主要：确定第6周的血小板反应率，证明阿伐曲泊帕对中国成人慢性ITP患者的疗效优于安慰剂。次要：根据所测量的第8天的血小板反应，证明阿伐曲泊帕优效于安慰剂。根据6周内所测定血小板反应率累积周数（定义为：在无补救治疗情况下，血小板计数≥50×109/L的累计周数），证明阿伐曲泊帕优效于安慰剂。评估阿伐曲泊帕在减少出血和补救治疗方面的疗效优于安慰剂。评估阿伐曲泊帕长期治疗的有效性。评估阿伐曲泊帕安全性和耐受性。

开始日期2021-03-02

申办/合作机构

Kawashima Group Co., Ltd.

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项与 Dova Pharmaceuticals, Inc. 相关的文献（医药）

2022-05-01·British journal of haematology2区 · 医学

Adults with immune thrombocytopenia who switched to avatrombopag following prior treatment with eltrombopag or romiplostim: A multicentre US study

2区 · 医学

Article

作者: Liebman, Howard ; Vredenburg, Michael ; Jiang, Debbie ; Gernsheimer, Terry ; Cuker, Adam ; Wojdyla, Matthew ; Al‐Samkari, Hanny ; Lee, Susie

Summary:

Patients with immune thrombocytopenia (ITP) may respond to one thrombopoietin receptor agonist (TPO‐RA) but not another. Limited data are available describing outcomes in patients who switched from romiplostim or eltrombopag to avatrombopag, a newer oral TPO‐RA. We performed a retrospective observational study of adults with ITP who switched from eltrombopag or romiplostim to avatrombopag at four US tertiary ITP referral centres. Forty‐four patients were included, with a mean ITP duration of 8.3 years and a median (range) of four prior ITP treatments. On avatrombopag, 41/44 patients (93%) achieved a platelet response (≥50 × 109/l) and 38/44 patients (86%) achieved a complete response (≥100 × 109/l). In all patients, the median platelet count on eltrombopag or romiplostim was 45 × 109/l vs 114 × 109/l on avatrombopag (p < 0.0001); in patients switched for ineffectiveness of romiplostim/eltrombopag, it was 28 × 109/l on romiplostim/eltrombopag vs 88 × 109/l on avatrombopag (p = 0.025). Fifty‐seven percent of patients receiving concomitant ITP medications before switching discontinued them after switching, including 63% of patients receiving chronic corticosteroids. In a heavily pretreated chronic ITP population, avatrombopag was effective following therapy with romiplostim or eltrombopag, with high response rates even in patients with inadequate response to a prior TPO‐RA.

2022-01-01·Therapeutic advances in gastroenterology

Avatrombopag ethnic sensitivity analysis in chronic liver disease and thrombocytopenia patients: individual-level pooled analysis

Article

作者: Hui, Ai-Min ; Jamieson, Brian D. ; Lu, Jun

Introduction::

Few data have been published on the ethnic sensitivity of effectiveness, pharmacokinetics (PK), and pharmacodynamics (PD) of avatrombopag for the management of thrombocytopenia in patients with chronic liver disease (CLD).

Methods::

An ethnic sensitivity analysis was performed based on the results from two phase III studies (ADAPT-1 and ADAPT-2), with a primary endpoint of the proportion of patients without the requirement of platelet transfusion or rescue treatment for bleeding after randomization to 7 days following a scheduled procedure, and three phase I studies in healthy subjects. Cochran–Mantel–Haenszel and Fisher’s exact tests were used to compare the differences in effectiveness in different ethnicities and overall population.

Results::

In total, 435 patients (placebo, n = 158; avatrombopag, n = 277) were stratified into various ethnic groups: 121 East Asians, including the subgroup of 27 Chinese, and 259 Caucasians. The proportion of patients who did not receive a platelet transfusion and those with a platelet count ⩾50 × 109/L in the avatrombopag 40 and 60 mg groups were higher than that of placebo for all ethnicities and in the overall population. Statistical significance was obtained in the overall population and for all ethnicities other than Chinese patients, a group with a very small sample size. No significant difference was observed in the proportion of responders in each ethnic group compared to overall population ( p > 0.05). The incidence of adverse events in East Asians was similar to that in both Caucasians and the overall population.

