GSK plc (LSE/NYSE: GSK) today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has approved Omjjara (momelotinib) for the treatment of myelofibrosis. Omjjara is a once-a-day, oral JAK1/JAK2 and activin A receptor type 1 (ACVR1) inhibitor. The approval is based on data from the pivotal phase III MOMENTUM and SIMPLIFY-1 trials..

葛兰素史克股份有限公司（伦敦证交所/纽约证交所：葛兰素史克）今天宣布，日本厚生劳动省（MHLW）已批准Omjjara（莫莫替尼）用于治疗骨髓纤维化。Omjjara是一种每天一次的口服JAK1/JAK2和激活素a受体1型（ACVR1）抑制剂。该批准基于关键的第三阶段动量和SIMPLINE-1试验的数据。。

This is the fourth major regulatory approval for GSK’s momelotinib in the treatment of myelofibrosis, following approval under the brand name Ojjaara from the US Food and Drug Administration and authorisations under the brand name Omjjara from the European Commission and the Medicines and Healthcare products Regulatory Agency in the UK..

这是葛兰素史克莫莫替尼治疗骨髓纤维化的第四次重大监管批准，此前美国食品和药物管理局以Ojjaara品牌批准，欧盟委员会和英国药品和保健品管理局以Omjjara品牌授权。。

Nina Mojas, Senior Vice President, Oncology Global Product Strategy, GSK, said: “Myelofibrosis has a heavy disease burden, with symptomatic patients experiencing spleen enlargement, fatigue, night sweats and bone pain, along with anaemia which can lead to treatment discontinuation and dependence on regular blood transfusions.

葛兰素史克肿瘤学全球产品战略高级副总裁尼娜·莫哈斯（NinaMojas）说：“骨髓纤维化是一种沉重的疾病负担，有症状的患者会出现脾肿大、疲劳、盗汗和骨痛，以及贫血，这可能导致停药和依赖常规输血。

With the approval of Omjjara, myelofibrosis patients in Japan will have a new treatment option for this complex blood cancer.”.

在Omjjara的批准下，日本的骨髓纤维化患者将为这种复杂的血癌提供新的治疗选择。”。

Myelofibrosis is a blood cancer that affects approximately 1 in 500,000 people worldwide, with up to 5,000 patients impacted in Japan.4,5,6 In Japan, about 70% of patients diagnosed with primary myelofibrosis, and about half of those patients diagnosed with secondary myelofibrosis, have moderate to severe anaemia at the time of diagnosis.1,2,3 Nearly all patients are estimated to develop anaemia over the course of the disease.7,8,9,10 Myelofibrosis patients with anaemia require additional supportive care, including transfusions, and more than 30% will discontinue treatment with established therapies due to anaemia.11 Patients who are anaemic and transfusion dependent have a poor prognosis and shortened survival.12,13,14,15,16,17,18,19,20.

骨髓纤维化是一种血癌，影响全球约50万人中的1人，日本有多达5000名患者。4,5,6在日本，约70%的原发性骨髓纤维化患者和约一半的继发性骨髓纤维化患者在诊断时患有中度至重度贫血。1,2,3据估计，几乎所有患者在疾病过程中都会发生贫血。7,8,9,10贫血的骨髓纤维化患者需要额外的支持治疗，包括输血，超过30%的患者将因贫血而停止既定疗法的治疗。11贫血和输血依赖的患者预后不良，生存期缩短。12,13,14,15,16,17,18,19,20。

The approval is based on data from the MOMENTUM and SIMPLIFY-1 pivotal phase III trials. MOMENTUM was designed to evaluate the safety and efficacy of momelotinib versus danazol for the treatment and reduction of key manifestations of myelofibrosis in an anaemic, symptomatic, JAK inhibitor-experienced population.

该批准基于MOMENTUM和SIMPLIFY-1关键III期试验的数据。MOMENTUM旨在评估莫莫替尼与达那唑治疗贫血，有症状，JAK抑制剂经验丰富的人群中骨髓纤维化关键表现的安全性和有效性。

SIMPLIFY-1 was designed to evaluate the efficacy and safety of momelotinib versus ruxolitinib in myelofibrosis patients who had not received a prior JAK inhibitor therapy..

