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

DRKS00028305N/A

Prospective, descriptive, non-interventional, German multicenter study to evaluate the quality of life of Wilson disease patients after switching from D-penicillamine to trientine tetrahydrochloride and to assess clinical and patient reported outcomes - PACUS

开始日期2022-04-01

申办/合作机构-

IRCT20200105046010N34 / Recruiting

In- Vivo Bioequivalence study of Trientine hard capsules 250 mg with brand drug (CYPRINE® 250 mg Merck & Co., Germany) in Iranian healthy volunteers.

开始日期2021-07-01

申办/合作机构-

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100 项与四盐酸曲恩汀相关的专利（医药）

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项与四盐酸曲恩汀相关的文献（医药）

2024-11-01·JIMD reports

Clinical experience on switching trientine tetrahydrochloride to trientine dihydrochloride in Wilson disease patients

Article

作者: Weber, Christophe ; Leidner, Viola Yuriko ; Poujois, Aurélia ; Langel, Andrea ; Schmitt, Timo ; Mohr, Isabelle ; Langel, Jessica ; Weiss, Karl Heinz ; Schall, Nicolas ; Merle, Uta

Abstract:

This study evaluates the effectiveness and safety of trientine dihydrochloride (TETA 2‐HCl) in patients with Wilson disease (WD) following a switch from trientine tetrahydrochloride (TETA 4‐HCl). A total of 30 WD patients with stable copper metabolism were identified for treatment with TETA 2‐HCl (Cufence™) after prior use of TETA 4‐HCl (Cuprior™). Biochemical markers including urinary copper, non‐ceruloplasmin bound copper (NCC) and liver function were analyzed at baseline and followed up over 12 months. Safety was assessed based on reported adverse events (AEs). Urinary copper levels and NCC remained stable across all follow‐ups, indicating adequate copper metabolism control. Reported AEs during TETA 2‐HCl treatment were mostly gastrointestinal discomfort (n = 6). In two patients, progressive elevation of transaminases occurred (despite stable copper metabolism). AEs led to discontinuation of treatment in five cases. Median baseline dose per day was 10.2 mg TETA 4‐HCl/kg bodyweight, whereas median baseline dose after therapeutic switch to TETA 2‐HCl was 12.8 mg/kg bodyweight. Median daily dose at 12 months did not differ significantly from TETA 2‐HCl dose at switching timepoint, with stable biochemical markers and markers of copper metabolism in most (25/30) of the patients. Transitioning from TETA 4‐HCl to TETA 2‐HCl maintained stable copper parameters and liver function in most of analyzed patients. TETA 2‐HCl treatment was generally well tolerated, suggesting that switching medications is safe and effective. In our real‐life cohort, adjustment factor of ~1.25× for the switch of TETA 4‐HCl to TETA 2‐HCl resulted in adequate copper metabolism control.

2023-02-01·Journal of gastroenterology and hepatology

Experience on switching trientine formulations in Wilson disease: Efficacy and safety after initiation of TETA 4HCl as substitute for TETA 2HCl

Article

作者: Morgil, Müzeyyen ; Poujois, Aurelia ; Mohr, Isabelle ; Bourhis, Hélène ; Morvan, Erwan ; Denk, Gerald ; Woimant, France ; Merle, Uta ; Weiss, Karl Heinz ; Obadia, Mickael Alexandre

Abstract:

Background and Aim:

This retrospective, multicenter study aims to assess the efficacy and safety in Wilson disease (WD) patients treated with trientine tetrahydrochloride (TETA 4HCl) after switch from trientine dihydrochloride (TETA 2HCl).

Methods:

In total, 68 WD patients with stable copper metabolism were identified to receive TETA 4HCl (Cuprior™) after previous treatment with TETA 2HCl. We analyzed biochemical markers such as urinary copper, serum copper, non‐coeruloplasmin bound copper (NCC), and transaminases as well as clinical scores (APRI; FIB‐4 score) at baseline with a follow‐up (FU) of 12 months. Safety of TETA 4HCl treatment was based on reported adverse events (AEs).