Conclusion::

Avatrombopag was effective and safe in the management of thrombocytopenia in Chinese patients with CLD. Ethnicity does not appear to influence the efficacy, safety, PK, or PD of avatrombopag.

2021-06-01·Advances in therapy3区 · 医学

Efficacy and Safety of Avatrombopag in Patients with Chronic Immune Thrombocytopenia: A Systematic Literature Review and Network Meta-Analysis

3区 · 医学

Review

作者: Vredenburg, Michael ; Wojciechowski, Piotr ; Wilson, Koo ; Pochopien, Michał ; McCrae, Keith R ; Smela, Beata ; Jurczak, Wojciech ; Nazir, Jameel ; Pustułka, Iwona ; Tytuła, Anna

INTRODUCTION:

A network meta-analysis (NMA) was performed to assess the efficacy and safety of avatrombopag, relative to eltrombopag, romiplostim, and fostamatinib, for patients with chronic immune thrombocytopenia (ITP) not responding adequately to corticosteroids.

METHODS:

A systematic search of publication and clinical trial databases was conducted to identify relevant randomized controlled trials (RCTs) and observational studies. Data from eligible studies were extracted and analyzed in a Bayesian framework using relative effect sizes vs placebo. Outcomes included durable platelet response; need for rescue therapy; reduction in use of concomitant ITP medication; incidence of any or World Health Organization (WHO) grade 2-4 bleeding events, and any adverse events. Results were reported as odds ratios or incidence rate ratios (IRR) with 95% credible intervals (CrIs).

RESULTS:

The NMA included seven phase 3 RCTs. Compared with placebo, avatrombopag was associated with statistically significant improvements in durable platelet response, reduction in use of concomitant ITP medication, and incidence of any bleeding events. Statistically significant differences vs placebo were also observed for durable platelet response and need for rescue therapy (eltrombopag, romiplostim, and fostamatinib); reduction in use of concomitant ITP medication (eltrombopag and romiplostim); incidence of any bleeding events (fostamatinib); and incidence of WHO grade 2-4 bleeding events (romiplostim and fostamatinib). No statistically significant differences were observed for any adverse events. Avatrombopag was associated with a statistically significant lower incidence of any bleeding events vs eltrombopag (IRR 0.38 [95% CrI 0.19, 0.75]) and romiplostim (IRR 0.38 [95% Crl 0.17, 0.86]); no other between-treatment differences were observed.

CONCLUSION:

In this NMA, avatrombopag significantly increased the chance of achieving durable platelet response and reducing the use of concomitant ITP medication vs placebo, and significantly reduced the incidence of any bleeding events compared with placebo, eltrombopag, and romiplostim. The study aims to help guide clinicians managing patients with chronic ITP and insufficient response to previous treatment.

