The purpose of this study is to find out whether upfront emapalumab treatment can help in sAA (Aplastic Anemia) treatment planning and increase the effectiveness of standard treatment options.

开始日期2024-05-21

申办/合作机构

Memorial Sloan Kettering Cancer Center

CTR20221155 / Recruiting临床2/3期

一项在斯蒂尔病（包括症状性青少年特发性关节炎和成人发病型斯蒂尔病）继发巨噬细胞活化综合征 (MAS) 或系统性红斑狼疮继发 MAS 的儿童和成人患者中评价 Emapalumab 的疗效、安全性和耐受性、药代动力学及药效学的两队列、开放性、单臂、多中心研究

本研究主要目的是证明 Emapalumab 治疗症状性青少年特发性关节炎和成人发病型斯蒂尔病继发巨噬细胞活化综合征患者及儿童和成人系统性红斑狼疮继发巨噬细胞活化综合征患者的疗效。本研究次要目的是证明Emapalumab 在糖皮质激素逐渐减量方面的疗效；评估 Emapalumab 治疗起效时间；评估 Emapalumab 在总体缓解方面的疗效；评估Emapalumab 治疗的持续疗效；评估患者接受 Emapalumab 治疗后的生存情况；评估 Emapalumab 的安全性和耐受性；评估接受 Emapalumab 治疗的患者报告的巨噬细胞活化综合征结局；确定 Emapalumab 的药代动力学特征；确定 Emapalumab 的药效学特征；确定Emapalumab 的免疫原性。

开始日期2023-08-02

申办/合作机构

苏庇医药（广州）有限公司

100 项与依帕伐单抗相关的临床结果

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100 项与依帕伐单抗相关的转化医学

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100 项与依帕伐单抗相关的专利（医药）

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项与依帕伐单抗相关的文献（医药）

2024-03-07·Blood

Survival in primary hemophagocytic lymphohistiocytosis, 2016 to 2021: etoposide is better than its reputation

Article

作者: Lindemans, Caroline ; Beier, Rita ; Kühnle, Ingrid ; Lang, Peter ; Ifversen, Marianne ; Naumann-Bartsch, Nora ; Dürken, Matthias ; Böhm, Svea ; Henter, Jan-Inge ; Yacobovich, Joanne ; Marsh, Rebecca ; Jordan, Michael ; Hönig, Manfred ; Jorch, Norbert ; Russo, Alexandra ; Talano, Julie-An ; Gil-Herrera, Juana ; Prader, Seraina ; Nichols, Kim E ; Schulz, Ansgar ; Lehmberg, Kai ; Bakhtiar, Shahrzad ; Streiter, Monika ; Formankova, Renata ; Behnisch, Wolfgang ; Nathrath, Michaela ; Malinowska, Iwona ; Ehl, Stephan ; Pachlopnik Schmid, Jana ; Hines, Melissa ; Mauz-Körholz, Christine ; Wustrau, Katharina ; Crooks, Bruce ; Speckmann, Carsten ; Ahlmann, Martina

Abstract:

Primary hemophagocytic lymphohistiocytosis (pHLH) is a life-threatening hyperinflammatory syndrome that develops mainly in patients with genetic disorders of lymphocyte cytotoxicity and X-linked lymphoproliferative syndromes. Previous studies with etoposide-based treatment followed by hematopoetic stem cell transplantation (HSCT) resulted in 5-year survival of 50% to 59%. Contemporary data are lacking. We evaluated 88 patients with pHLH documented in the international HLH registry from 2016-2021. In 12 of 88 patients, diagnosis was made without HLH activity, based on siblings or albinism. Major HLH-directed drugs (etoposide, antithymocyte globulin, alemtuzumab, emapalumab, ruxolitinib) were administered to 66 of 76 patients who were symptomatic (86% first-line etoposide); 16 of 57 patients treated with etoposide and 3 of 9 with other first-line treatment received salvage therapy. HSCT was performed in 75 patients; 7 patients died before HSCT. Three-year probability of survival (pSU) was 82% (confidence interval [CI], 72%-88%) for the entire cohort and 77% (CI, 64%-86%) for patients receiving first-line etoposide. Compared with the HLH-2004 study, both pre-HSCT and post-HSCT survival of patients receiving first-line etoposide improved, 83% to 91% and 70% to 88%. Differences to HLH-2004 included preferential use of reduced-toxicity conditioning and reduced time from diagnosis to HSCT (from 148 to 88 days). Three-year pSU was lower with haploidentical (4 of 9 patients [44%]) than with other donors (62 of 66 [94%]; P < .001). Importantly, early HSCT for patients who were asymptomatic resulted in 100% survival, emphasizing the potential benefit of newborn screening. This contemporary standard-of-care study of patients with pHLH reveals that first-line etoposide-based therapy is better than previously reported, providing a benchmark for novel treatment regimes.