Results:

The study cohort reflects a common WD cohort with a mean age of 20.3 years at diagnosis and 38.3 years at baseline. There are no significant differences concerning serum copper, NCC, transaminases, APRI, and FIB‐4 score in the 3‐month FU. Six‐month FU revealed a decreased AST (P = 0.008), APRI (P = 0.042), and FIB‐4 score (P = 0.039). GGT varied only borderline significantly in the 3‐month, but not in the 6‐month FU. Comparison of urinary copper within the subsets did not reveal a difference to baseline in all FUs, suggesting stable control of copper metabolism. Few AEs during TETA 4HCl treatment were reported, most commonly gastrointestinal discomfort. Only three treatments with TETA 4HCl were discontinued.

Conclusion:

Copper parameters and liver function were stable after treatment switch to TETA 4HCl. Treatment with TETA 4HCl was generally well tolerated. This study indicates that the switch from TETA 2HCl to TETA 4HCl is safe and viable.

2022-12-01·The lancet. Gastroenterology & hepatology

Trientine tetrahydrochloride versus penicillamine for maintenance therapy in Wilson disease (CHELATE): a randomised, open-label, non-inferiority, phase 3 trial

Article

作者: To, Uyen ; Ala, Aftab ; Dubois, Nicolas ; Obadia, Alexandre ; Lachaux, Alain ; Sandahl, Thomas D ; Czlonkowska, Anna ; Weiss, Karl Heinz ; Belmalih, Abdelouahed ; Denk, Gerald ; Schilsky, Michael L ; Cury, Rubens G ; Piechal, Agnieszka ; Patel, Amar ; Langel, Andrea ; Moore, Joanna ; Litwin, Tomasz ; Cassiman, David ; Gerdes, Lisbet ; Mohr, Isabelle ; Couchonnal-Bedoya, Eduardo ; Monico, Sara ; Barbosa, Egberto Reis ; Poujois, Aurelia ; Freitas, Christian ; Cosgrove, Jeremy ; Boogers, Alexandra ; Ott, Peter ; Twardowschy, Carlos ; Giorgini, Alessia ; Hettiarachchi, Daksshi ; Rahli, Djamila ; D'Hollander, Koenraad ; Zuin, Massimo ; Kamlin, C Omar F ; D'Inca, Renata ; Gondim, Francisco de Assis A ; Skowronska, Marta

BACKGROUND:

Wilson disease is an inherited disorder of copper transport. Whereas penicillamine is used therapeutically to re-establish copper balance, trientine is indicated for patients with penicillamine intolerance. We aimed to compare penicillamine with trientine tetrahydrochloride (TETA4) for maintenance therapy in patients with Wilson disease.

METHODS:

We conducted a randomised, open-label, non-inferiority, phase 3 trial at 15 health-care centres across nine countries (patients were recruited from 13 of these health-care centres across Brazil, Europe, and the USA). We enrolled patients aged 18-75 years with stable Wilson disease who were treated for at least 1 year with penicillamine. Patients entered a 12-week period to determine stability through clinical assessment by site investigators and predefined thresholds for serum non-caeruloplasmin-bound copper (NCC; by an exchangeable copper assay; 25-150 μg/L), 24 h urinary copper excretion (100-900 μg/24 h), and alanine aminotransferase (ALT; <2 × upper limit of normal). Stable patients were randomly assigned (1:1) to continue receiving the maintenance twice daily dose of oral penicillamine or switched mg-for-mg to oral TETA4 centrally with a web-based system using minimisation. The primary endpoint, assessed 24 weeks after randomisation, was NCC by speciation assay. The non-inferiority margin of mean difference in NCC by speciation assay was -50 μg/L, as estimated by a general linear model for repeated visits, adjusted for baseline values. Further data on safety and efficacy were collected during a 24-week extension period. Data were analysed using an intention-to-treat approach. Safety was assessed in all patients who received at least one dose of study treatment. This study is registered with ClinicalTrials.gov, NCT03539952 (active, not recruiting).

