A Phase 3 Multicenter Study to Evaluate Efficacy, Safety, and Pharmacokinetics of Upadacitinib With Open-Label Induction, Randomized, Double-Blind Maintenance and Open-Label Long-Term Extension in Pediatric Subjects With Moderately to Severely Active Crohn's Disease and Inadequate Response, Intolerance, or Medical Contraindications to Corticosteroids, Immunosuppressants, and/or Biologic Therapy

Crohn's disease (CD) is a long-lasting disease that causes severe inflammation (redness, swelling), in the digestive tract, most often affecting the bowels. It can cause many different symptoms including abdominal pain, diarrhea, tiredness, and weight loss. This study will assess how safe and effective oral Upadacitinib is in treating moderately to severely active Crohn's Disease in pediatric participants aged 2 to 18 years old who have had inadequate response, loss of response, intolerance, or medical contraindications to corticosteroids, immunosuppressants, and/or biologic therapy.
Upadacitinib (RINVOQ) is a drug approved in adults for moderate- to severely active CD and is being developed for moderate- to severely active CD in pediatric participants. This study is conducted in 2 periods: Period 1 is comprised of two phases: a 12-week open-label induction phase which means that the study doctor and participants know that participants will receive UPA Dose-A (or the adult equivalent based on body weight) followed by a 52-week double-blind maintenance phase meaning that neither the participants nor the study doctors will know which dose of upadacitinib will be given(UPA Dose B or Dose C). Period 2 is a 156-week open-label extension of Period 1. Approximately 110 pediatric participants with moderate to severely active CD will be enrolled at approximately 92 sites worldwide.
Participants will receive upadacitinib oral tablets once daily or oral solution twice daily at approximately the same time each day, with or without food. Participants will have a safety follow up for 30 days after discontinuation from any time point within the study.
There may be higher treatment burden for participants in this trial compared to their standard of care (due to study procedures). Participants will attend regular (weekly, monthly) visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.

开始日期2024-07-26

申办/合作机构

AbbVie, Inc.

NCT06461897 / Not yet recruiting临床3期

A Phase 3, Open-label, Efficacy-Assessor-Blinded Study, Comparing the Safety and Efficacy of Upadacitinib to Dupilumab in Children From 2 to Less Than 12 Years of Age With Moderate to Severe Atopic Dermatitis

Atopic dermatitis (AD) is a skin condition that may cause a rash and itching due to inflammation of the skin. Topical therapies applied over the skin may not be enough to control the AD in trial participants who require systemic anti-inflammatory treatment. This study compares upadacitinib to dupilumab in pediatric participants with moderate to severe AD who are candidates for systemic therapy. Adverse events and change in the disease activity will be assessed.
Upadacitinib is an approved drug for treating AD patients aged 12 or older. Participants will receive upadacitinib (given as daily dose) or dupilumab (given at label indicated dose every 2 or 4 weeks). Participants will be stratified depending on disease severity, age and response to previous treatment. There is 1 in 5 chance for participants to receive dupilumab during the randomized cohort. Approximately 675 participants aged 2 to less than 12 years of age will be enrolled in this study at approximately 150 sites worldwide. The study population (As defined by participants age or prior treatment) to be enrolled in the study is dependent on local regulatory requirement and/or agreement.
Participants will receive upadacitinib oral tablets once daily (or oral solution twice a day) for 160 weeks, or dupilumab as per its label for 52 weeks, and followed for 30 days.
There may be higher treatment burden for participants in this trial compared to their standard of care . Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by clinical assessments, blood tests, checking for side effects and completing questionnaires.

开始日期2024-07-26

申办/合作机构

AbbVie, Inc.

NCT06227910 / Not yet recruiting临床3期

A Randomized, Double-Blind, Placebo-Controlled Phase 3b Study to Evaluate the Short- and Long-Term Efficacy and Safety of Dual Targeted Therapy With Intravenous Vedolizumab and Oral Upadacitinib Compared With Intravenous Vedolizumab Monotherapy for the Treatment of Adult Participants With Moderately to Severely Active Crohn's Disease

The main aim of this study is to learn whether vedolizumab and upadacitinib given together (also called dual targeted therapy or DTT) reduces bowel inflammation and ulcers in the bowel compared to vedolizumab only (also called monotherapy) in adults with moderately or severely active Crohn's Disease (CD) after 12 weeks of treatment. Other aims are to learn how safe and effective DTT is compared to monotherapy for these participants.
All participants will receive DTT (either vedolizumab and upadacitinib or vedolizumab and placebo) for 12 weeks. Participants responding to the treatment will then receive vedolizumab only (monotherapy) for an additional 40 weeks.
During the study, participants will visit their study clinic 15 times.

开始日期2024-07-22

申办/合作机构

Takeda Pharmaceutical Co., Ltd.