项与 Dova Pharmaceuticals, Inc. 相关的新闻（医药）

2026-09-03

·医药专利

赛诺菲利扎布替尼获批，首个BTK抑制剂攻入ITP赛道，2026年中国第67款新药

1新药定义为含中国首次上市的活性成分，含原研在国外已批准在申报进中国的化药5.1类和生物药3.1类，不含非原研仿制或进口的化药3类、生物药2类 67 利扎布替尼片（2026.9.3）免疫性血小板减少症（ITP） 2026年9月3日，国家药品监督管理局批准Genzyme Corporation（赛诺菲子公司）申报的1类创新药利扎布替尼片（商品名：赛立止®）上市，用于既往对糖皮质激素、免疫球蛋白等治疗反应不佳或不耐受的持续性或慢性原发免疫性血小板减少症（ITP）成人患者。利扎布替尼是全球首个获批用于ITP的BTK抑制剂，也是继2025年8月FDA批准、2025年12月欧盟批准、2026年5月英国批准、2026年6月日本批准后，该药在中国市场的正式落地。从2020年赛诺菲以36.8亿美元收购Principia Biopharma囊获该药，到2026年全球五大市场全面获批，利扎布替尼走了一条典型的"原研收购→全球开发→多国同步上市"路径。一、药品基本信息项目内容药品名称利扎布替尼片（赛立止®）英文名/代号 Rilzabrutinib / PRN1008 / Wayrilz® 注册分类化药1类（进口原研）靶点/机制口服可逆共价BTK抑制剂适应症持续性或慢性ITP，既往激素/免疫球蛋白治疗失败用法用量 400mg 每日两次口服原研/申报企业 Genzyme Corporation（赛诺菲）技术来源 Principia Biopharma（2020年赛诺菲36.8亿美元收购）全球首批美国FDA 2025年8月中国获批时间 2026年9月3日二、ITP：被忽视的"血小板吞噬"疾病原发免疫性血小板减少症（ITP）是一种获得性自身免疫性疾病，核心病理是机体免疫系统异常攻击自身血小板，导致血小板被巨噬细胞加速吞噬破坏，同时骨髓生成血小板的能力也受到抑制。患者血小板计数常低于100×10⁹/L，严重者低于30×10⁹/L甚至个位数，表现为皮肤瘀斑、牙龈出血、鼻出血，严重时可发生颅内出血等致命性并发症。 ITP并非罕见病。2025年中国约有21.64万名ITP患者，中国ITP药品市场规模约9亿美元，预计2035年将增长至约36亿美元。患者中位病程长达6-8年，46%的患者曾接受过5种以上治疗方案，28%接受过脾切除——这些数字说明ITP是一种亟需新机制的难治性疾病。目前ITP的标准治疗路径为：一线使用糖皮质激素或静脉注射免疫球蛋白（IVIG）快速提升血小板；二线及以后使用血小板生成素受体激动剂（TPO-RA，包括艾曲泊帕、海曲泊帕、阿伐曲泊帕、罗普司亭等）、利妥昔单抗或脾切除。但即使经过多线治疗，仍有相当比例的患者血小板计数无法达标，或出现严重副作用（激素长期使用、肝毒性、血栓风险），存在明确的未满足临床需求。三、作用机制：从TPO-R到BTK，ITP治疗机制的范式转换利扎布替尼是一种口服、可逆、共价BTK抑制剂，但其与传统的BTK抑制剂（如伊布替尼、阿卡替尼）有本质区别——高选择性可逆共价结合，而非不可逆共价结合，降低了脱靶效应和毒性风险。 BTK（布鲁顿酪氨酸激酶）在B细胞发育、抗体产生、巨噬细胞Fcγ受体信号传导、NLRP3炎症小体活化等多个免疫通路中发挥核心作用。在ITP中，利扎布替尼通过三条路径发挥作用：①减少B细胞产生抗血小板自身抗体；②抑制巨噬细胞通过Fcγ受体介导的血小板吞噬；③调节天然免疫细胞炎症反应。这种"多免疫调节"（multi-immune modulation）机制，使其区别于仅作用于血小板生成的TPO-RA类药物。 Mechanism of action of rilzabrutinib. Rilzabrutinib, a selective and reversible Bruton tyrosine kinase (BTK) inhibitor, modulates immune responses through two key mechanisms. (Left panel) In B cells, Rilzabrutinib inhibits BTK activation downstream of the B-cell receptor (BCR), thereby reducing anti-platelet autoantibody production. (Right panel) In macrophages, Rilzabrutinib blocks Fcγ receptor (FcγR)-mediated signaling by preventing BTK activation, thereby inhibiting the phagocytosis of antibody-coated platelets. Unlike irreversible BTK inhibitors, Rilzabrutinib binds non-covalently to BTK, preserving platelet function and reducing off-target effects. 从治疗逻辑来看，TPO-RA是"多造血小板"，利扎布替尼是"少毁血小板"——一个从源头减少免疫破坏，是机制层面的治疗范式转换。