Jazz制药公司收到CHMP对Zanidatamab用于治疗晚期HER2阳性胆道癌的积极意见-动脉网

Jazz Pharmaceuticals Receives CHMP Positive Opinion for Zanidatamab for the Treatment of Advanced HER2-Positive Biliary Tract Cancer

CISION 等信源发布 2025-04-25 23:27

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可切换为仅中文







DUBLIN

都柏林

，

April 25, 2025

2025年4月25日

/PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq:

/PRNewswire/ -- Jazz Pharmaceuticals plc（纳斯达克：

JAZZ

爵士乐

) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending the conditional marketing authorization of zanidatamab, an investigational dual human epidermal growth factor receptor 2 (HER2)-targeted bispecific antibody, as monotherapy for the treatment of adults with unresectable locally advanced or metastatic HER2-positive (IHC 3+).

）今天宣布，欧洲药品管理局（EMA）人用医药产品委员会（CHMP）已采纳积极意见，建议对研究中的双特异性抗体zanidatamab（一种针对人类表皮生长因子受体2（HER2）的双特异性抗体）授予附条件上市许可，作为单一疗法用于治疗无法切除的局部晚期或转移性HER2阳性（IHC 3+）成人患者。

†

biliary tract cancer (BTC) previously treated with at least one prior line of systemic therapy.

既往接受过至少一线系统治疗的胆道癌（BTC）。

'This positive CHMP opinion is a welcome step for physicians and patients in

“这一积极的CHMP意见是医生和患者迈出的可喜一步

Europe

欧洲

who face a critical unmet need in HER2-positive biliary tract cancers, a rare and aggressive group of cancers with poor prognosis and limited treatment options,' said

“这些患者面临HER2阳性胆道癌中一个关键的未满足需求，这是一种罕见且侵袭性的癌症群体，预后较差且治疗选择有限，”表示

Robert Iannone

罗伯特·伊安诺内

, MD., M.S.C.E., executive vice president, global head of research and development, and chief medical officer of Jazz Pharmaceuticals. 'If approved, zanidatamab would be the first HER2-targeted therapy licensed for this difficult-to-treat cancer in the EU, marking an important milestone in addressing this unmet need.

医学博士，理学硕士，爵士制药公司执行副总裁，全球研发主管及首席医学官。“如果获得批准，zanidatamab 将成为欧盟首个针对这种难治性癌症的 HER2 靶向疗法，标志着在满足这一未竟需求方面迈出了重要的一步。

We look forward to the European Commission's decision and the opportunity to provide a new treatment option for patients.'.

我们期待欧盟委员会的决定，并为患者提供新的治疗选择的机会。'

The CHMP recommendation is based on data from the Phase

CHMP的建议是基于第三阶段的数据

HERIZON-BTC-01 trial, which evaluated zanidatamab in previously treated, inoperable, and advanced or metastatic HER2-positive BTC.

HERIZON-BTC-01 试验，评估了扎尼达单抗在既往治疗过、不可手术、晚期或转移性 HER2 阳性胆道癌（BTC）中的疗效。

2,3

While biliary tract cancers (BTCs), which include gallbladder cancer (GBC) and cholangiocarcinoma (CCA),

胆道癌 (BTC) 包括胆囊癌 (GBC) 和胆管癌 (CCA)，

account for less than 1% of all human cancers,

占所有人类癌症的比例不到1%，

CCA is the second most common primary liver cancer after hepatocellular carcinoma (HCC).

CCA是继肝细胞癌（HCC）之后第二常见的原发性肝癌。

It comprises approximately 10–15% of all primary liver cancers,

它约占所有原发性肝癌的10%-15%，

，

and its global mortality rate has risen in recent decades.

其全球死亡率在近几十年有所上升。

Most BTC cases are diagnosed at an advanced stage due to the vague or nonspecific nature of early disease symptoms,

由于早期疾病症状的模糊或非特异性，大多数BTC病例在晚期才被诊断出来，

making curative surgery an option for only a minority of patients.

使治愈性手术成为仅少数患者的选择。

5,7,8

Although chemotherapy and, more recently, immunotherapy-based combinations are used in the first-line setting, disease progression is common, and second-line treatment options are, in the absence of molecular analysis, largely limited to chemotherapy.

