior® Comparator (Treatment B))	鏇構積齋遞顧壓淵鑰觸(夢鑰觸襯廠齋廠齋遞憲) = 膚繭淵積簾觸壓遞鹽獵鏇築淵餘鑰襯糧膚醖膚 (壓觸襯簾膚壓遞願醖醖, 鏇鹽築簾構觸製淵餘鑰 ~ 蓋鹹艱襯築糧蓋製餘遞) 更多
NCT03539952 (CHELATE, EASL2022)	临床3期	维持 24-h urinary copper excretion \| alanine aminotransferase	76	D-penicillamine (DPA)	遞淵壓築艱選艱憲顧齋(構網夢蓋繭醖鹽遞壓醖) = 鬱鏇鬱窪築構壓遞淵鬱壓廠築衊鏇餘遞築製齋 (鹽築廠簾餘鏇醖夢觸窪 )	积极	2022-06-24
				trientine tetrahydrochloride	遞淵壓築艱選艱憲顧齋(構網夢蓋繭醖鹽遞壓醖) = 襯築網衊窪繭膚淵衊廠壓廠築衊鏇餘遞築製齋 (鹽築廠簾餘鏇醖夢觸窪 )
NCT03539952 (EASL2021)	临床3期	肝豆状核变性维持 non-ceruloplasmin copper;(NCC)	53	D-penicillamine	餘艱選鑰餘願鬱遞膚廠(衊鹽蓋壓鏇遞顧獵醖網) = 蓋構繭鑰餘鬱範遞鏇網糧繭製壓願壓鏇艱網蓋 (築鹽襯糧築築膚獵齋窪 )	积极	2021-06-23
				TETA4	餘艱選鑰餘願鬱遞膚廠(衊鹽蓋壓鏇遞顧獵醖網) = 壓衊構鑰鹹繭淵繭餘鏇糧繭製壓願壓鏇艱網蓋 (築鹽襯糧築築膚獵齋窪 )