FINDINGS:

Between June 4, 2018, and March 10, 2020, 77 patients were screened. 53 patients were randomly assigned (27 to the penicillamine group and 26 to the TETA4 group). After 24 weeks, the mean difference in serum NCC by speciation assay between the penicillamine group and TETA4 group was -9·1 μg/L (95% CI -24·2 to 6·1), with the lower limit of the 95% CI within the defined non-inferiority margin. At 24 weeks, urinary copper excretion was lower with TETA4 than with penicillamine (mean difference 237·5 μg/24 h (99% CI 115·6 to 359·4). At 48 weeks, TETA4 remained non-inferior to penicillamine in terms of NCC by speciation assay (mean difference NCC -15·5 μg/L [95% CI -34·5 to 3·6]). Urinary copper excretion at 48 weeks remained in the expected range for well treated patients in both study groups, and the mean difference (124·8 μg/24 h [99% CI -37·6 to 287·1]) was not significantly different. At 24 weeks and 48 weeks, masked clinical adjudication of stability assessed by three independent clinicians confirmed clinical stability (100%) of all participants, in agreement with the stability seen with the NCC by speciation assay. There were no notable changes in either the Clinical Global Impression of Change or Unified Wilson Disease Rating Scale (neurological assessment) from baseline (pre-randomisation) at weeks 24 and 48. The mean change in serum total copper from baseline to 24 weeks was 17·6 μg/L (99% CI -9·5 to 44·7) with penicillamine and -6·3 μg/L (-34·7 to 22·1) with TETA4, and the mean change in serum total caeruloplasmin from baseline to 24 weeks was 1·8 mg/L (-19·2 to 22·8) with penicillamine and -2·2 mg/L (-6·1 to 1·7) with TETA4. All liver enzymes were similar at 24 weeks and 48 weeks, with the exception of elevated ALT concentration at 48 weeks for patients in the TETA4 group. Penicillamine was associated with three post-randomisation serious adverse events (leukopenia, cholangiocarcinoma, and hepatocellular cancer); none were reported for TETA4. The most common treatment-emergent adverse events were headache for penicillamine (five [19%] of 27 patients vs two [8%] of 26) and abdominal pain for TETA4 (one [4%] vs four [15%]); all treatment-emergent adverse events resolved and were mild to moderate. One patient developed a rash with TETA4 that resolved on discontinuation of therapy.

INTERPRETATION:

The efficacy of TETA4 as oral maintenance therapy was non-inferior to penicillamine and well tolerated in adults with Wilson disease.

FUNDING:

Orphalan.