尽管在一线治疗中使用化疗，以及最近基于免疫疗法的组合，疾病进展仍然常见，并且在缺乏分子分析的情况下，二线治疗选项大多局限于化疗。

，

，9

HER2 overexpression or amplification has been identified as a distinct molecular subtype of BTC

HER2过表达或扩增已被确定为BTC的一种独特分子亚型。

10,11

10，11

and is associated with a worse prognosis compared to HER2-negative BTC.

与HER2阴性的BTC相比，其预后更差。

Yet, no HER2-targeted therapies are currently approved for this indication in the European Union (EU).

然而，目前在欧盟（EU）尚无获批用于该适应症的HER2靶向疗法。

The CHMP's recommendation will now be reviewed by the European Commission which has the authority to approve medicines in all EU Member States,

CHMP的建议现在将由欧洲委员会审查，该委员会有权批准所有欧盟成员国的药品。

Iceland

冰岛

，

Norway

挪威

, and

，以及

Liechtenstein

列支敦士登

, and is expected to make a final decision.

，并有望做出最终决定。

About Zanidatamab

关于Zanidatamab

Zanidatamab is a dual HER2-targeted bispecific antibody that simultaneously binds extracellular domains 2 and 4 on separate HER2 monomers (binding in trans). Binding of zanidatamab with HER2 results in internalization leading to a reduction of the receptor on the cell surface. Zanidatamab induces complement-dependent cytotoxicity (CDC), antibody-dependent cellular cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP).

Zanidatamab是一种双HER2靶向的双特异性抗体，能够同时结合在不同HER2单体上的胞外结构域2和4（跨结合）。Zanidatamab与HER2的结合会导致内化，从而减少细胞表面的受体数量。Zanidatamab诱导补体依赖性细胞毒性（CDC）、抗体依赖性细胞毒性（ADCC）和抗体依赖性细胞吞噬作用（ADCP）。

These mechanisms result in tumor growth inhibition and tumor cell death..

这些机制导致肿瘤生长抑制和肿瘤细胞死亡。

开

November 20, 2024

2024年11月20日

, in the United States, the U.S. Food and Drug Administration (FDA) granted accelerated approval of zanidatamab-hrii (

，在美国，美国食品药品监督管理局（FDA）加速批准了zanidatamab-hrii（

Ziihera

梓赫拉

) for the treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer (BTC), as detected by an FDA-approved test.

) 用于治疗先前接受过治疗、无法切除或转移性的HER2阳性（IHC 3+）胆道癌（BTC）成年患者，需经FDA批准的检测方法确认。

This accelerated approval was granted based on objective response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the ongoing Phase 3 HERIZON-BTC-302 confirmatory trial.

此次加速批准是基于客观缓解率和缓解持续时间。该适应症的持续批准可能取决于正在进行的第3期HERIZON-BTC-302确证性试验中对临床获益的验证和描述。

Zanidatamab is also being investigated in multiple other clinical trials as a targeted treatment option for patients with solid tumors that express HER2. Zanidatamab is being developed by Jazz and BeiGene, Ltd. (BeiGene) under license agreements from Zymeworks, which first developed the molecule. Jazz has rights to commercialize zanidatamab in the U.S., .

Zanidatamab 也在多项其他临床试验中作为针对表达 HER2 的实体瘤患者的靶向治疗选择进行研究。Zanidatamab 由 Jazz 和百济神州（BeiGene）根据与 Zymeworks 的许可协议共同开发，Zymeworks 是最初开发该分子的公司。Jazz 拥有在美国商业化 zanidatamab 的权利。

Europe

欧洲

，

Japan

日本

and all other territories except for those

以及除上述以外的所有其他领土

Asia/Pacific

亚洲/太平洋地区

territories that Zymeworks previously licensed to BeiGene, Ltd. [which are

Zymeworks先前授权给百济神州有限公司的领域 [即

Asia

亚洲

(excluding

（不包括

Japan

日本

Australia

澳大利亚

and

和

New Zealand

新西兰

The FDA granted Breakthrough Therapy designation for zanidatamab development in patients with previously treated HER2 gene-amplified BTC, and two Fast Track designations for zanidatamab: one as a single agent for refractory BTC and one in combination with standard-of-care chemotherapy for 1L gastroesophageal adenocarcinoma (GEA).

