Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced the U.S. Food and Drug Administration (FDA) has approved WELIREG

默克公司（纽约证券交易所代码：MRK），在美国和加拿大以外地区以MSD之名运营，今天宣布美国食品药品监督管理局（FDA）已批准WELIREG。

(belzutifan), Merck’s oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor, for the treatment of adult and pediatric patients 12 years and older with locally advanced, unresectable, or metastatic pheochromocytoma or paraganglioma (PPGL). Pheochromocytoma and paraganglioma are rare tumors that come from the same tissue, but pheochromocytoma form in the adrenal gland and paraganglioma form outside the adrenal gland.

(belzutifan)，默克公司的口服低氧诱导因子-2α（HIF-2α）抑制剂，用于治疗12岁及以上的局部晚期、不可切除或转移性嗜铬细胞瘤或副神经节瘤（PPGL）的成人和儿童患者。嗜铬细胞瘤和副神经节瘤是来自相同组织的罕见肿瘤，但嗜铬细胞瘤形成于肾上腺，而副神经节瘤形成于肾上腺之外。

These tumors can be caused by certain genetic syndromes or mutations. The approval is based on data from the single-arm LITESPARK-015 clinical trial, where the primary endpoint was objective response rate (ORR)..

这些肿瘤可能由某些遗传综合症或基因突变引起。此次批准基于单臂LITESPARK-015临床试验的数据，其主要终点是客观缓解率（ORR）。

“PPGL, sometimes referred to as pheo para, is a rare condition affecting up to 2,000 people each year in the United States. Patients with these tumors, which arise from the adrenal glands and the extra-adrenal paraganglia, may require specialized care due to their complexity and rare nature, often posing significant challenges for both diagnosis and treatment,” said Dr.

“PPGL，有时被称为嗜铬细胞瘤-副神经节瘤，是一种罕见的疾病，每年在美国影响多达2000人。这些肿瘤源于肾上腺和肾上腺外的副神经节，由于其复杂性和罕见性，患者可能需要特殊的护理，常常给诊断和治疗带来重大挑战，”博士说道。

Camilo Jimenez, professor, department of endocrine neoplasia and hormonal disorders, The University of Texas MD Anderson Cancer Center. “This approval, which is based on objective response rate data from the LITESPARK-015 trial, introduces belzutifan as the only approved and available non-surgical option for locally advanced, unresectable, or metastatic PPGL and could represent a change to the treatment paradigm for eligible patients.”.

德克萨斯大学MD安德森癌症中心内分泌肿瘤和激素紊乱系教授卡米洛·希门尼斯表示：“此次批准基于LITESPARK-015试验的客观缓解率数据，引入了belzutifan作为唯一获批且可用的局部晚期、不可切除或转移性PPGL非手术治疗选择，可能代表了符合条件患者治疗模式的转变。”

“For patients with advanced PPGL, there has been a lack of approved systemic treatment options available to help manage their disease, underscoring the importance of this approval in the U.S.,” said Dr. Marjorie Green, senior vice president and head of oncology, global clinical development, Merck Research Laboratories.

“对于晚期PPGL患者，目前缺乏获批的系统性治疗选择来帮助管理他们的疾病，这凸显了此次在美国获得批准的重要性，”默克研究实验室肿瘤学高级副总裁兼全球临床开发主管玛乔丽·格林博士表示。

“This approval marks the third indication for WELIREG in the U.S. and demonstrates our company’s commitment to providing innovative cancer therapies for patients in need, including those with rare diseases.”.

“此次批准标志着WELIREG在美国的第三个适应症，也体现了我们公司致力于为有需要的患者，包括罕见病患者，提供创新的癌症治疗方案。”

The WELIREG label contains a boxed warning that exposure to WELIREG during pregnancy can cause embryo-fetal harm. Verify pregnancy status prior to the initiation of WELIREG. Advise patients of these risks and the need for effective non-hormonal contraception. WELIREG can render some hormonal contraceptives ineffective.

WELIREG的标签包含一个加框警告，指出在怀孕期间接触WELIREG可能会对胚胎和胎儿造成伤害。在开始使用WELIREG之前，应确认患者的怀孕状态。告知患者这些风险以及需要采取有效的非激素避孕措施。WELIREG可能会使某些激素类避孕药无效。

WELIREG can cause severe anemia that can require a blood transfusion. Monitor for anemia before initiation of and periodically throughout treatment with WELIREG. WELIREG can cause severe hypoxia that may require discontinuation, supplemental oxygen, or hospitalization. Monitor oxygen saturation before initiation of and periodically throughout treatment with WELIREG.

WELIREG 可能导致需要输血的严重贫血。在开始使用 WELIREG 之前以及整个治疗期间应定期监测贫血情况。WELIREG 可能导致需要停止用药、补充氧气或住院治疗的严重低氧血症。在开始使用 WELIREG 之前以及整个治疗期间应定期监测血氧饱和度。

For more information, see “Selected Safety Information” below..