项与四盐酸曲恩汀相关的新闻（医药）

2024-10-25

·药时代

买下“废弃管线”后，这家Biotech股价暴涨605.4%

买下“废弃管线”后，这家Biotech股价暴涨605.4%。 2024年10月24日，Monopar Therapeutics宣布与阿斯利康旗下罕见病公司Alexion达成合作，获得 ALXN-1840（四硫钼酸双胆碱）的全球独家许可。根据协议，Monopar将以现金加股权的形式向Alexion支付预付款，具体金额未披露。该消息如同巨石，投下了Monopar长期波澜不惊的二级市场，股价暴涨605.4%，报32.67美元/股。 Monopar的股价 ALXN-1840是一种用于治疗威尔逊病的候选药物，该产品并非阿斯利康原研。2018年4月，Alexion以8.55亿美元的金额并购了Wilson，获得WTX101（后更名为ALXN-1840）。 2021年，阿斯利康斥资390亿美元收购Alexion，除了获得C5补体抑制剂依库珠单抗之外，ALXN-1840也在其中。此次ALXN-1840落入Monopar手中，算是三次易主了。威尔逊病（Wilson disease，WD）学名“肝豆状核变性”，是一种常染色体隐性遗传病，因参与“铜代谢”的基因出了问题，使其功能降低或丧失，肝合成铜蓝蛋白（cNCC）速度减慢，胆汁排铜明显减少，最终导致“铜中毒”。因此临床上，患者多表现为肝脏疾病和神经系统疾病的症状，由于缺乏独特的指征，非常考验医生经验，误诊率很高。威尔逊病发病率约在万分之一到3万分之一之间，在小众疾病里属于发病率较高的。因此，尽管是一种罕见的遗传病，但威尔逊病却并非罕见病。为了便于理解，“铜娃娃”的概念于2011年出现，在中国，成为这类患者的代称。目前，关于“铜娃娃”的治疗包括药物治疗与非药物治疗两大类，药物治疗中主要分为铜螯合剂（青霉胺、曲恩汀、二巯基琥珀酸、四硫钼酸铵、甲烷氧化菌素等）、锌剂、靶向分子治疗、姜黄素及其衍生物。而ALXN-1840就是一种铜螯合剂，属于四硫钼酸铵类似物，即硫四硫钼酸铵二胆碱，较四硫钼酸铵更加稳定。在早期研究中，ALXN-1840展示出了极强的铜结合力，比其他的螯合剂强1万倍。 2018年，当ALXN-1840在Wilson手上的时候，就已经启动了3期临床试验。2021年，阿斯利康接手后跟进3期临床试验，达到主要试验终点。结果显示，ALXN-1840在治疗威尔逊病方面优于标准疗法，其清除体内铜的能力是其他药物的三倍。阿斯利康在2022年财报中表示，ALXN-1840将于2023年下半年获批上市。可世事无常，后续两项规模较小的2期机制试验结果不佳，显示铜在体内重新分布而非被有效清除。 2023年5月，也就是阿斯利康2022年财报公布的2个月后，阿斯利康宣布终止ALXN-1840的开发。为什么两项规模较小的2期机制试验结果，可以影响到阿斯利康的最终决策，即使在三期临床结果阳性的情况下。据悉，ALXN-1840的三期临床FoCus研究主要终点为非铜蓝蛋白接合铜（dNCC），即游离铜。但对患者来说，最致命的是那些藏在器官里的铜，游离铜和结合铜只是间接标准。而两项规模较小的2期机制试验目的就是为了证明，ALXN-1840可以清除器官里的铜。样本量都不大，一个9人，一个31人。主要终点为：患者体内每日铜平衡，以及患者治疗48周后肝铜含量的变化。结果不尽人意，阿斯利康无法证明清除器官里的铜。那么，FoCus的研究结果便没有意义。阿斯利康首席执行官 Pascal Soriot在媒体会上表示：“如果我们不能证明铜确实被清除了，那么就存在一种可能性，铜只是被重新分配到了其他组织中，而非排出体外。” 此次，Monopar愿意买下这条“废弃管线”，原因主要包括两点：（1）ALXN-1840处于临床后期，属于成熟资产，这对只有早期管线的Monopar而言，对公司估值有所帮助，股价表现立竿见影。（2）ALXN-1840与Monopar的联合创始人兼首席执行官Chandler Robinson颇有渊源。早在二十年前，他于西北大学求学期间，便参与了ALXN-1840的早期发现工作，并在《科学》杂志上发表了相关成果。据Chris Starr所述，在阿斯利康宣布停止开发后，威尔逊病协会的患者、医生及董事会成员纷纷呼吁重启ALXN-1840。他认为这是一个机会，并且自己也对该药知根知底，买下了说不定有机会。梦想是要有的，万一成功了呢？封面图来源：pixabay 版权声明/免责声明本文为原创文章。本文仅作信息交流之目的，不提供任何商用、医用、投资用建议。文中图片、视频、字体、音乐等素材或为药时代购买的授权正版作品，或来自微信公共图片库，或取自公司官网/网络，部分素材根据CC0协议使用，版权归拥有者，药时代尽力注明来源。如有任何问题，请与我们联系。衷心感谢! 药时代官方网站:www.drugtimes.cn 联系方式: 电话:13651980212 微信:27674131 邮箱:contact@drugtimes.cn 为什么中国创新药企，缺少商业化能力？有救了！30分钟改善老花眼！箕星药业这次算是押中了重要新政！生物制品分段生产试点工作启动点击查看更多精彩！