SIMPLIFY-1旨在评估莫莫替尼与ruxolitinib在未接受过JAK抑制剂治疗的骨髓纤维化患者中的疗效和安全性。。

About Omjjara (momelotinib)

关于Omjjara（momelotinib）

Momelotinib has a differentiated mechanism of action, with inhibitory ability along three key signalling pathways: Janus kinase (JAK) 1, JAK2, and activin A receptor, type I (ACVR1).1,21,22,23 Inhibition of JAK1 and JAK2 may improve constitutional symptoms and splenomegaly.1,21,23 Additionally, inhibition of ACVR1 leads to a decrease in circulating hepcidin levels, potentially contributing to anaemia-related benefit.1,21,22,23.

Momelotinib具有不同的作用机制，具有三种关键信号通路的抑制能力：Janus激酶（JAK）1，JAK2和激活素a受体I型（ACVR1）。1,21,22,23抑制JAK1和JAK2可能会改善体质症状和脾肿大[1,21,23]。此外，抑制ACVR1会导致循环铁调素水平降低，可能有助于贫血相关的益处[1,21,22,23]。

In September 2023, the US Food and Drug Administration licensed24 momelotinib under the brand name Ojjaara for the treatment of intermediate or high-risk myelofibrosis, including primary myelofibrosis or secondary myelofibrosis (post-polycythaemia vera and post-essential thrombocythemia), in adults with anaemia..

2023年9月，美国食品和药物管理局（FDA）以Ojjaara的名义批准了24莫莫替尼用于治疗贫血成人的中高危骨髓纤维化，包括原发性骨髓纤维化或继发性骨髓纤维化（真性红细胞增多症后和原发性血小板增多症后）。。

In January 2024, the European Commission granted marketing authorisation25 for Omjjara for disease-related splenomegaly (enlarged spleen) or symptoms in adult patients with moderate to severe anaemia who have primary myelofibrosis, post polycythaemia vera myelofibrosis or post essential thrombocythemia myelofibrosis and who are Janus kinase (JAK) inhibitor naïve or have been treated with ruxolitinib.

2024年1月，欧盟委员会授予Omjjara上市许可25，用于患有原发性骨髓纤维化，真性红细胞增多症后骨髓纤维化或原发性血小板增多症后骨髓纤维化的成年中度至重度贫血患者的疾病相关脾肿大（脾肿大）或症状，以及Janus激酶（JAK）抑制剂幼稚或已接受ruxolitinib治疗的患者。

Omjjara was also approved26 by the Medicines and Healthcare products Regulatory Agency (MHRA) in the United Kingdom to treat the symptoms experienced by adult myelofibrosis patients who have moderate or severe anaemia..

。。

Please refer to the updated Product Information (PI) for precautions concerning indication and important dosage, administration, and safety information in Japan which will shortly be updated at this link: Japan Pharmaceuticals and Medical Devices Agency.27

有关日本适应症和重要剂量，管理和安全信息的注意事项，请参阅更新的产品信息（PI），该信息不久将在此链接更新：日本药品和医疗器械管理局

About myelofibrosis

关于骨髓纤维化

Myelofibrosis is a rare blood cancer that disrupts the body’s normal production of blood cells because of dysregulated JAK-signal transducer and activator of transcription protein signalling. The clinical hallmarks of myelofibrosis are splenomegaly (enlarged spleen), severely low blood counts, including anaemia and thrombocytopenia, and debilitating constitutional symptoms, such as fatigue, night sweats and bone pain, attributable to ineffective haematopoiesis and excessive production of proinflammatory cytokines.28,29.

骨髓纤维化是一种罕见的血癌，由于JAK信号转导和转录蛋白信号激活因子失调，破坏了人体正常的血细胞生成。骨髓纤维化的临床特征是脾肿大（脾肿大），严重低血细胞计数，包括贫血和血小板减少症，以及由于造血功能低下和促炎细胞因子过度产生而导致的虚弱体质症状，如疲劳，盗汗和骨痛[28,29]。

About the pivotal clinical trials

关于关键的临床试验

MOMENTUM was a phase III, global, multicentre, randomised, double-blind study investigating momelotinib versus danazol in patients (n=195) with myelofibrosis who were symptomatic and anaemic and had been previously treated with a licensed JAK inhibitor. The trial was designed to evaluate the safety and efficacy of momelotinib for treating and reducing key hallmarks of the disease: symptoms, blood transfusions (due to anaemia) and splenomegaly.