FDA授予zanidatamab用于治疗先前接受过HER2基因扩增胆道癌（BTC）患者的突破性疗法认定，并给予zanidatamab两项快速通道认定：一项是作为单药治疗难治性BTC，另一项是与标准护理化疗联合用于一线胃食管腺癌（GEA）。

Additionally, zanidatamab has received Orphan Drug designations from FDA for the treatment of BTC and GEA, as well as Orphan Drug designation from the European Medicines Agency for the treatment of BTC and gastric cancer. .

此外，zanidatamab 已获得 FDA 授予的用于治疗 BTC 和 GEA 的孤儿药资格，以及欧洲药品管理局授予的用于治疗 BTC 和胃癌的孤儿药资格。

About Biliary Tract Cancer

关于胆道癌

Biliary tract cancers (BTC), including gallbladder cancer and intrahepatic and extrahepatic cholangiocarcinoma, account for <1% of all adult cancers worldwide and are often associated with a poor prognosis.

胆道癌 (BTC)，包括胆囊癌以及肝内和肝外胆管癌，占全球所有成人癌症的不到 1%，并且通常预后较差。

，

Approximately 26% of patients with BTC are HER2-positive.

大约26%的BTC患者为HER2阳性。

The human epidermal growth factor receptor 2 (HER2) is a well-validated target for antitumor therapy in other cancers.

人表皮生长因子受体2（HER2）是其他癌症中抗肿瘤治疗的一个得到充分验证的靶点。

,15

，15

Across the U.S., Europe, and Japan, approximately 12,000 people are diagnosed with HER2+ BTC annually.

在美国、欧洲和日本，每年大约有12,000人被诊断出患有HER2+ BTC。

Most patients (>65%) are diagnosed with tumors that cannot be removed surgically.

大多数患者（>65%）被诊断为无法通过手术切除的肿瘤。

About Jazz Pharmaceuticals plc

关于Jazz制药公司

Jazz Pharmaceuticals plc (Nasdaq:

Jazz Pharmaceuticals plc（纳斯达克：

JAZZ

爵士乐

) is a global biopharma company whose purpose is to innovate to transform the lives of patients and their families. We are dedicated to developing potentially life-changing medicines for people with serious diseases — often with limited or no therapeutic options. We have a diverse portfolio of marketed medicines, including leading therapies for sleep disorders and epilepsy, and a growing portfolio of cancer treatments.

）是一家全球生物制药公司，其宗旨是通过创新来改变患者及其家人的生活。我们致力于为患有严重疾病的人群开发可能改变生命的药物——这些疾病通常缺乏或没有治疗选择。我们拥有多种已上市的药品组合，包括针对睡眠障碍和癫痫的领先疗法，并且我们的癌症治疗产品组合也在不断增长。

Our patient-focused and science-driven approach powers pioneering research and development advancements across our robust pipeline of innovative therapeutics in oncology and neuroscience. Jazz is headquartered in Dublin, Ireland with research and development laboratories, manufacturing facilities and employees in multiple countries committed to serving patients worldwide.

我们以患者为中心、以科学为驱动的方法，推动了我们在肿瘤学和神经科学领域强大的创新治疗管道中的开创性研发进展。Jazz 总部位于爱尔兰都柏林，在多个国家设有研发实验室、制造设施和员工，致力于为全球患者服务。

Please visit .

请访问。

www.jazzpharmaceuticals.com

for more information.

更多信息，请访问。

Jazz Pharmaceuticals plc Caution Concerning Forward-Looking Statements

Jazz制药公司关于前瞻性声明的谨慎声明

This press release contains forward-looking statements, including, but not limited to, statements related to the European Commission's potential approval of zanidatamab for the treatment of adults with BTC in the EU and other statements that are not historical facts. These forward-looking statements are based on Jazz Pharmaceuticals' current plans, objectives, estimates, expectations and intentions and inherently involve significant risks and uncertainties.