100 项与乌帕替尼相关的临床结果

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100 项与乌帕替尼相关的转化医学

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100 项与乌帕替尼相关的专利（医药）

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693

项与乌帕替尼相关的文献（医药）

2024-12-31·Journal of medical economics

Cost-effectiveness analysis of upadacitinib as a treatment option for patients with rheumatoid arthritis in the Kingdom of Saudi Arabia

Article

作者: Al-Homood, Ibrahim ; Alsaqa'aby, Mai ; Sharma, Yuvraj ; Alzahrani, Zeyad ; Hamad, Tharwat M ; Anwar, Ali ; Husain, Waleed ; Mohamed, Omneya ; Al-Abdulkarim, Hana ; Al Omari, Bedor ; Jaheen, Ahmed M ; Attar, Suzan M

AIM:

To evaluate cost-effectiveness of upadacitinib (targeted synthetic-disease modifying anti-rheumatic drug [ts-DMARD]) as first-line (1 L) treatment versus current treatment among patients with rheumatoid arthritis (RA) in the Kingdom of Saudi Arabia (KSA), who had an inadequate response to prior conventional-synthetic (csDMARDs) and/or biologic-DMARDs (bDMARDs).

METHODS:

This Excel-based model included patients with moderate (Disease Activity Score [DAS28]: >3.2 to ≤5.1) or severe RA (DAS28 > 5.1). Cost-effectiveness of current treatment (1 L: adalimumab-originator/biosimilar; second-line (2 L): other bDMARDs/tofacitinib) was compared against a new treatment involving two scenarios (1 L: upadacitinib, 2 L: adalimumab-biosimilar [scenario-1]/adalimumab-originator [scenario-2]) for a 10-year time-horizon from societal perspective. Model outcomes included direct and indirect costs, quality-adjusted life-years (QALYs), hospitalization days, number of orthopedic surgeries, and incremental cost-utility ratio (ICUR) per QALY.

RESULTS:

With the current pathway, estimated total societal costs for 100 RA patients over 10-year period were Saudi Riyal (SAR) 50,450,354 (United States dollars [USD] 13,453,428) (moderate RA) and SAR50,013,945 (USD13,337,052) (severe RA). New pathway (scenario-1) showed that in patients with moderate-to-severe RA, upadacitinib led to higher QALY gain (+8.99 and +15.63) at lower societal cost (cost difference: -SAR2,023,522 [-USD539,606] and -SAR3,373,029 [-USD899,474], respectively). Thus, as 1 L, upadacitinib projects "dominant" ICUR per QALY over current pathway. Moreover, in alternate pathway (scenario-2), upadacitinib also projects "dominant" ICUR per QALY for patient with severe RA (QALY gain: +15.63; cost difference: -SAR 164,536 [-USD43,876]). However, moderate RA was associated with additional cost of SAR1,255,696 (USD334,852) for improved QALY (+8.99) over current pathway (ICUR per QALY: SAR139,742 [USD37,264]). Both scenarios resulted in reduced hospitalization days (scenario-1: -14.83 days; scenario-2: -11.41 days) and number of orthopedic surgeries (scenario-1: -8.36; scenario-2: -6.54) for moderate-to-severe RA over the current treatment pathway.

CONCLUSION:

Upadacitinib as 1 L treatment in moderate-to-severe RA can considerably reduce healthcare resource burden in KSA, majorly due to reduced drug administration/monitoring/hospitalization/surgical and indirect costs.

2024-12-31·The Journal of dermatological treatment

Complete response of extensive alopecia areata refractory to baricitinib after five months of treatment with upadacitinib

Article

作者: Velasco-Amador, Juan Pablo ; Prados-Carmona, Álvaro ; De la Torre-Gomar, Francisco Javier ; Ruiz-Villaverde, Ricardo

A case study of 45-yr-old woman presented with a 9-yr history of alopecia areata and atopic dermatitis for evaluation of a new relapse.The authors assessed the response after the patient experienced a relapse of his pathol. while being treated with baricitinib, which supports the importance of selective JAK1 inhibition to improve alopecia areata and suggests the possible efficacy of upadacitinib after loss of response to baricitinib.

2024-12-31·The Journal of dermatological treatment

Upadacitinib for the management of bullous pemphigoid coexisting with psoriasis vulgaris: a case report and literature review

Review

作者: Qin, Qunshi ; Su, Fangying ; Xie, Zhi ; Wang, Tai

Both bullous pemphigoid (BP) and psoriasis are common immune-related dermatological conditions in clinical practice, but the co-occurrence of these two diseases is rare. Currently, there is no consensus on the long-term safe and effective treatment for patients with both BP and psoriasis. JAK inhibitors are emerging as targeted therapeutic drugs that act by inhibiting Janus kinase activity, regulating the JAK/STAT pathway, blocking the transduction pathway of key proinflammatory cytokines, and influencing T-cell differentiation. These cytokines upstream of the JAK/STAT pathway play a pivotal role in the pathogenesis of numerous inflammatory and autoimmune disorders. Upadacitinib, a second-generation JAK inhibitor with high selectivity, demonstrates promising potential.This case report aims to provide a description of the successful treatment of bullous pemphigoid (BP) and psoriasis vulgaris by using upadacitinib, highlighting significant clinical outcomes. Additionally, we aim to analyze the underlying mechanism of upadacitinib in treating these two comorbidities by reviewing relevant literature from both domestic and international sources. Based on our clinical observations, upadacitinib appears to be a promising and well-tolerated therapeutic option for patients with concurrent BP and psoriasis, offering valuable insights for developing appropriate treatment strategies in clinical practice.