财报并购临床3期

2024-08-19

·PRNewswire

Behind The Mystery Unveils Treatment in Wilson Disease with Cuviror® by Orphalan

Wilson Disease, a rare, genetic disorder, hampers the body's ability to eliminate copper, leading to serious health issues. Cuvrior® (trientine tetrahydrochloride) is the only FDA-approved treatment in nearly 30 years. FORT LAUDERDALE, Fla., Aug. 19, 2024 /PRNewswire/ -- This special episode of Behind the Mystery features insightful discussions with a dedicated physician, an advocacy leader, and a CEO whose company has pioneered the first FDA approved treatment for Wilson Disease in nearly 30 years. Mandy, diagnosed with Wilson Disease at a young age, shares her journey living with the condition on the show, addressing the challenges faced by patients in managing their treatment regimens and the need for more convenient options. In addition, Hepatologist Dr. Cheung delves into the complexities of Wilson Disease, including its genetic origins, symptoms, and the critical importance of early diagnosis, management, and adherence to treatment. "I'm proud to be part of the 'Behind the Mystery' team, where we use our platform to raise awareness for the rare disease community. Our series underscored the immense challenges faced by patients, as well as the dedication of physicians, the pharmaceutical industry, and advocacy leaders to provide meaningful solutions to the community," said Senior Writer and Producer of Behind the Mystery, Brittany Cocilova. Our latest episode highlights Orphalan's commitment to developing the only FDA-approved therapy for Wilson Disease in nearly 30 years, showcasing the continued efforts to help patients living with and managing their rare disease. Hosts Montel Williams and Olga Villaverde spotlight the Wilson Disease Association's (WDA) efforts to support patients, improve awareness, and facilitate access to developments. Rhonda Rowland, VP of the WDA, discusses the evolution of treatment options and the organization's role in fostering partnerships between patients, physicians, and the pharmaceutical industry. "Orphalan has a tremendous commitment to educating not only the Wilson Disease community and the general public, but also physicians," says Rhonda Rowland, Vice President of Wilson Disease Association. "We've seen a partnership evolve between advocacy and the pharmaceutical industry which enables an open line of communication addressing the unmet needs in our community." Dr. Naseem Amin, CEO of Orphalan, details how Orphalan is addressing unmet needs in the patient community and emphasizes the importance of empathy in the pharmaceutical industry. CUVRIOR®, a treatment marketed by Orphalan, offers a twice-daily dosing and is a non-refrigerated option for long-term maintenance therapy for Wilson Disease. Behind the Mystery - Wilson Disease will air on Lifetime on August 19th and August 27th at 7:30 AM ET/PT and will remain on the website. By sharing these intimate narratives, the series aims to educate the public, inspire empathy, and advocate for better research, treatments, and support systems. INDICATION CUVRIOR is a copper chelator indicated for the treatment of adult patients with stable Wilson Disease who are de-coppered and tolerant to penicillamine. IMPORTANT SAFETY INFORMATION Do not use CUVRIOR if you are allergic to trientine or to any of the ingredients in CUVRIOR. Your Wilson's Disease symptoms could get worse when you start treatment. This could happen because too much copper is removed from the body in a short period of time. Your doctor may need to reduce your dose or stop CUVRIOR treatment. Copper deficiency may develop following treatment with CUVRIOR. Your doctor will do tests to monitor your urine and blood for copper. Iron deficiency may develop while taking CUVRIOR. If this happens, your doctor may tell you to take iron supplements for a limited time. Allergic reactions, such as a rash, have been reported with trientine, the active ingredient in CUVRIOR. If a rash or other allergic reaction occurs, stop taking CUVRIOR and get emergency medical help. The most common side effects are pain in the abdomen, change in bowel habits, rash, hair loss (alopecia), and mood swings. Take CUVRIOR at least 1 hour apart from any other oral medicine. Tell your doctor about all the medicines you're taking, including over-the-counter medicines, vitamins, and herbal supplements. Avoid taking supplements when taking CUVRIOR. Taking CUVRIOR with mineral supplements (e.g., iron, zinc, calcium, magnesium) can reduce the effectiveness of CUVRIOR. If iron supplementation is necessary, take CUVRIOR at least 2 hours before or 2 hours after taking iron; for other mineral supplements (e.g., zinc, calcium, magnesium), take CUVRIOR at least 1 hour before or 2 hours after. Do not start or stop any medicines while taking CUVRIOR without talking to your doctor. These are not all the possible side effects of CUVRIOR. You are encouraged to report negative side effects of prescription drugs. Contact Orphalan at 1-800-961-8320 or [email protected] or FDA at 1-800-FDA-1088 or medwatch. Please see full Prescribing Information at: About Behind the Mystery: Behind the Mystery™ is the first and only television series dedicated to raising awareness about rare and genetic diseases through powerful patient storytelling. Each episode delves into the personal journeys of individuals and families affected by these conditions, highlighting their struggles, triumphs, and daily realities. By sharing these intimate narratives, the series aims to educate the public, inspire empathy, and advocate for better research, treatments, and support systems. Behind the Mystery not only sheds light on the medical aspects of rare diseases but also celebrates the resilience and strength of the human spirit. Join us weekday mornings on Lifetime®. About Orphalan Orphalan is an international orphan drug development and commercialization company headquartered in Paris. Founded in 2011, the company develops innovative therapies for people living with rare and debilitating diseases. Our trientine tetrahydrochloride product for the treatment of Wilson disease is approved and available in more than 20 countries, branded as CUPRIOR® in EU, the UK, Saudi Arabia, Switzerland, Columbia and China, and as CUVRIOR® in the United States. For more information visit and follow us on LinkedIn. SOURCE Behind The Mystery

