GSK Singapore today announced that Omjjara (momelotinib) has been locally approved for treatment of disease-related splenomegaly or symptoms in adult patients with moderate to severe anaemia who have primary myelofibrosis, post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis and who are Janus Kinase (JAK) inhibitor naive or have been treated with ruxolitinib.[4].

新加坡葛兰素史克公司今天宣布，Omjjara（莫洛替尼）已在当地获批用于治疗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中至重度贫血成年患者的疾病相关脾肿大或症状，这些患者为Janus激酶（JAK）抑制剂初治或曾接受鲁索替尼治疗。[4]

Omjjara is a once-a-day, oral JAK1/JAK2 and activin A receptor type 1 (ACVR1) inhibitor.[4]

Omjjara是一种每日一次的口服JAK1/JAK2和激活素A受体1型（ACVR1）抑制剂。[4]

Myelofibrosis can lead to low blood counts, including anaemia and thrombocytopaenia; constitutional symptoms such as fatigue, night sweats, and bone pain; and splenomegaly. About 40% of patients globally have moderate to severe anaemia at the time of diagnosis, and nearly all patients are estimated to develop anaemia over the course of the disease.[5-8] These patients may become transfusion-dependent and more than 30% will discontinue treatment due to anaemia.[3] Patients who are transfusion-dependent are often associated with a poor quality of life and have a poor prognosis with shortened survival.[1,9-16].

骨髓纤维化可导致血细胞计数减少，包括贫血和血小板减少；全身症状如疲劳、夜间盗汗和骨痛；以及脾肿大。全球约40%的患者在诊断时出现中度至重度贫血，据估计几乎所有患者在病程中都会发展为贫血。[5-8] 这些患者可能会变得依赖输血，超过30%的患者会因贫血而停止治疗。[3] 依赖输血的患者通常生活质量较差，预后不佳，生存期缩短。[1,9-16]

Earlier treatment options for managing myelofibrosis-related anaemia have demonstrated limited efficacy and durability of response; and while JAK inhibitors are often used, they can sometimes exacerbate anaemia.[17] This highlights an unmet need for effective treatments for myelofibrosis patients with anaemia.[17].

早期用于治疗骨髓纤维化相关贫血的方案显示出疗效有限且反应持续时间短；虽然JAK抑制剂常被使用，但有时会加重贫血。[17] 这凸显了骨髓纤维化伴贫血患者对有效治疗的需求尚未满足。[17]

Dr. Stephanie Ambrose, GSK Singapore Medical Director, said: 'We note that myelofibrosis is a complex disease and there are significant challenges in the management of the disease, particularly for patients with anaemia and splenomegaly. We look forward to working with the myelofibrosis community and making a difference by improving the outcome for and quality of life of the patients.'.

新加坡GSK医学总监Stephanie Ambrose博士表示：“我们知道骨髓纤维化是一种复杂的疾病，在管理这种疾病方面存在重大挑战，特别是对于那些贫血和脾肿大的患者。我们期待与骨髓纤维化群体合作，通过改善患者的治疗效果和生活质量来有所作为。”

Clinical Professor Goh Yeow Tee, Senior Consultant, Department of Haematology, Singapore General Hospital (SGH), who was involved in both the MOMENTUM and SIMPLIFY trials, said: 'SGH has been able to help bring new treatment options to our patients through collaborations in clinical studies with global healthcare providers and industry partners.

新加坡中央医院血液科高级顾问、临床教授吴耀智曾参与MOMENTUM和SIMPLIFY试验，他表示：“通过与全球医疗保健提供者和行业伙伴在临床研究方面的合作，新加坡中央医院已经能够帮助为我们的患者带来新的治疗选择。

With approximately 30 new myelofibrosis cases each year, we welcome this additional treatment option which could potentially reduce patients' dependence on blood transfusions and improve their quality of life.'.

每年大约有30例新发的骨髓纤维化病例，我们欢迎这一额外的治疗选择，它可能减少患者对输血的依赖并改善他们的生活质量。

Dr. Daryl Tan of Clinic for Lymphoma, Myeloma and Blood Disorder, Singapore, said: 'The approval of Omjjara marks an important step forward for patients with myelofibrosis, especially those with moderate to severe anaemia who require new treatment options to alleviate the challenges of the disease. The introduction of this single therapy addressing all key manifestations of myelofibrosis is a significant advancement for patients with myelofibrosis.'.

新加坡淋巴瘤、骨髓瘤和血液疾病诊所的陈达尔博士表示：“奥姆加拉的获批标志着骨髓纤维化患者向前迈出了重要一步，特别是那些患有中度至重度贫血需要新治疗选择来缓解疾病挑战的患者。这种单一疗法能够解决骨髓纤维化的所有关键症状，对骨髓纤维化患者来说是一项重大进步。”

GSK's submission for the approval of Omjjara was supported by data from the pivotal MOMENTUM study and a subpopulation of adult patients with anaemia from the SIMPLIFY-1 phase III trial. 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.