2024-02-01·Pediatric blood & cancer

Exposure–safety relationship for patients with primary hemophagocytic lymphohistiocytosis treated with emapalumab

Article

作者: Jordan, Michael B ; Locatelli, Franco

Abstract:

Primary hemophagocytic lymphohistiocytosis (pHLH) is an immune‐mediated, hyperinflammatory disorder. Interferon‐γ (IFNγ) plays a key role in the pathophysiology of pHLH. Emapalumab, a fully human, anti‐IFNγ monoclonal antibody neutralizes both free and receptor‐bound IFNγ. However, inhibiting IFNγ‐mediated signaling could result in immune dysfunction and immunosuppression. This exploratory exposure–safety analysis investigated the relationship between emapalumab and the incidence of adverse events in patients with pHLH. Increased exposure to emapalumab was not associated with an increased predicted risk of severe adverse events, infection, or infusion‐related reactions. Emapalumab was associated with a favorable and manageable safety profile across all assessed doses and treatment durations.

2023-11-28·Blood advances

Schuelke MR, Bassiri H, Behrens EM, et al. Emapalumab for the treatment of refractory cytokine release syndrome in pediatric patients. Blood Adv. 2023;7(18):5603–5607.

Article

项与依帕伐单抗相关的新闻（医药）

2024-05-27

·GlobeNewswire-Health Care

Sobi Receives US FDA Fast Track Designation for Emapalumab-lzsg being investigated in Macrophage Activation Syndrome

May 24, 2024 - Sobi North America, the North American affiliate of Swedish Orphan Biovitrum AB (Sobi®), today announced that the US Food and Drug Administration (FDA) has granted Fast Track designation to emapalumab-lzsg being investigated as a potential therapeutic option in patients with Macrophage Activation Syndrome (MAS). Emapalumab is a fully human, anti-IFNγ monoclonal antibody that binds free and receptor-bound IFNγ, neutralizing its biological activity. Emapalumab is currently in Phase lll development for MAS. Fast track designation is designed to facilitate the development and expedite the review of medicines to treat serious conditions that may fill an unmet medical need. MAS is a severe complication of rheumatic diseases, most frequently in Still’s disease including systemic juvenile idiopathic arthritis (sJIA). Interferon gamma (INFy) is a key cytokine in the development of MAS. MAS is classified as a form of haemophagocytic lymphohistiocytosis (HLH) and characterized by fever, hepatosplenomegaly, liver dysfunction, cytopenias, coagulation abnormalities and hyperferritinaemia, possibly rapidly progressing to multiple organ failure and death. Emapalumab-lzsg is the first and only medicine approved in the US for primary HLH, a rare syndrome of hyperinflammation that can rapidly become fatal unless diagnosed and treated. Emapalumab-lzsg is a fully human, anti-IFNγ monoclonal antibody that binds free and receptor-bound IFNγ, neutralizing its biological activity. In the US, emapalumab-lzsg is indicated for pediatric (newborn and older) and adult primary hemophagocytic lymphohistiocytosis (HLH) patients with refractory, recurrent or progressive disease, or intolerance to conventional HLH therapy. Emapalumab-lzsg is the first and only medicine approved in the US for primary HLH, a rare syndrome of hyperinflammation that usually occurs within the first year of life and can rapidly become fatal unless diagnosed and treated. The FDA approval in 2018 was based on data from the phase 2/3 studies (NCT01818492 and NCT02069899). Emapalumab is indicated for administration through intravenous infusion over one hour twice per week until hematopoietic stem cell transplantation (HSCT). The efficacy and safety of emapalumab are currently being evaluated in a phase 3 study in patients with MAS in Still’s disease or systemic lupus erythematosus (SLE) (EMERALD; NCT05001737). Gamifant® (emapalumab-lzsg) is an interferon gamma (IFNγ)–blocking antibody indicated for the treatment of adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent, or progressive disease or intolerance with conventional HLH therapy. As the North American affiliate of international biopharmaceutical company Sobi, the Sobi North America team is committed to Sobi’s vision of being a leader in providing innovative treatments that transform lives for individuals with rare diseases. Our product portfolio includes multiple approved treatments focused on immunology, hematology and specialty care. With U.S. headquarters in the Boston area, Canadian headquarters in the Toronto area, and field sales, medical and market access representatives spanning North America, our growing team has a proven track record of commercial excellence. More information is available at https://www.sobi.com/usa or at www.sobi.com. Sobi is a specialized international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of hematology, immunology and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia and Australia. In 2023, revenue amounted to SEK 22.1 billion. Sobi’s share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com, LinkedIn and YouTube. The content above comes from the network. if any infringement, please contact us to modify.