有关更多信息，请参阅下面的“精选安全信息”。

Study design

研究设计

LITESPARK-015 is an open-label, multicohort Phase 2 trial (ClinicalTrials.gov,

LITESPARK-015 是一项开放标签、多队列的 2 期试验 (ClinicalTrials.gov，

NCT04924075

NCT04924075

) evaluating the efficacy and safety of WELIREG monotherapy. The study enrolled 72 patients in a single cohort (Cohort A1) who had measurable disease verified by blinded independent central review (BICR) per RECIST v1.1, documented histopathological diagnosis of PPGL, locally advanced or metastatic disease that was not amenable to surgery or curative treatment, and adequately controlled blood pressure (defined as BP <150/90 mm Hg, <135/85 mm Hg for adolescents) with no change in antihypertensive medications for patients with concomitant hypertension for at least two weeks prior to start of study treatment.

）评估WELIREG单药治疗的疗效和安全性。该研究在单一队列（A1组）中招募了72名患者，这些患者经盲态独立中心审查（BICR）根据RECIST v1.1标准确认有可测量病灶，有PPGL的组织病理学诊断记录，患有局部晚期或转移性疾病，无法通过手术或治愈性治疗处理，并且血压得到充分控制（定义为血压<150/90 mm Hg，青少年<135/85 mm Hg），同时对于伴有高血压的患者，在研究治疗开始前至少两周内未改变抗高血压药物。

Patients with carcinomatous meningitis were excluded. Patients received WELIREG at a dose of 120 mg once daily until disease progression or unacceptable toxicity..

患有癌性脑膜炎的患者被排除在外。患者每日一次接受120毫克WELIREG治疗，直至疾病进展或出现不可接受的毒性。

The major efficacy outcome measure for the treatment of advanced PPGL was ORR measured by BICR using RECIST v1.1. Additional efficacy outcome measures included duration of response and time to response.

治疗晚期PPGL的主要疗效指标是通过BICR使用RECIST v1.1评估的ORR。其他疗效指标包括反应持续时间和反应时间。

About pheochromocytoma and paraganglioma

关于嗜铬细胞瘤和副神经节瘤

Pheochromocytoma and paraganglioma (PPGL), sometimes referred to as pheo para, are rare tumors that can be caused by certain genetic syndromes or mutations. It is estimated that up to 2,000 new cases of PPGL are diagnosed each year in the U.S., and up to 52,800 new cases are diagnosed each year worldwide.

嗜铬细胞瘤和副神经节瘤（PPGL），有时被称为嗜铬细胞瘤，是由某些遗传综合征或基因突变引起的罕见肿瘤。据估计，美国每年约有2000例新诊断的PPGL病例，而全球每年约有52800例新诊断的病例。

Pheochromocytoma form in nerve tissue in the center of the adrenal gland, whereas paraganglioma form in nerve tissue near certain blood vessels and nerves outside the adrenal glands..

嗜铬细胞瘤形成于肾上腺中心的神经组织，而副神经节瘤形成于肾上腺外某些血管和神经附近的神经组织。

Merck’s focus on cancer

默克公司对癌症的关注

Every day, we follow the science as we work to discover innovations that can help patients, no matter what stage of cancer they have. As a leading oncology company, we are pursuing research where scientific opportunity and medical need converge, underpinned by our diverse pipeline of more than 25 novel mechanisms.

每一天，我们都在追寻科学的脚步，努力发现能够帮助患者的创新方法，无论他们处于癌症的哪个阶段。作为一家领先的肿瘤学公司，我们正在科学机遇与医疗需求交汇的领域开展研究，依托我们多样化的研发管线，其中包含 25 种以上的新机制。

With one of the largest clinical development programs across more than 30 tumor types, we strive to advance breakthrough science that will shape the future of oncology. By addressing barriers to clinical trial participation, screening and treatment, we work with urgency to reduce disparities and help ensure patients have access to high-quality cancer care.

通过在超过30种肿瘤类型中开展规模最大的临床开发项目之一，我们致力于推动突破性科学的发展，这将塑造肿瘤学的未来。通过解决参与临床试验、筛查和治疗的障碍，我们紧急努力减少差异，帮助确保患者能够获得高质量的癌症护理。

Our unwavering commitment is what will bring us closer to our goal of bringing life to more patients with cancer. For more information, visit .

我们坚定不移的承诺将使我们更接近为更多癌症患者带来生命的目标。欲了解更多信息，请访问。

About Merck

关于默克公司

At Merck, known as MSD outside of the United States and Canada, we are unified around our purpose: We use the power of leading-edge science to save and improve lives around the world. For more than 130 years, we have brought hope to humanity through the development of important medicines and vaccines.

在默克（在美国和加拿大以外地区称为MSD），我们围绕一个使命团结一致：我们利用前沿科学的力量拯救生命并改善全球人类的生活。130多年来，我们通过研发重要的药物和疫苗，为人类带来了希望。

We aspire to be the premier research-intensive biopharmaceutical company in the world – and today, we are at the forefront of research to deliver innovative health solutions that advance the prevention and treatment of diseases in people and animals. We foster a diverse and inclusive global workforce and operate responsibly every day to enable a safe, sustainable and healthy future for all people and communities.

我们立志成为世界首屈一指的研究密集型生物制药公司——今天，我们站在研究的最前沿，提供创新的健康解决方案，推动人类和动物疾病预防与治疗的进步。我们培养多元化和包容性的全球员工队伍，并每天负责任地运营，以实现一个安全、可持续和健康的未来，造福所有人和社区。