四、临床数据：LUNA3研究，23% vs 0%的持久血小板应答利扎布替尼的获批基于关键性III期LUNA3研究（NCT04562766），这是一项多中心、随机、双盲、安慰剂对照试验，纳入202例既往经治的持续性或慢性ITP成人患者，按2:1随机分配至利扎布替尼400mg BID组或安慰剂组。患者基线中位血小板计数仅15×10⁹/L，中位病程6-8年，46%接受过5种以上既往治疗，28%曾接受脾切除——这是典型的"难治性ITP"人群。指标利扎布替尼安慰剂第25周持久血小板应答率（主要终点）23% 0% 中位起效时间36天 — ≥2级出血事件4% 12% 身体疲劳改善显著改善（含非持久应答者） — 长期扩展期（LTE）持续血小板应答，长期安全性良好（EHA 2026） LUNA3研究的主要终点——第25周持久血小板应答率（定义为连续至少6周血小板计数≥50×10⁹/L）——利扎布替尼组23% vs 安慰剂组0%，差异具有统计学意义（p<0.0001）。值得注意的是，即使在未达到持久应答的患者中，也观察到了出血事件减少和身体疲劳改善，提示利扎布替尼的治疗获益可能超越血小板计数本身。 2026年6月欧洲血液学协会（EHA）年会上公布的长期扩展期（LTE）数据显示，延长治疗可维持持续的血小板应答，且安全性良好。该研究结果于2025年发表于《Blood》期刊。五、竞争格局：BTK入局，ITP赛道加速分化 ITP治疗药物市场正在经历从"TPO-RA一统天下"到"多机制分化"的格局演变。目前中国ITP治疗市场的主要参与者如下：（一）已获批药物概览药物类别代表药物企业中国获批时间特点 TPO-RA 艾曲泊帕（瑞弗兰®）诺华 2017年全球首个口服TPO-RA，肝毒性需监测海曲泊帕（恒曲®）恒瑞医药 2021年6月国产首个1类创新TPO-RA，获中国专利金奖阿伐曲泊帕（苏可欣®）复星医药/AkaRx 2024年（ITP适应症） CLD+ITP双适应症，无食物限制罗普司亭（注射用）齐鲁制药 2024年长效TPO-RA，每周一次皮下注射BTK抑制剂利扎布替尼（赛立止®）赛诺菲2026年9月全球首个ITP BTKi，多免疫调节机制抗CD20 利妥昔单抗（美罗华®）罗氏超适应症使用 B细胞清除，起效慢但持久（二）在研ITP药物：BTK之外的新战场利扎布替尼的获批打开了BTK抑制剂治疗ITP的大门，但后续竞争者已迅速跟进。邦顺制药的CX1440（BTK抑制剂）已启动ITP III期临床试验，预计2027年第四季度提交NDA。此外，FcRn抑制剂（efgartigimod、rozanolixizumab）、BAFF-R抑制剂（ianalumab，诺华VAYHIT2研究已达主要终点）、抗CD38（mezagitamab，武田）等全新机制药物也在全球范围内推进ITP临床开发。 ITP赛道正从"TPO-RA主导"走向"TPO-RA + BTK抑制剂 + FcRn抑制剂 + 抗CD38"多机制并存的格局。对于中国的ITP患者而言，从2021年仅有海曲泊帕等TPO-RA可选，到2026年有了BTK抑制剂这一全新机制武器，治疗选择发生了质的变化。六、36.8亿美元收购：Principia的"两弹一星" 利扎布替尼的故事，本质上是赛诺菲2020年以36.8亿美元收购Principia Biopharma的缩影。这笔收购为赛诺菲带来了两款BTK抑制剂：利扎布替尼（rilzabrutinib，ITP）和托莱布替尼（tolebrutinib，多发性硬化症）。其中托莱布替尼已于2026年在欧盟获批用于非复发性继发进展型多发性硬化症（nrSPMS），形成"一石二鸟"的BTK抑制剂管线。 Principia的核心技术平台是"TAILORED COVALENCY®"——可逆共价结合技术，在保留共价抑制剂持久药效的同时，通过可逆设计降低脱靶毒性。这一技术路线使利扎布替尼在BTK抑制剂中脱颖而出：传统BTK抑制剂（如伊布替尼、阿卡替尼）均为不可逆共价结合，而利扎布替尼的可逆共价设计使其在ITP这一非肿瘤适应症中展现出更好的安全性。对于赛诺菲而言，利扎布替尼的获批具有双重战略意义：一是填补了其在血液免疫领域的空白，二是验证了"收购biotech技术平台→全球开发→多适应症拓展"的BD模式。在ITP之外，利扎布替尼还在IgG4相关疾病、慢性荨麻疹等适应症中探索，拓展空间值得关注。 2026中国获批新药链接 66.云顶艾曲帕米鼻喷获批，30年首款PSVT居家治疗新药，2026年中国第66款新药 65.丽珠莱康奇塔单抗获批，头对头击败可善挺，2026年中国第65款新药 64.智翔金泰纬利妥米单抗获批，2026年中国第64款新药 63.同源康氘申艾替尼获批，击败奥希替尼，2026年中国第63款新药 62.和黄医药凡瑞格拉替尼获批，2026年中国第62款新药 61.强生口服IL-23R多肽Icotyde获批，2026年中国第61款新药 60.三生国健IL-1β单抗益赛那获批，2026年中国第60款新药 59.恒瑞立康可泮获批，国产第3款CFB的"头对头"底气，2026年中国第59款新药 58.天境生物CD38单抗菲泽妥单抗获批，2026年中国第58款新药 57.药捷安康胆管癌新药替恩戈替尼获批，2026年中国第57款新药免责声明：本报告仅供参考，不构成投资建议© 2026 医药专利版权所有欢迎加入星球VIP群完整版（含数据表格）已同步至知识星球 📌 关注"医药专利"，获取最新医药资讯和评论洞见