上市批准快速通道临床结果

2024-04-25

·PRNewswire

Sobi Q1 2024 report: Strong sales reflecting the strength of the portfolio

STOCKHOLM, April 25, 2024 /PRNewswire/ -- Swedish Orphan Biovitrum AB (publ) (Sobi®) today announced its report for the first quarter 2024 First Quarter 2024 Total revenue increased 19 per cent, 20 per cent at constant exchange rates, (CER)1, to SEK 6,256 M (5,239) Haematology revenue increased 46 per cent at CER to SEK 4,075 M (2,815), reflecting growth in all medicines, mainly driven by strong sales of Doptelet® of SEK 756 M (475), sales of Vonjo® of SEK 320 M (—), and sales of Aspaveli®/Empaveli® of SEK 240 M (95) Immunology revenue decreased 11 per cent at CER to SEK 1,908 M (2,151), mainly reflecting a significant drop in Synagis® sales to SEK 520 M (1,398), partly compensated by strong sales of Gamifant® of SEK 438 M (219), and royalty on Beyfortus™ of SEK 318 M (—) Revenue from medicines in the strategic portfolio more than doubled in the quarter, to SEK 2,193 M (794), driving the majority of the growth. The strategic portfolio grew by 177 per cent at CER and helped to further transform the company The adjusted EBITA margin1,2 was 37 per cent (40), excluding items affecting comparability (IAC)2. EBITA was SEK 2,177 M (2,121), corresponding to a margin of 35 per cent (40). EBIT was SEK 1,313 M (1,495) Earnings per share (EPS) before dilution was SEK 2.35 (3.44)3. Adjusted EPS before dilution1 was SEK 2.70 (3.44)3. Cash flow from operating activities was SEK 2,256 M (1,983) Outlook 2024 - unchanged Revenue is anticipated to grow by a high single-digit percentage at CER The adjusted EBITA margin is anticipated to be in the mid-30s percentage of revenue The strategic portfolio includes Sobi's medicines Aspaveli/Empaveli, Doptelet excluding China, Gamifant, Vonjo and Zynlonta®, and royalty on Sanofi's sales on Altuviiio™ and Beyfortus. Investors, analysts and media are invited to participate in a conference call, which will include a presentation of the results and a Q&A session on the same day at 16:00 CEST. The presentation can be followed live here or afterwards on sobi.com. The slides will be made available on sobi.com prior to the conference call. To participate in the conference call, please use the following dial-in details: Sweden: +46 8 5051 0031 United Kingdom: +44 207 107 06 13 United States: +1 631 570 56 13 For other countries, please find the details here. Sobi Sobi® is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, immunology and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia and Australia. In 2023, revenue amounted to SEK 22.1 billion. Sobi's share (STO: SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information is information that Sobi is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out below, on 25 April 2024 at 08:00 CEST. Gerard Tobin Head of Investor Relations This information was brought to you by Cision The following files are available for download:

财报生物类似药

2024-03-06

·动脉网

拒绝辉瑞、渤健、阿斯利康争相收购，瑞典公司Sobi如何在巨头布局的罕见病赛道杀出重围？

2021年9月，初秋的瑞典逐渐转凉，彼时，医疗健康市场的热度尚未在北欧留下火花，但一起重磅收购案件依然引爆了平和多年的北欧资本市场：私募巨头安宏资本（Advent International）和GIC Special Investments关联公司Aurora对瑞典罕见病制药公司Sobi提出了76亿美元的收购计划。两家机构的出价为235瑞典克朗/股（25.72美元/股），与Sobi于2021年8月25日的开盘价相比，溢价34.5%。在当年，这称得上是几年来北欧市场中金额最大的收购案。不过，如果这两大投资机构要顺利完成收购，需要得到Sobi公司90%股东的支持。只是难得的高额收购项目名震一时，鲜有人去深入讨论这项条款。随着瑞典入冬，进入12月，就在所有人以为此次收购板上钉钉时，巨头阿斯利康突然介入，以扣留其持有的8%股份为由，阻止了这起万众瞩目的交易。关于个中理由，业界一时间众说纷坛。根据彭博社消息，若Sobi最终被出售，那么Sobi拥有的帕丽珠单抗——针对儿童严重呼吸道合胞病毒（RVS）的药物Synagis的权益将流失，而这项权益原属于阿斯利康。据悉，在2018年底，阿斯利康才同意将Synagis的美国权益剥离出售给Sobi。表面看来，似乎是阿斯利康这个半路杀出的程咬金阻止了这起收购，但早在2015年，Sobi就因“报价过低”，拒绝过来自辉瑞和渤健的收购提议。究竟是谁给了Sobi这样的底气和硬气，让这样一家多次处于风口浪尖的北欧公司在几多波澜中保持独立经营？在往昔的合作伙伴纷纷以被收购退出时，又是什么令Sobi在经历市值大跌后屹立至今？重组、合并与再生：从啤酒厂诞生的瑞典罕见病巨头Sobi官网表示，公司历史可以追溯到上个世纪30年代，其前身Kabivitrum最初由两家企业Kabi和Vitrum合并而成；如果我们追根溯源，会发现Sobi的历史可以一直追溯到17世纪的一家酒厂Kongens Bryghus。酒厂Kongens Bryghus原位于丹麦首都哥本哈根市腓特烈斯霍尔姆运河的拐角处，于三十年战争中历经两次焚毁与重建，直至搬迁过河，作为仓库运营至19世纪初期。到19世纪末，Kongens Bryghus已成为赫赫有名的酒厂招牌，并顺势与其他几家啤酒厂合并，于1931年在瑞典马尔默市成立全新公司Kärnbolaget Aktiebolag Biokemisk Industri，Kabi作为其子公司也随之成立。不过，此时的Kabi仅仅承担部分生产维生素C制剂的业务，距离正式步入医疗领域，还尚有一段时日。公司真正介入医疗健康领域，是在第二次世界大战期间。战场上急需一种安全、有效、体积小、便于保存和运输的血浆容量扩张剂抢救伤员。与此同时，美国哈佛大学E.J.Cohn教授和其工作小组研究发明出一种被称为低温乙醇法的工艺从人血中提纯出人血清白蛋白，第一种血液制品从此诞生。为响应战时政策，Kabi开始为武装部队生产干血浆，随后又开始将生产范围扩大到包括白蛋白在内的各类血液制品。随着肌注人免疫球蛋白、人凝血因子VIII的成功分离和血液制品工业化生产体系成型，Kabi不断发展血液制品业务。1972年，在合并了公司Vitrum后，Kabi最终于正式更名为KabiVitrum，也就是Sobi的前身。20世纪80年代，KabiVitrum与基因泰克联手，合作开发去蛋氨酸生长激素Genotropin。哪知KabiVitrum之名也并未长久保留。1990年，KabiVitrum的所有者、国有企业Procordia购买了瑞典知名制药企业Pharmacia的股权，并将Pharmacia旗下多个部门合并，成立Biovitrum。此时的Biovitrum，几经并购与重组，已经完全脱离了17世纪的河畔酒厂，脱胎换骨成一个全新的现代化药企，并先后与惠氏（现并入辉瑞）、Syntonix（后来的Biogen、Bioverativ、现在的赛诺菲）签署系列协议，这一些列合作协议精准提升了Biovitrum在血液学领域的研发实力，似乎也在为公司的下一步发展暗中铺垫。终于，在2010年，Biovitrum投资者收购了瑞典的孤儿药先驱Swedish Orphan International，并成立了新公司Swedish Orphan Biovitrum AB，即Sobi。