。该试验旨在评估莫莫替尼治疗和减少疾病关键特征的安全性和有效性：症状，输血（由于贫血）和脾肿大。

The MOMENTUM trial met all its primary and key secondary endpoints, demonstrating statistically significant response with respect to constitutional symptoms, splenic reduction and transfusion independence in patients treated with momelotinib versus danazol (Total Symptom Score reduction of 50% or greater: 25% momelotinib, 9% danazol, p=0.0095; reduction of spleen volume by 35% or greater: momelotinib 22%, danazol 3%, p=0.0011; no transfusions and all haemoglobin values ≥8 g/dL in the 12 weeks prior to week 24: momelotinib 30%, danazol 20%).30 The most common non-haematological treatment-emergent adverse events in momelotinib-treated patients over the entire study period as of the data cutoff were diarrhoea (45 [26%] of 171) and asthenia (28 [16%]); the most common grades 3 and 4 treatment-emergent adverse events were thrombocytopenia (33 [19%]) and anaemia (19 [11%]).31 Results from the 24-week randomised treatment period were presented at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting and subsequently published in The Lancet,32,33 with 48-week data presented at the 64th American Society of Hematology (ASH) Annual Meeting and Exposition in December 2022 and subsequently published in The Lancet Hematology.31,34.

MOMENTUM试验达到了所有主要和关键的次要终点，显示了莫莫替尼与达那唑治疗的患者在体质症状，脾脏减少和输血独立性方面的统计学显着反应（总症状评分降低50%或更高：25%莫莫替尼，9%达那唑，p=0.0095；脾脏体积减少35%或更高：莫莫替尼22%，达那唑3%，p=0.0011；在第24周之前的12周内未输血且所有血红蛋白值≥8 g/dL：莫莫替尼30%，达那唑20%）。30最常见的非血液学治疗紧急不良事件截至数据截止，在整个研究期间，莫莫替尼治疗的患者出现腹泻（171例中有45例（26%）和虚弱（28例（16%））；最常见的3级和4级治疗紧急不良事件是血小板减少症（33[19%]）和贫血（19[11%]）。24周随机治疗期的结果在2022年美国临床肿瘤学会（ASCO）年会上发表，随后发表在《柳叶刀》上，32,33在2022年12月第64届美国血液学会（ASH）年会和博览会上发表了48周的数据，随后发表在《柳叶刀血液学》上[31,34]。

SIMPLIFY-1 was a multicentre randomised, double-blind, phase III study that compared the safety and efficacy of momelotinib to ruxolitinib in patients with myelofibrosis who had not received prior treatment with a JAK inhibitor (momelotinib: n=215 and ruxolitinib: n=217). SIMPLIFY-1 met its primary endpoint, demonstrating non-inferiority of momelotinib to ruxolitinib in spleen volume response (reduction by 35% or greater) with a difference of 9% (95% CI 2%-16%), and substantial improvements in transfusion independence rates (66.5% for momelotinib compared to 49.3% for ruxolitinib), a difference of 18% (95% CI 9%-26%).35,36 The most common grade 3 or higher haematologic abnormalities in either group were thrombocytopenia and anaemia.

SIMPLIFY-1是一项多中心随机，双盲，III期研究，比较了莫莫替尼与ruxolitinib在未接受JAK抑制剂治疗的骨髓纤维化患者中的安全性和有效性（莫莫替尼：n=215和ruxolitinib：n=217）。SIMPLIFY-1达到了其主要终点，证明莫莫替尼在脾脏体积反应（减少35%或更高）方面不劣于鲁索替尼，差异为9%（95%可信区间2%-16%），输血独立率显着提高（莫莫替尼为66.5%，鲁索替尼为49.3%），差异为18%（95%可信区间9%-26%）。35,36两组中最常见的3级或更高血液学异常是血小板减少症和贫血。

Grade 3 or higher infections occurred in 7% of patients who received momelotinib and 3% of patients who received ruxolitinib.37.

在接受莫美洛替尼治疗的患者中，有 7% 出现了 3 级或以上感染，在接受鲁索利替尼治疗的患者中，有 3% 出现了 3 级或以上感染。

GSK in oncology

葛兰素史克与肿瘤学

GSK is committed to maximising patient survival through transformational medicines, with a current focus on breakthroughs in immuno-oncology and tumour-cell targeting therapies, and development in haematologic malignancies, gynaecologic cancers, and other solid tumours.

葛兰素史克致力于通过转化药物最大限度地提高患者的生存率，目前专注于免疫肿瘤学和肿瘤细胞靶向治疗的突破，以及血液系统恶性肿瘤，妇科癌症和其他实体瘤的发展。

About GSK

GSK

GSK is a global biopharma company with a purpose to unite science, technology, and talent to get ahead of disease together. Find out more at gsk.com.

葛兰素史克是一家全球性生物制药公司，旨在将科学、技术和人才团结起来，共同战胜疾病。。