孤儿药上市批准

2024-08-09

·艾美达医药咨询

01 行业新闻丨北京上海开启创新药加速审批试点 8月2日，国家药监局发布关于同意在北京上海开展优化创新药临床试验审评审批试点的批复：经审核，同意在北京、上海开展优化创新药临床试验审评审批试点。7月31日，国家药监局发布《优化创新药临床试验审评审批试点工作方案》。方案提到，其工作目标是实现30个工作日内完成创新药临床试验申请审评审批，缩短药物临床试验启动用时。不过，纳入试点工作有严格的条件限制。试点项目范围是1类创新药的临床试验申请，细胞和基因治疗产品、疫苗产品等除外。申请人不受区域限制，但需在境内外至少获批过3个创新药临床试验申请，这就将不少药企排除在外. 此外，试点药物临床试验机构原则上为试点区域内的国家医学中心或者国家临床医学研究中心，承担试点项目相关专业已在药物临床试验机构备案管理信息平台备案，且在该专业领域已作为组长单位牵头完成过至少3项创新药临床试验。主要研究者作为组长单位的主要研究者也需主持完成过至少3项创新药临床试验。经确认的试点项目，药审中心会在受理临床试验申请后30个工作日内完成审评审批。审批通过的，申请人与试点机构应于临床试验申请获批后12周内启动临床试验（第一例受试者签署知情同意书），并在临床试验全过程实施风险管理。试点工作为期1年。试点期间，试点区域内至少完成10个品种的临床试验申请审评审批并启动临床试验。（健识局）丨440个药品通过2024年国家医保药品目录初步形式审查 8月7日，国家医保局对2024年国家医保药品目录初步形式审查结果进行公示，2024年7月1日9时至7月14日17时，共收到企业申报信息626份，涉及药品574个。经审核，440个药品通过初步形式审查。与2023年相比，申报药品数量有所增加。此次公示时间截至8月13日，国家医保局将根据公示期间收到的反馈意见，进一步核实相关信息，确定最终通过形式审查的药品名单，向社会公布，并推进专家评审、谈判竞价等后续工作。值得注意的是，肿瘤治疗领域，康方生物的卡度尼利单抗和依沃西单抗两款全球首创双抗药物入选；CAR-T细胞治疗产品方面，本次合源生物的纳基奥仑赛注射液、科济药业的泽沃基奥仑赛注射液、复星凯特的阿基仑赛注射液、药明巨诺的瑞基奥仑赛注射液通过了初审。本次医保目录初审名单中有不少罕见病药物，如盐酸曲恩汀片（肝豆状核变性）、替度格鲁肽（短肠综合征）、醋酸氟氢可的松片（先天性肾上腺皮质增生症和Addison病）等，显示出国家医保局对罕见病患者群体的关注和支持。同时，维拉苷酶α、氯巴占片、盐酸沙丙蝶呤片等纳入国家鼓励研发儿童药目录的药品也均在本次通过的初审名单中。（医药经济报） 02 企业动态丨恒瑞医药夫那奇珠单抗再获批3项临床 8月5日，中国国家药监局药品审评中心（CDE）官网公示，恒瑞医药夫那奇珠单抗注射液获得临床试验默示许可，拟开发治疗适合接受系统治疗或光疗的6至小于18岁儿童和青少年中重度斑块状银屑病患者。夫那奇珠单抗是恒瑞医药自主研发的一种靶向人IL-17A的重组人源化单克隆抗体，此前已经向NMPA递交多项适应症上市申请，包括治疗成人中重度斑块状银屑病适应症。丨海思科1类创新药获批临床 8月6日，CDE官网公示，海思科1类新药HSK44459片获得临床试验默示许可，拟开发治疗间质性肺疾病。根据海思科此前公告介绍，这是其自主研发的一个全新的治疗间质性肺疾病的药物，为一种高选择性的环核苷酸磷酸二酯酶4B（PDE4B）抑制剂。HSK44459片作为一种高选择性的PDE4B抑制剂，可以通过抑制炎性因子的渗出（主要）和成纤维细胞的增殖活化，达到抗炎和抗纤维化的作用。相较于传统泛靶点PDE4抑制剂，选择性PDE4B抑制剂预期在保留抗炎效果的基础上，能减少胃肠道、中枢神经等不良反应。（海思科）丨英矽智能抗肿瘤新药在美国获批临床 8月7日，英矽智能宣布，该公司自主研发的泛TEAD抑制剂ISM6331于2024年7月获得美国FDA的IND批件，用于治疗间皮瘤。这是该产品继今年6月获得针对间皮瘤适应症的孤儿药资格后，再次取得新进展。根据英矽智能新闻稿介绍，此次IND获批使英矽智能获得临床试验批件的创新分子数量达到9个。ISM6331的研发进程在人工智能的帮助下大大提速。临床前研究显示，该产品在多个细胞系中展现出广谱的抗肿瘤效果，在动物模型中低剂量起效，同时具备良好的安全性和ADMET特征。（英矽智能）丨GSK抗PD-1单抗在新加坡获批上市 8月7日，葛兰素史克（GSK）新加坡公司宣布，新加坡卫生科学管理局（HSA）已批准Jemperli（dostarlimab）的一项新适应症，即与卡铂和紫杉醇联合使用，随后Jemperli作为单一药物用于治疗原发性晚期或复发性子宫内膜癌患者，这些患者存在错配修复缺陷（dMMR）或高度微卫星不稳定性高（MSI-H）。