607

项与乌帕替尼相关的新闻（医药）

2024-06-24

·生物制品圈

2023年全球畅销药TOP 30专利到期日汇总

如今，许多大型制药企业都正面临着重大的“专利悬崖”，到2030年，这将使其每年面临超过2000亿美元的收入风险。值得注意的是，与过往主要是化学药占主导地位不同，此次许多即将失去市场独占权的药物都是生物药。如艾伯维的抗炎药Humira、默沙东的抗癌药物Keytruda、百时美施贵宝（BMS）的免疫疗法Opdivo、强生的免疫疾病药物Stelara和再生元的眼科治疗药物Eylea等，或是已经专利到期，或是即将在2030年前后结束专利保护。BioPharma Dive汇总了2023年全球销售额排名前30的畅销药物的主要专利到期日期。表1：2023年全球销售额TOP 30畅销药主要专利到期日期在美国授予的专利的标准期限为20年。但实际上，由于制药商通常是在药品上市之前就获得了新药的相关专利，因此药品上市的受保护时间相比标准期限会更短。专利到期意味着仿制药可以进入市场，药物的销售额下降在所难免。根据Evaluate Pharma的数据，就Humira而言，预计该药物销售额将在2024年保留其2022年美国收入的1/3以上，并在2030年之前保持超过20亿美元的收入。图1：美国药品收入与主要专利到期日期后年数的关系，专利到期年份设为0年艾伯维吸取了先前专利悬崖的教训，通过2种新的替代产品——用于牛皮癣和克罗恩病的Skyrizi，以及用于类风湿性关节炎等炎症性疾病的Rinvoq，来抵御Humira销售额下降的预期侵蚀。公司预计到2027年，这两种药物的总收入将达到210亿美元，大约相当于Humira的峰值销售额。此外，利用一些专利策略，制药商也可以延长药物的专利到期时间。对于专利到期的药物，制药企业采取的另一种策略是通过构建“专利丛林（patent thicketing）”来延长仿制药或生物类似物的进入时间。所谓专利丛林，即在围绕药品基本专利层层申请次级专利，形成专利群，不断扩大和延长专利保护的范围和时间，加上药品专利链接制度，使得仿制药公司很难获得仿制药的批准，从而阻碍或延长了仿制药进入市场。这种情况特别容易出现在”重磅炸弹”药（blockbuster drugs）上，如欧委会报告提出的，有的”重磅炸弹”药受到多达100多个产品专利族、1300多个专利和在申请专利的保护，这使得仿制药很难进入市场。如艾伯维的Humira正是采用的这种策略，其在美国拥有数十项专利，将生物仿制药的进入时间在原有的专利期上推迟了七年。其他的如默沙东的Keytruda、强生的多发性骨髓瘤药物Darzalex和BMS的Opdivo，均在试图利用这种策略延长专利期。图2：30款畅销药品的长期专利保护（线条显示了市场垄断的时间跨度，从美国首次获批到目前预期的专利到期日）除了专利丛林外，其他通过专利延长保护期的方法还有：产品跳转（product hopping），即在药品专利即将到期时，对药品进行微小改进并申请专利，形成新一代专利药品，并花大成本进行推广，形成对原专利药的替代，同时在原专利药的仿制药进入市场前将其从市场上撤出，通过这一方式，阻碍仿制药进入市场。专利常青（patent evergreening），专利丛林策略和产品跳转策略相互结合，加上专利药品的各种推广策略，使得专利药品一直受到专利保护，仿制药无法进入相关市场，形成专利常青。通过一个或多个策略的结合，以保持药物的市场独占权。这些策略有时可以避免20年甚至更长时间的仿制药竞争。例如，安进的炎症性疾病药物Enbrel，在2029年关键专利到期时，将享有超过30年的上市保护期。参考资料： [1]Drug patents protect pharma profits. Track when they’ll expire here.By Jonathan Gardner.Published May 8, 2024 • Updated May 13, 2024. [2]Big pharma’s looming threat: a patent cliff of ‘tectonic magnitude’.By Jonathan Gardner.Published Feb. 21, 2023. [3]反垄断系列（二）丨中美欧药品行业反垄断不同情况及其启示.北京市竞天公诚律师事务所.2023-09-12. 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点，不代表本站立场。

专利到期上市批准免疫疗法

2024-06-24

·药智网

2024年“突破性疗法”再创新高，ADC成最靓的“崽”