2026-08-08

·亮元财经英语

空缺6个月，BioNTech终于敲定CEO

One of the most closely-watched vacancies in biopharma has now been filled, with Sobi CEO Guido Oelkers, Ph.D., moving across to take up the top job at mRNA pioneer BioNTech. 据FierceBiotech报道，生物制药行业最受关注的高管空缺之一现已填补---Sobi公司CEO吉多·奥尔克斯博士将转任mRNA先驱BioNTech公司的最高职位。 It’s been a six-month wait to find out who would head up the German company, after CEO Uğur Şahin, M.D., and his co-founder, chief medical officer and wife Özlem Türeci, M.D., announced in March that they will be heading off to launch a new company focused on “next-generation mRNA innovations.” 今年3月，Biotech公司CEO乌古尔·沙欣医学博士与其妻、联合创始人兼首席医学官厄兹勒姆·图雷西医学博士宣布，他们将离职创办一家专注于“下一代mRNA创新”的新公司。此后，外界一直在关注谁将执掌这家德国企业，而这一任命结束了长达六个月的等待。 After its founding in 2008, BioNTech rose to prominence and financial success thanks to its partnership with Pfizer for the blockbuster COVID-19 vaccine Comirnaty. BioNTech成立于2008年，得益于与辉瑞合作开发的重磅新冠疫苗复必泰(Comirnaty)而声名鹊起，并取得了财务成功。 More recently, BioNTech’s strategy to become a “fully integrated immunotherapy powerhouse” has seen the company gear up to push for the approval of an HER2-targeted antibody-drug conjugate called trastuzumab pamirtecan, which is licensed from China’s DualityBio. 近年来，BioNTech的战略是成为“全面整合的免疫疗法巨头”，公司正积极推动一款HER2靶向抗体药物偶联物（ADC）trastuzumab pamirtecan的获批，该药授权自中国的映恩生物。 BioNTech has already been touting 2026 as a “catalyst-rich year” that will see further phase 3 oncology readouts from the likes of trastuzumab pamirtecan and an OncoC4-partnered anti-CTLA-4 antibody called gotistobart. BioNTech早已将2026年视为“充满机遇的一年”：届时，诸如曲妥珠单抗帕米瑞替康这类药物在肿瘤治疗领域的第三阶段试验结果将会出炉，同时与OncoC4公司合作开发的抗CTLA-4抗体gotistobart也会取得相关试验数据。 The Mainz, Germany-based company recently touted “encouraging anti-tumor activity” from its Bristol Myers Squibb-partnered PD-L1xVEGF-A bispecific immunomodulator pumitamig. 总部位于德国美因茨的Biotech还表示，其与百时美施贵宝共同研发的PD-L1xVEGF-A双特异性免疫调节剂pumitamig也展现出了令人鼓舞的抗肿瘤效果。 This is the situation that Oelkers will be inheriting. As CEO of Swedish pharma Sobi since 2017, Oelkers has overseen the licensing of the respiratory syncytial virus therapy Synagis from AstraZeneca, the $915 million acquisition of Dova Pharmaceuticals for the thrombocytopenia medicine Doptelet and the launch of another bleeding disorder drug, Altuviiio, in partnership with Sanofi. 这就是奥尔克斯博士即将接手的局面。自2017年起担任瑞典制药公司Sobi的CEO期间，他主导了多项重要交易：从阿斯利康获得呼吸道合胞病毒疗法Synagis的授权，以9.15亿美元收购Dova Pharmaceuticals以获得血小板减少症药物Doptelet，以及与赛诺菲合作推出另一款出血性疾病药物Altuviiio。 In this morning’s release, BioNTech praised Oelkers for his “strong track record in transforming and scaling global organizations, driving sustainable growth through disciplined execution, focused capital allocation, and operational excellence.” 在今天上午的公告中，BioNTech称赞奥尔克斯“在转型和扩展全球组织、通过严格执行、专注的资本配置和卓越运营推动可持续增长方面拥有出色的履历”。 “I have come to know Guido Oelkers as a leader who combines a deep understanding of the pharmaceutical industry and strong strategic acumen with genuine respect for the culture and people of our organization,” outgoing BioNTech CEO Sahin said in the release. “With Guido Oelkers taking over, I feel confident that BioNTech is in a strong position to realize its vision.” 在新闻稿中，即将离任的Biotech公司CEO沙欣表示，“我认识的吉多·奥尔克斯是一位兼具深厚制药行业洞察力、强大战略眼光，并真正尊重我们组织文化和员工的领导者。由他接任公司CEO，我相信BioNTech一定能够实现自己的愿景。” Oelkers, who is due to take up his new post by February 1, 2027 “at the latest,” said BioNTech “stands out through its scientific foundation and particularly its strong late-stage oncology pipeline.” “最迟”将于2027年2月1日履新的奥尔克斯表示，BioNTech“以其科学根基和尤其强大的晚期肿瘤学管线而脱颖而出”。 “I am deeply honored to be leading BioNTech and prioritizing its evolution into a global biopharmaceutical company with multiple approved products by 2030, while continuing to advance its innovative pre-clinical and clinical portfolio in line with the company’s strategy,” he added. 他说，“能够领导BioNTech，将其发展成一家全球性的生物制药公司，争取在2030年前推出多款获批上市的药品，同时，继续推进其具有创新性的临床前及临床研究项目，以契合公司的战略目标，我深感荣幸。” The appointment was the result of a “comprehensive selection process” led by BioNTech’s supervisory board, the company said in an August 3 release. A search for Türeci’s replacement as CMO remains ongoing. 在8月3日发布的新闻稿中，Biotech表示，此次任命是由监事会主导的“全面遴选流程”的结果。目前，寻找首席医学官厄兹勒姆接替者的工作仍在进行中。

2026-08-07

·adalvo.com

Adalvo Announces Successful Bioequivalence Study for Avatrombopag

Zug, Switzerland, 7 Aug 2026: Adalvo today announced the successful completion of the bioequivalence study for its generic avatrombopag, confirming the programme is on track for US ANDA filing. Developed as a global dossier programme spanning the US, EU and Rest of World, it is designed to position Adalvo and its commercial partners for Day-1 launches in major markets worldwide, upon expiry of applicable exclusivities and IP rights. Developed based on the reference brand Doptelet®, avatrombopag is a second-generation thrombopoietin (TPO) receptor agonist indicated for adults with primary chronic immune thrombocytopenia (ITP), and for thrombocytopenia in adults with chronic liver disease (CLD) who are scheduled to undergo a medical procedure. Net sales of the reference brand Doptelet, as reported by Sobi, reached SEK 5,265 million (approximately USD 555 million) in full-year 2025 and SEK 3,061 million (approximately USD 323 million) in the first half of 2026.¹ These developments reflect Adalvo's commitment to advancing high-quality, differentiated generic medicines and expanding future treatment options for patients through rigorous development and regulatory execution. Doptelet® is a registered trademark of AkaRx, Inc., a Sobi company.

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