此次获批使得Jemperli成为新加坡dMMR/MSI-H原发性晚期或复发性子宫内膜癌的首个一线免疫肿瘤治疗药物，可用于该类患者的早期治疗。Jemperli早前已获得新加坡HSA的批准，作为dMMR复发或晚期子宫内膜癌成人患者的单药治疗，这些患者在任何情况下在先前含铂方案中或之后进展，并且不适合治疗性手术或放疗。（葛兰素史克）丨精准生物CAR-T突破性疗法拟纳入优先审评 8月7日，CDE官网公示，重庆精准生物的普基仑赛注射液（pCAR-19B细胞自体回输制剂）的新药上市申请拟纳入优先审评，用于治疗3～21岁CD19阳性的复发或难治性B细胞急性淋巴细胞白血病患者，有望在中国加速获批上市。普基仑赛注射液用于儿童、青少年B细胞急性淋巴细胞白血病（B-ALL）适应症于2019年在中国获批开展注册临床研究。（精准生物）丨信达生物一款双抗ADC癌症1类新药申报临床 8月9日，CDE官网公示，信达生物1类新药IBI3001新药临床试验申请获得受理。公开资料显示，IBI3001是一款糖基化定点偶联、针对B7-H3和EGFR的双特异性ADC。该产品的临床前研究结果入选了2024年美国癌症研究协会年会Late-Breaking Research（突破性研究）环节。本次为该产品首次在中国申报IND。（信达生物）丨恒瑞医药5款创新药获批临床 8月9日，恒瑞医药发布公告称，该公司有多款创新药获批临床，其中包括多款抗肿瘤新药以及炎症性疾病、自身免疫性疾病新药。分别为：HRS-4508片，一款化药1类新药，为一种新型、高效、选择性的酪氨酸激酶抑制剂，通过抑制肿瘤细胞增殖发挥抗肿瘤作用；SHR-3821注射液，恒瑞医药自主研发的人源化抗体药物，拟用于治疗晚期恶性实体肿瘤，能够通过在肿瘤组织中特异性激活免疫细胞，发挥特异性抗肿瘤作用；SHR-7787注射液，为1类治疗用生物制品，通过诱导激活T细胞，使其发挥靶向杀伤恶性实体肿瘤细胞的作用；SHR-1819注射液获批开展用于结节性痒疹（PN）患者的2/3期临床试验；夫那奇珠单抗注射液获批开展3期临床试验，适应症为用于治疗适合接受系统治疗或光疗的6至小于18岁儿童和青少年中重度斑块状银屑病患者。（恒瑞医药）丨鼎成肽源生物TCR-T细胞产品获批临床 8月9日，CDE官网公示，鼎成肽源生物申报的1类创新药获批临床，拟用于治疗KRAS G12D突变阳性、基因型为HLA-A 11:01的晚期实体瘤患者。根据鼎成肽源生物早先新闻稿介绍，DCTY1102注射液为一款靶向HLA-A*11:01基因型、KRAS G12D突变的TCR-T细胞治疗产品，适应症为晚期胰腺癌、结直肠癌等恶性实体肿瘤。TCR-T类细胞药物的适用人群与HLA基因型密切相关。人类白细胞抗原（HLA）系统是免疫系统重要组成部分，能将抗原肽递呈给T细胞表面的受体（TCR）。HLA-A*11:01为中国人群中表型频率较高的HLA，占比超过20%。8月6日，东北制药集团股份有限公司（东北制药）宣布拟收购鼎成肽源70%的股权。(鼎成肽源生物) 丨石药集团1类CAR-T疗法获批临床 8月9日，CDE官网公示，石药集团中奇制药申报的1类新药SYS6020注射液获批临床，拟用于难治性活动性系统性红斑狼疮。公开资料显示，SYS6020为一款基于mRNA-LNP的嵌合抗原受体（CAR）-T细胞注射液，也是石药集团布局的首个细胞治疗在研产品。随着此次获批临床，它也将成为石药集团首个迈入自身免疫疾病临床开发的细胞疗法在研产品。SYS6020作为一款基于mRNA-LNP的细胞治疗产品，可通过表达可特异性识别BCMA抗原的CAR，进而靶向识别患者体内BCMA阳性的细胞并对其进行杀灭。与传统CAR-T产品相比，SYS6020具有细胞活率高、CAR阳性率高、无基因组整合引起的致瘤风险，以及细胞因子风暴（CRS）等副作用低的优点。（石药集团） *声明：本文不构成任何投资建议，仅出于传递更多信息之目的。 ------------------------------ 「长按」二维码添加小达「进群」与更多行业伙伴共探市场前沿资讯艾美达医药咨询艾美达（北京）医药信息咨询有限公司，成立于2014年4月，是一家专业的医药行业咨询服务提供商。公司致力于将产业政策研究与真实世界的数据挖掘深度结合，洞悉行业政策对市场的影响，通过专业的研究提供前瞻性的市场分析，为企业产品上市后的市场准入提供整体解决方案。