2012年7月，美国FDA继快速通道、加速批准、优先审评之后，首创了“突破性疗法”的审评工作程序，旨在加快针对严重或危及生命的疾病的药物开发和审查，在创新药产业中含金量很高。 2020年7月，国家药监局药品审评中心（CDE）效仿FDA，中国版的“突破性疗法”正式问世。据CDE数据显示，4年光景中，从南京传奇生物“西达基奥仑赛”成为首款中国“突破性疗法”，用于单药治疗复发或难治性多发性骨髓瘤，到现在已有242款药品获批“突破性疗法”（受理号计）。数据来源：CDE官网并且从趋势上看，纳入突破性疗法的产品数量逐年增加，2023年共计69个受理号被纳入，而以目前2024年获批的数量，或有希望超越去年，再创新高。 2024年纳入“突破性疗法”的新药序号产品药物类型靶点注册申请人公示日期适应症（简要） 1 Tucidinostat HDAC抑制剂 - 微芯生物 5/31/24 结直肠癌 2 格舒瑞昔D-1553 KRAS G12C抑制剂 KRAS 益方生物 5/31/24 胰腺导管腺癌、结直肠癌 3 SHR-A1811 ADC HER2 恒瑞医药盛迪亚生物 5/31/24 1.胆道癌2.胃癌或胃食管结合部腺癌（2024/2/1） 4 BAY-2927088 酪氨酸激酶抑制剂 EGFR exon 20 ; HER2 exon 20 拜耳医药 5/31/24 非小细胞肺癌 5 甲磺酸奥希替尼osimertinib mesylate 选择性EGFR突变抑制剂 EGFR T790M 阿斯利康 5/31/24 非小细胞肺癌 6 BAY 2927088 酪氨酸激酶抑制剂 EGFR exon 20 ; HER2 exon 20 拜耳医药 5/31/24 非小细胞肺癌 7 洛拉替尼 lorlatinib 第三代酪氨酸激酶（ALK/ROS1 TKI）双靶点抑制剂(ALK-TKI) ROS1 ; ALK ; CLIP1-LTK fusion 基石药业 5/31/24 非小细胞肺癌 8 维特立妥组单抗telisotuzumab vedotin ADC MET AbbVie Inc；BSP Pharmaceuticals S.p.A；艾伯维 5/31/24 非小细胞肺癌 9 survodutide 胰高血糖素受体/胰高糖素样肽-1受体（GCGR/GLP-1R）双重激动剂 GLP-1R ; GCGR Zealand Pharma A/S;勃林格殷格翰 5/30/24 脂肪性肝炎（MASH） 10 维迪西妥单抗 disitamab vedotin ADC HER2 荣昌生物 5/22/24 膀胱癌 11 SHRA-1921 ADC TACSTD2 盛迪亚生物恒瑞医药 5/21/24 上皮性卵巢癌、输卵管癌或原发性腹膜癌 12 乌帕替尼 upadacitinib JAK抑制剂 JAK1 AbbVie Inc;艾伯维 5/16/24 非节段型白癜风 13 ssCART-19 CAR-T CD19 优卡迪生物 5/15/24 急性淋巴细胞白血病 14 VIR-3434 单抗 HBsAg 腾盛博药医药;药明生物 5/7/24 HBV/HDV 15 VIR-2218 sirna HBV ; ASGPR 腾盛博药医药 5/7/24 HBV/HDV 16 rocbrutinib - BTK 麓鹏制药 4/28/24 弥漫性大B细胞淋巴瘤 17 IBI-343 ADC CLDN18.2 信达生物 4/25/24 胃/胃食管交界处腺癌 18 BL-B01D1 ADC EGFR ; ERBB3 百利药业 4/9/24 鼻咽癌 19 信迪利单抗 sintilimab 单抗 PD-1 信达生物 3/29/24 结肠癌 20 伊匹木单抗 ipilimumab (单抗)生物类似药 CTLA 信达生物 3/29/24 结肠癌 21 氟唑帕利 fuzuloparib parp 抑制剂 PARP 恒瑞医药 3/25/24 乳腺癌 22 甲磺酸阿帕替尼 apatinib mesylate 酪氨酸激酶抑制剂 VEGFR-2 恒瑞医药 3/25/24 乳腺癌 23 伊那利塞inavolisib PI3Kα抑制剂 PIK3CA F.Hoffmann-La Roche Ltd;罗氏 3/25/24 乳腺癌 24 TRS-005 单抗 CD20 特瑞思药业 3/18/24 弥漫大B细胞淋巴瘤 25 DB-1303 ADC HER2 映恩生物 3/4/24 子宫内膜癌 26 PM8002 双抗 PD-L1 ; VEGF 普米斯生物 3/1/24 