本新闻稿包含前瞻性陈述，包括但不限于与欧洲委员会可能批准zanidatamab用于治疗欧盟内的BTC成人患者相关的陈述，以及其他非历史事实的陈述。这些前瞻性陈述基于Jazz Pharmaceuticals当前的计划、目标、估计、预期和意图，且不可避免地涉及重大风险和不确定性。

Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with the successful completion of regulatory activities and uncertain regulatory approval, and other risks and uncertainties affecting Jazz Pharmaceuticals and its development programs, including those described from time to time under the caption 'Risk Factors' and elsewhere in Jazz Pharmaceuticals plc's Securities and Exchange Commission filings and reports (Commission File No.

实际结果和事件发生的时间可能与这些前瞻性陈述中预期的情况存在重大差异，这是由于这些风险和不确定性所致，其中包括（但不限于）与监管活动的成功完成及不确定的监管批准相关的风险和不确定性，以及其他影响Jazz Pharmaceuticals及其开发项目的风险和不确定性，包括Jazz Pharmaceuticals plc在证券交易所委员会文件和报告中不时描述的“风险因素”及其他内容（委员会文件编号）。

001-33500), including Jazz Pharmaceuticals' Annual Report on Form 10-K for the year ended .

001-33500），包括Jazz Pharmaceuticals年度报告表格10-K截至当年结束。

December 31, 2024

2024年12月31日

, and future filings and reports by Jazz Pharmaceuticals. Other risks and uncertainties of which Jazz Pharmaceuticals is not currently aware may also affect Jazz Pharmaceuticals' forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated.

以及Jazz Pharmaceuticals未来的文件和报告。Jazz Pharmaceuticals目前尚未意识到的其他风险和不确定性也可能影响其前瞻性陈述，并可能导致实际结果和事件的时间与预期有重大差异。

The forward-looking statements herein are made only as of the date hereof or as of the dates indicated in the forward-looking statements, even if they are subsequently made available by Jazz Pharmaceuticals on its website or otherwise. Jazz Pharmaceuticals undertakes no obligation to update or supplement any forward-looking statements to reflect actual results, new information, future events, changes in its expectations or other circumstances that exist after the date as of which the forward-looking statements were made..

本前瞻性声明仅于本声明发布之日或前瞻性声明中指明的日期作出，即使 Jazz Pharmaceuticals 随后在其网站上或其他方式提供这些声明。Jazz Pharmaceuticals 没有义务更新或补充任何前瞻性声明，以反映实际结果、新信息、未来事件、预期变化或其他在前瞻性声明发布日期之后存在的情况。

Media Contact:

媒体联系人：

Kristin Bhavnani

克里斯汀·巴夫纳尼

Head of Global Corporate Communications

全球企业传播主管

Jazz Pharmaceuticals plc

爵士制药公司

CorporateAffairsMediaInfo@jazzpharma.com

公司事务媒体信息@jazzpharma.com

Ireland

爱尔兰

+353 1 637 2141

U.S. +1 215 867 4948

美国 +1 215 867 4948

Investors:

投资者：

Jeff Macdonald

杰夫·麦克唐纳

Executive Director, Investor Relations

投资者关系执行董事

Jazz Pharmaceuticals plc

爵士制药公司

investorinfo@jazzpharma.com

投资者信息@爵士制药.com

Ireland

爱尔兰

+353 1 634 3211

U.S. +1 650 496 2717

美国 +1 650 496 2717

†

IHC3+ stands for ImmunoHistoChemistry 3+ and refers to the highest level of HER2 protein overexpression in cancer cells

IHC3+ 代表免疫组织化学 3+，指的是癌细胞中 HER2 蛋白过度表达的最高水平。

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参考文献

_______________________

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人类用药产品委员会（CHMP）会议重点

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美国食品药品监督管理局。Ziihera®（扎尼达单抗-hrii）处方信息。Accessdata.fda.gov。更新于

November 2024

2024年11月

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2025年4月22日

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https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/761416s000lbl.pdf

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Meric-Bernstam, F., Beeram 等。Zanidatamab，一种新型双特异性抗体，用于治疗局部晚期或转移性HER2表达或HER2扩增的癌症：一项1期剂量递增和扩展研究。

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《柳叶刀·肿瘤学》

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爵士制药公司，数据存档。

SOURCE Jazz Pharmaceuticals plc

来源：Jazz Pharmaceuticals plc

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