细胞疗法疫苗免疫疗法加速审批临床研究

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
-	-	A16AX	-

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


NCT03539952 (EASL2021)	临床3期	肝豆状核变性维持 non-ceruloplasmin copper;(NCC)	53	D-penicillamine	鹽鹹鬱醖築憲獵廠廠衊(顧鬱醖網夢鬱願鹹淵顧) = 襯蓋遞願窪製鹽繭糧範憲齋憲窪憲襯繭糧構構 (鏇壓鏇淵簾餘齋衊顧醖 )	积极	2021-06-23
				TETA4	鹽鹹鬱醖築憲獵廠廠衊(顧鬱醖網夢鬱願鹹淵顧) = 廠簾簾觸壓鏇願廠鹹窪憲齋憲窪憲襯繭糧構構 (鏇壓鏇淵簾餘齋衊顧醖 )
ESC2017	N/A	肥大型心肌病 Copper	20	Trientine dihydrochloride	鹹壓顧築築糧壓膚積齋(襯醖獵鏇積艱艱鹽網醖) = 觸遞網齋糧網繭憲醖範膚築築齋膚鬱製遞廠餘 (艱範繭網鹹鏇膚糧鹹淵 ) 更多	-	2017-08-28
				trientine	鹹壓顧築築糧壓膚積齋(襯醖獵鏇積艱艱鹽網醖) = 願遞網鏇衊鬱網壓膚簾膚築築齋膚鬱製遞廠餘 (艱範繭網鹹鏇膚糧鹹淵 ) 更多