复发转移性三阴性乳腺癌 27 sacituzumab tirumotecan ADC TACSTD2 科伦博泰生物 3/1/24 三阴性乳腺癌 28 纳武利尤单抗 nivolumab pd-1抑制剂 PD-1 BMS 3/1/24 结直肠癌 29 伊匹木单抗 ipilimumab 单抗 CTLA BMS 3/1/24 结直肠癌 30 托雷奇单抗 tozorakimab 单抗；白细胞介素 IL33 阿斯利康 2/7/24 病毒性肺部感染 31 司哌博替基 botaretigene sparoparvovec 基因疗法 RPGR Janssen Research & Development, LLC;强生; 2/4/24 视网膜色素变性 32 QX005N 单抗 IL4RA 荃信生物 1/23/24 结节性痒疹 33 VSA-003 siRNA ANGPTL3 Visirna Therapeutics HK Limited;维亚臻生物技术（上海）有限公司; 1/18/24 纯合子型家族性高胆固醇血症 34 甲磺酸伏美替尼 furmonertinib mesilate 酪氨酸激酶抑制剂 EGFR T790M 上海艾力斯医药科技股份有限公司 1/8/24 非小细胞肺癌数据来源：CDE官网突破性疗法的“含金量” 据“突破性疗法”制度建立之初的工作程序内容显示，有足够证据表明具有明显临床优势的创新药或者改良型新药等，可以在Ⅰ、Ⅱ期临床试验阶段，通常不晚于Ⅲ期临床试验开展前申请适用突破性治疗药物程序。原则上获得了“突破性疗法”称谓的药品一方面能获得审批流程上更为密切的指导，也可以让政府与企业间的沟通更为紧密，更利于市场亟待需要的药物快速上市。据历年药品审评报告中数据显示，能够有资格申请突破性疗法的品种已是少数，而能够获批纳入突破性疗法的品种更是凤毛麟角。 2021年需技术审评的9231件注册申请中，共有263个突破性治疗药物程序申请，却仅有53个被纳入了突破性治疗品种，占比分别为2.85%与0.57%； 2022年需技术审评的9301件注册申请中，共有200个突破性治疗药物程序申请，纳入了突破性治疗品种信息仅56个，占比分别为2.15%与0.6%； 2023年需技术审评的13144件注册申请中，共收到286个突破性治疗药物程序申请，仅有70个被纳入了突破性治疗品种，占比分别为2.1%与0.53%；虽说，获批突破性疗法并不等同于获批上市，仅仅只是临床有效性、安全性等数据上有希望超越“前浪”，从历史数量上来看，接近300余款突破性疗法（FDA+CDE）中，能成功上市也只是少数，但这也都妨碍突破性疗法在如今的新药研发产业中的重要地位，仍是目前含金量最高的称谓。资本寒冬下 2024年“突破性疗法”热度不减过去两年，对于创新药而言，是投融资与新药研发热度骤降的时期，彼时异常火爆的赛道也似乎变得门可罗雀，越来越多投资机构不再将目光集中于明星团队、新药故事上，而是选择关注更优秀的临床数据、更差异化的临床靶点，简单来讲就是“更确定性”的疗法。很明显，CDE认定的“突破性疗法”就是最大“确定性”数据库，也是探寻有价值新药项目的最高切入点。截止目前，2024年内CDE共计将39件（34个品种）纳入了突破性疗法，比之去年同期（32件）略有提升，年度突破性疗法数量有望突破70件。而从药品类型上看，在纳入CDE突破性疗法的这34款药物中，其基本涵盖了当下较为热门的创新疗法，如ADC、CAR-T、双抗、基因疗法等。而在数量上，小分子抑制剂数量最多，共有13款药物，占比38.23%；其次是ADC，共计9款药物，占比26.47%；之后是单抗疗法共计7款药物，占比20.58%；最后是siRNA、car-t、基因疗法等则占比较少（如下图）。数据来源：CDE官网并且，统计2021年至今CDE“突破性疗法”中的ADC项目数，逐年上升的趋势之下，2024年的ADC项目数已超过了去年全年ADC项目数，预计2024年将成为ADC领域蓬勃发展的一年（如下图）。数据来源：CDE官网从药品相应的适应症上来看，2024年获批突破性疗法的药品中，抗肿瘤药物的数量优势依旧明显，共计26项目，占比74%，是毫无疑问的热门赛道，而从更细节角度看，突破性疗法中的非小细胞肺癌适应症集中度最高，共有6项，占比17.14%；其次是结直肠癌、乳腺癌等重点领域（如下图）。数据来源：CDE官网而在适应症最集中的非小细胞肺癌领域，则又是中美突破性疗法竞争最激烈的领域，跨国MNC里的拜耳、阿斯利康与艾伯维与国内biotech基石药业、艾力斯双方角力，其中酪氨酸激酶抑制剂是最主要战场，除艾伯维外，其余药企的药物类型均为此。