GSK提交的Omjjara批准申请得到了关键性MOMENTUM研究的数据支持，以及来自SIMPLIFY-1 III期试验中贫血成人患者亚群的数据支持。MOMENTUM研究旨在评估莫洛替尼与达那唑在贫血、有症状且曾接受过JAK抑制剂治疗的骨髓纤维化人群中，针对治疗和减轻主要表现的安全性和有效性。

The MOMENTUM trial met all its primary and key secondary endpoints, demonstrating statistically significant response with respect to constitutional symptoms, splenic response and transfusion independence, in patients treated with momelotinib versus danazol. 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.

MOMENTUM试验达到了所有主要和关键的次要终点，与达那唑相比，莫洛替尼在治疗患者的体质症状、脾脏反应和输血独立性方面表现出统计学显著反应。SIMPLIFY-1旨在评估莫洛替尼与鲁索替尼在未接受过JAK抑制剂治疗的骨髓纤维化患者中的疗效和安全性。

Safety and efficacy results for SIMPLIFY-1 were based upon a subset of patients with anaemia. [4].

SIMPLIFY-1 的安全性和有效性结果基于一部分贫血患者的数据。[4]。

The most common adverse reactions following use of Omjjara were diarrhoea, thrombocytopenia, nausea, headache, dizziness, fatigue, asthenia, abdominal pain, and cough.[4]

使用Omjjara后最常见的不良反应是腹泻、血小板减少症、恶心、头痛、头晕、疲劳、乏力、腹痛和咳嗽。[4]

About myelofibrosis

关于骨髓纤维化

Myelofibrosis is a rare blood cancer that results from dysregulated JAK-signal transducer and activator of transcription protein signalling and is characterised by constitutional symptoms, splenomegaly, and progressive anaemia.[1,19,20]

骨髓纤维化是一种罕见的血液癌症，由失调的JAK-信号转导和转录激活因子蛋白信号传导引起，其特征为全身症状、脾肿大和进行性贫血。[1,19,20]

About the pivotal MOMENTUM clinical trial

关于关键的MOMENTUM临床试验

MOMENTUM was a phase III, global, multicentre, randomised, double-blind study investigating momelotinib versus danazol in patients with myelofibrosis who were symptomatic and anaemic and had been previously treated with an approved 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.[2] Results from the 24-week treatment period were presented at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting and subsequently published in The Lancet.[21,22].

MOMENTUM是一项III期、全球性、多中心、随机、双盲研究，旨在评估莫洛替尼与达那唑在先前接受过获批的JAK抑制剂治疗的症状性贫血骨髓纤维化患者中的疗效。该试验旨在评估莫洛替尼治疗和减轻该疾病主要特征的安全性和有效性：症状、输血（因贫血）和脾肿大。[2] 为期24周的治疗期结果在2022年美国临床肿瘤学会（ASCO）年会上公布，并随后发表在《柳叶刀》上。[21,22]

About the SIMPLIFY-1 clinical trial

关于SIMPLIFY-1临床试验

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. Safety and efficacy results for SIMPLIFY-1 were based upon a subset of patients with anaemia (haemoglobin <10 g/dL) at baseline.

SIMPLIFY-1 是一项多中心、随机、双盲的 III 期研究，比较了莫洛替尼与鲁索替尼在未接受过 JAK 抑制剂治疗的骨髓纤维化患者中的安全性和有效性。SIMPLIFY-1 的安全性和有效性结果基于基线时患有贫血（血红蛋白 <10 g/dL）的患者子集。

The efficacy of momelotinib in the treatment of patients with myelofibrosis in SIMPLIFY-1 was based on spleen volume response (reduction by 35% or greater).[18].

在SIMPLIFY-1试验中，莫洛替尼治疗骨髓纤维化患者的疗效基于脾脏体积反应（减少35%或更多）。[18]

About Omjjara (momelotinib)

关于Omjjara（莫梅洛替尼）

Omjjara has a different mechanism of action, with inhibitory ability along three key signalling pathways: Janus kinase (JAK) 1, JAK2, and activin A receptor, type 1 (ACVR1).[2,20,23,24]

奥姆贾拉具有不同的作用机制，能够抑制三条关键信号通路：Janus激酶（JAK）1、JAK2和激活素A受体1型（ACVR1）。[2,20,23,24]

Inhibition of JAK1 and JAK2 may improve constitutional symptoms and splenomegaly.[2,20,24] Additionally, inhibition of ACVR1 leads to a decrease in circulating hepcidin, which is elevated in myelofibrosis and contributes to anaemia.[2,20,23,24]

抑制 JAK1 和 JAK2 可能会改善全身症状和脾肿大。[2,20,24] 此外，抑制 ACVR1 会导致循环中 hepcidin 的减少，这种蛋白在骨髓纤维化中升高并导致贫血。[2,20,23,24]

GSK in Oncology

葛兰素史克在肿瘤学领域

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.

GSK致力于通过变革性药物最大化患者的生存率，目前专注于免疫肿瘤学和肿瘤细胞靶向治疗的突破，并在血液恶性肿瘤、妇科癌症和其他实体瘤方面进行开发。

About GSK

关于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 https://www.gsk.com/en-gb/locations/singapore/

GSK是一家全球生物制药公司，致力于联合科学、技术和人才，共同战胜疾病。欲了解更多信息，请访问 https://www.gsk.com/en-gb/locations/singapore/