只不过，细分领域上，药企间选择了不同突变类型来形成差异化竞争，如艾力斯甲磺酸伏美替尼突破性疗法适应症是针对EGFR-20外显子插入突变的NSCLC，而阿斯利康的甲磺酸奥希替尼则是(EGFR)外显子19缺失(Ex19del)或外显子21(L858R)置换突变NSCLC。从药企分布上来看，2024年突破性疗法的获批主体与几年有了较大的变化，MNC不再是绝对的主力，反而是越来越多国内biotech企业上榜，以受理号数据为准，恒瑞医药共有4款新药的5个适应症获批突破性疗法，成最大赢家；其次信达生物的伊匹木单抗、信迪利单抗与IBI-343以3款药品上榜紧随其后；剩下的腾盛博药、益方生物、百利药业、科伦博泰、荃信生物等是十数家biotech也有产品获批。数据来源：CDE官网从目前情况来看，我国“突破性疗法”的获批主体已被biotech严格占据，越来越多名不禁传的创新企业用实力证明了自身，联想到近段时间以来的资本寒冬，或许中国创新药事业并非想象中那么贫乏，仍有不少数据优益创新药与实力强劲的创新药企值得关注。恒瑞医药 2024年里，恒瑞医药共有4款药品获批“突破性疗法”，分别是SHR-A1811、SHRA-1921、氟唑帕利与甲磺酸阿帕替尼，前两者是恒瑞医药的两款重磅ADC产品，后两者则是恒瑞最新的两款小分子抗癌抑制剂。而ADC作为恒瑞医药近年来肿瘤领域最为重要的布局，基于其超过10年的ADC研发经验，恒瑞医药打造了模块化HRMAP技术平台，可快速开发各种差异化的ADC产品。截止目前，其管线中已有超过8个的临床阶段ADC产品。 SHR-A1811作为恒瑞医药最重要且进展最快的ADC管线，是一款直接对标于第一三共T-DXd（DS-8201）的HER2 ADC新药，目前其已推进至3期临床，先后6次被CDE纳入突破性治疗品种，分别用于治疗：HER2阳性的复发或转移性乳腺癌、HER2低表达的复发或转移性乳腺癌、HER2突变的晚期非小细胞肺癌、HER2阳性结直肠癌、HER2阳性晚期胃癌或胃食管结合部腺癌、HER2阳性不可切除或转移性胆道癌。而之所以如此殊荣，得益于其新型载荷拓扑异构酶 I抑制剂SHR9265，拥有更强的渗透性的同时，还能提高ADC的旁观者杀伤效应，并且连接处还引进了一个手性环丙基，适当提高其稳定性，降低荷载药物的外周血释放，理论上具有更好的疗效与安全性参数。信达生物 2024年，信达生物共有3款药品获批了“突破性疗法”，分别是IBI-343、信迪利单抗与伊匹木单抗，除伊匹木单抗为生物类似药，前两者均为创新药产品，分别是ADC与单抗产品。与恒瑞医药类似，信达生物同样将ADC作为其新药研发中最主要方向之一，截止目前信达生物共有6款ADC产品进入了临床阶段，IBI-343就是其中进展最快的ADC管线，目前已至临床Ⅲ期阶段，也是全球首款率先启动Ⅲ期临床的 CLDN-18.2 ADC。 IBI-343使用了TOPO1i payload采取定点偶联技术位点特异性将乙二醇与Exatecan偶联，平均药抗比（DAR）为3.6，同时通过Fc Silence突变可以减少非特异性摄取，具备旁观者效应。6月13日，IBI343成功获得美国FDA授予快速通道资格，用于治疗CLDN18.2阳性的晚期胰腺导管腺癌。除上述之外，2024年获批“突破性疗法”的产品还有很多重点创新产品，如正大天晴与益方生物联合开发的D-1553、基石药业的洛拉替尼、荣昌生物的维迪西妥单抗、腾盛博药的VIR-3434、百利药业的BL-B01D1等等，而在ADC领域之外，也有更多如基因疗法、小核酸疗法、car-t疗法等领域值得被重点关注。结语与展望总结而言，能够获批CDE突破性治疗认定的创新药项目，显而易见的在项目质量，尤其是临床数据方面确有优势，很大程度上也确实能影响后续获批上市、对外授权许可交易等商业化预期。以过去FDA的突破性疗法历史，大约可平均缩短2.5年的上市周期，是创新药企争相追求的必然目标。但是，优厚的资源倾斜、越来越多创新药企的集中申请之下，也意味着CDE对突破性治疗药物的筛选或许也将更加严格，更新技术之下，必定要求更真实的“突破”意义。来源 | 博药（药智网获取授权转载）撰稿 | 弎责任编辑 | 八角声明：本文系药智网转载内容，图片、文字版权归原作者所有，转载目的在于传递更多信息，并不代表本平台观点。如涉及作品内容、版权和其它问题，请在本平台留言，我们将在第一时间删除。商务合作 | 王存星 19922864877（同微信）阅读原文，是昨天最受欢迎的文章哦

抗体药物偶联物突破性疗法优先审批免疫疗法细胞疗法

2024-06-21

·drugs

FDA Expands Pediatric Indication for Rinvoq

FRIDAY, June 21, 2024 -- The U.S. Food and Drug Administration has expanded indications for Rinvoq (upadacitinib) to now include pediatric patients (ages 2 years and older) with polyarticular juvenile idiopathic arthritis (pJIA) and psoriatic arthritis (PsA). Rinvoq is indicated for pediatric patients with an inadequate response or intolerance to one or more tumor necrosis factor (TNF) blockers. In addition, Rinvoq LQ, a new weight-based oral solution, is available for these pediatric patients. The expanded indication is based on pharmacokinetic data from 51 patients with pJIA with active polyarthritis and safety data from 83 patients with pJIA with active polyarthritis. At the recommended doses, upadacitinib plasma exposures in pediatric patients with pJIA and PsA are expected to be comparable to those observed in adults with rheumatoid arthritis and PsA based on population pharmacokinetic modeling and simulation. "Pediatric patients with pJIA and PsA can be severely limited in their ability to complete daily physical tasks and participate in everyday activities. Understanding their needs today and knowing the likelihood of disease in adulthood underscores the need for additional treatment options," Aarat Patel, M.D., from the Bon Secours Rheumatology Center of St. Mary’s Hospital in Richmond, Virginia, said in a statement. "Having a treatment option available for patients who do not respond well to a TNF inhibitor addresses a need for the health care community, patients, and their families." Expanded approval of Rinvoq was granted to AbbVie. More Information Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.

上市批准加速审批

100 项与乌帕替尼相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D10994	乌帕替尼	L04AA44	DB15091

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
多关节型幼年特发性关节炎	美国	AbbVie, Inc.	2024-04-26
活动性重度克罗恩病	欧盟	AbbVie Deutschland GmbH & Co. KG	2023-04-24
活动性重度克罗恩病	冰岛	AbbVie Deutschland GmbH & Co. KG	2023-04-24
活动性重度克罗恩病	列支敦士登	AbbVie Deutschland GmbH & Co. KG	2023-04-24
活动性重度克罗恩病	挪威	AbbVie Deutschland GmbH & Co. KG	2023-04-24
克罗恩病	加拿大	AbbVie AS	2020-01-16
强直性脊柱炎	欧盟	AbbVie Deutschland GmbH & Co. KG	2019-12-16
强直性脊柱炎	冰岛	AbbVie Deutschland GmbH & Co. KG	2019-12-16
强直性脊柱炎	列支敦士登	AbbVie Deutschland GmbH & Co. KG	2019-12-16
强直性脊柱炎	挪威	AbbVie Deutschland GmbH & Co. KG	2019-12-16
银屑病关节炎	欧盟	AbbVie Deutschland GmbH & Co. KG	2019-12-16
银屑病关节炎	冰岛	AbbVie Deutschland GmbH & Co. KG	2019-12-16
银屑病关节炎	列支敦士登	AbbVie Deutschland GmbH & Co. KG	2019-12-16
银屑病关节炎	挪威	AbbVie Deutschland GmbH & Co. KG	2019-12-16
中轴性脊柱关节炎	欧盟	AbbVie Deutschland GmbH & Co. KG	2019-12-16
中轴性脊柱关节炎	冰岛	AbbVie Deutschland GmbH & Co. KG	2019-12-16
中轴性脊柱关节炎	列支敦士登	AbbVie Deutschland GmbH & Co. KG	2019-12-16
中轴性脊柱关节炎	挪威	AbbVie Deutschland GmbH & Co. KG	2019-12-16
溃疡性结肠炎	欧盟	AbbVie Deutschland GmbH & Co. KG	2019-12-16
溃疡性结肠炎	冰岛	AbbVie Deutschland GmbH & Co. KG	2019-12-16

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
睡眠异常	临床3期	-	AbbVie, Inc.	2024-05-23
非节段型白癜风	临床3期	美国	AbbVie, Inc.	2023-12-19
非节段型白癜风	临床3期	日本	AbbVie, Inc.	2023-12-19
非节段型白癜风	临床3期	阿根廷	AbbVie, Inc.	2023-12-19
非节段型白癜风	临床3期	比利时	AbbVie, Inc.	2023-12-19
非节段型白癜风	临床3期	保加利亚	AbbVie, Inc.	2023-12-19
非节段型白癜风	临床3期	加拿大	AbbVie, Inc.	2023-12-19
非节段型白癜风	临床3期	智利	AbbVie, Inc.	2023-12-19
非节段型白癜风	临床3期	法国	AbbVie, Inc.	2023-12-19
非节段型白癜风	临床3期	德国	AbbVie, Inc.	2023-12-19

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适应症

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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


NCT02706847 (RA-V, FDA_CDER) 人工标引	临床3期	类风湿关节炎	499	RINVOQ (Background cDMARDs)	鬱鹽鬱衊顧鏇選簾築醖(鹹網蓋構構夢網鏇築艱) = 網齋願憲夢夢膚壓製繭繭觸艱簾範艱醖醖艱觸 (範窪醖鏇襯窪艱觸範膚 ) 达到更多	积极	2024-04-26
NCT03725007 (FDA_CDER) 人工标引	临床1期	多关节型幼年特发性关节炎	83	RINVOQ LQ	-	积极	2024-04-26
NCT02819635 (UC-3, FDA_CDER) 人工标引	临床2/3期	溃疡性结肠炎	451	RINVOQ 15 mg Once Daily	範觸網積齋蓋顧醖襯願(鹹淵膚簾淵艱簾廠鹽構) = 膚夢鏇淵廠鹹製齋網觸襯醖築淵壓餘衊鏇襯齋 (憲顧積憲醖蓋衊範製獵 ) 更多	积极	2024-04-26
NCT02819635 (UC-3, FDA_CDER) 人工标引	临床2/3期	溃疡性结肠炎	451	RINVOQ 30 mg Once Daily	範觸網積齋蓋顧醖襯願(鹹淵膚簾淵艱簾廠鹽構) = 積願醖願鬱獵齋襯獵膚襯醖築淵壓餘衊鏇襯齋 (憲顧積憲醖蓋衊範製獵 ) 更多	积极	2024-04-26
NCT03345836 (CD-1, FDA_CDER) 人工标引	临床3期	克罗恩病	857	RINVOQ 45 mg Once Daily	範鑰艱淵艱觸膚獵艱窪(鑰蓋襯構壓憲蓋積構餘) = 膚醖憲夢獵蓋憲壓齋選繭鬱醖網窪繭獵鑰鑰構 (齋願顧遞齋鏇築襯鑰鹽 ) 更多	积极	2024-04-26
NCT03568318 \| NCT03569293 \| NCT03607422 (AD-3, FDA_CDER) 人工标引	临床3期	特应性皮炎	2,584	RINVOQ 15 mg (Trial AD-1)	膚夢製壓膚遞鬱糧鏇衊(範廠鹹積壓鏇網夢餘顧) = 糧選壓鹹鑰製積淵遞壓選壓繭襯夢鑰衊積衊顧 (齋艱鹽衊觸構憲夢鏇襯 ) 更多	积极	2024-04-26
	临床3期	特应性皮炎	2,584	RINVOQ 30 mg (Trial AD-1)	膚夢製壓膚遞鬱糧鏇衊(範廠鹹積壓鏇網夢餘顧) = 簾衊鏇鏇顧鏇廠觸壓鑰選壓繭襯夢鑰衊積衊顧 (齋艱鹽衊觸構憲夢鏇襯 ) 更多	积极	2024-04-26
NCT03104374 (SELECT - PsA 2, FDA_CDER) 人工标引	临床3期	银屑病关节炎	642	RINVOQ (bDMARD-IR)	願醖積繭築齋蓋夢鑰簾(顧鏇構醖範簾鬱醖網齋) = 獵艱鹹鹽膚構鑰鏇襯獵餘壓鹽顧淵窪艱齋製鏇 (鏇鹹壓衊醖獵製窪鹽廠 ) 更多	积极	2024-04-26
NCT02675426 (SELECT-NEXT, FDA_CDER) 人工标引	临床3期	类风湿关节炎	661	RINVOQ (Background cDMARDs)	餘製範襯鏇糧獵艱願夢(鏇築窪簾築艱襯觸憲製) = 網鏇襯繭顧鏇醖鏇齋製簾廠夢選艱壓齋艱鑰膚 (鹹積遞選積鏇鹹鹹願鏇 ) 达到更多	积极	2024-04-26
NCT03345823 (CD-3, FDA_CDER) 人工标引	临床3期	克罗恩病	343	RINVOQ 15 mg Once Daily	襯遞壓觸築窪蓋築製鹹(觸鏇窪襯範簾壓鹽鬱蓋) = 艱繭繭製遞襯鹽製鹹觸積鏇膚糧選範獵鹽艱鑰 (積鹹淵簾製齋鑰襯觸鬱 ) 更多	积极	2024-04-26
NCT03345823 (CD-3, FDA_CDER) 人工标引	临床3期	克罗恩病	343	RINVOQ 30 mg Once Daily	襯遞壓觸築窪蓋築製鹹(觸鏇窪襯範簾壓鹽鬱蓋) = 選網衊觸鏇獵憲築觸獵積鏇膚糧選範獵鹽艱鑰 (積鹹淵簾製齋鑰襯觸鬱 ) 更多	积极	2024-04-26
NCT04169373 (nr-axSpA, FDA_CDER) 人工标引	临床3期	非放射性轴性脊柱关节炎	314	RINVOQ 15 mg	鏇糧構願蓋齋選窪醖鹹(網蓋積憲醖選製衊遞廠) = 餘獵餘築簾簾齋遞壓蓋膚齋膚築簾齋鏇齋廠願 (築夢糧顧衊餘鹹蓋願膚 ) 达到更多	积极	2024-04-26
NCT03104400 (SELECT - PsA 1, FDA_CDER) 人工标引	临床3期	银屑病关节炎	1,705	RINVOQ (non-biologic DMARD-IR)	襯繭餘遞鹽壓衊衊廠餘(鏇積鏇簾廠選鑰鹽壓廠) = 觸獵鹽鬱築獵糧範膚艱鏇鏇製鹽壓齋淵願網鬱 (鹽顧觸憲衊願鏇範鏇築 ) 更多	积极	2024-04-26