我们需要格外注意Sobi的横空出世，因为此次收购带来了Orfadin®（尼替西农），后来成为了Sobi的重要药物；也正是在这一年，公司领导层决定将旗下已有的两种血友病候选药物推进至关键的3期临床试验，并隐隐摸索出了Sobi日后的发展方向。大胆砍掉早期管线，精准布局核心核心领域2013年，也就是在成立仅3年后，依托往日的深厚积累和日益探索出的发展模式，Sobi在纳斯达克斯德哥尔摩证券交易所上市，成为瑞典近8年来首家上市的制药公司。此后，Sobi一方面通过系列收购来建立罕见病业务，一方面也以价格为由接连拒绝了辉瑞和渤健的收购提议。可以说，正是因为明白并购重组对Sobi一路走来的意义，才会在对待自身的退出方式上格外谨慎。2018年，Sobi通过从抗体药物开发商Novimmune获得Gamifant® （emapalumab）的全球权利，这也是FDA批准的治疗原发性噬血细胞性淋巴组织细胞增多征（HLH）的首个疗法。此举加强了Sobi在免疫学的专营权。2019年对Sobi来说是关键的一年。一方面是加速收购：除了完成对阿斯利康下呼吸道感染药物Synagis（帕丽珠单抗）在美国权益的收购，以便有权获得阿斯利康候选药物MEDI8897在美国市场未来收益的50%分成外，Sobi还以9.15亿美元收购美国生物制药公司Dova，获得Doptelet®（avatrombopag），用于对其他疗法无效的原发性慢性免疫性血小板减少症（ITP）成年患者，彼时正处于3期临床试验。另一方面，或许是明白对于大公司而言，为了填补管线，除了M&A，难以找到更好的方法，且适合M&A的标的并不多，因此，Sobi除了精确锁定标的外，也竭尽全力争取开发和独家商业化权利，这些在和Apellis Pharmaceuticals、ADC Therapeutics等企业的合作项目中可见一斑。此外，Sobi还做出了一个大胆的决定：加强对血液学和免疫学后期研发的关注，并停止核心领域以外的发现或早期研究。当前，Sobi以罕见病作为原点，围绕血液学、免疫学和专项护理学三大领域开展业务，其已注册上市和尚在二期和三期临床的产品规划分明。信息源自Sobi官网，动脉网制图信息源自Sobi官网，动脉网制图■ Synagis®（帕利珠单抗）帕利珠单抗属于人源化单克隆抗体，人源化单克隆抗体通常是用非人源单克隆抗体的互补决定区（complementarity determining region，CDR）替换人源单克隆抗体氨基酸序列，保留抗体的中和活性，降低外源抗体的免疫原性。帕利珠单抗由鼠源单克隆抗体1129进行人源化改造，将1129的CDR区嫁接到人IgG1抗体框架上，获得帕利珠单抗。该类生物制品最初由MedImmune研发，并于1998年获得美国FDA批准。帕利珠单抗后于2007年被阿斯利康收购，又于2008年被Sobi收购，是目前唯一被批准使用的预防性单克隆抗体。帕利珠单抗价格昂贵，而且半衰期短，在整个HRSV感染季节，需要每月肌肉注射一次，因此只推荐用于早产儿、免疫低下的儿童和患有肺部相关疾病的老年人。Synagis®（帕利珠单抗）为Sobi的主要产品及营收主要来源之一。■ VONJO®（帕克替尼）帕克替尼原为由百特国际和合作伙伴CTI Biopharma开发的一种口服酪氨酸激酶抑制剂，现已被Sobi收购。帕克替尼具有针对JAK2和FLT3的双重活性。已有研究表明，这些激酶的突变与各类血液相关癌症的形成直接相关，包括骨髓增生性肿瘤、白血病和淋巴瘤。帕克替尼能够有效治疗疾病症状，同时具有更少的药物出现的血小板减少症及贫血，这些副作用常见于目前已获批及在研的JAK抑制剂；因此，与其他JAK抑制剂相比，帕克替尼具有优势。■ Orfadin® （尼替西农）尼替西农是一种羟基苯丙酮酸双加氧酶抑制剂，结合酪氨酸和苯丙氨酸饮食限制，用于治疗成人和儿童酪氨酸血症I型（HT-1）。尼替西农是用于治疗HT-1的唯一有效药物，为国外指南推荐的首选治疗方案。当前，Orfadin® （尼替西农）为Sobi专项护理业务板块中的重要组成部分。收购风波导致市值暴跌，核心产品营收力挽狂澜回顾2023年，生物医药产业面临了种种困境，尤其体现在大公司纷纷砍掉管线、市场逐步显示出“少产品”趋势。处于其中的Sobi尽管在2023年的依然实现了18%的营收增长，但也并不意味着公司正式成立后的发展就一帆风顺。让我们再度回顾2021年底那起未完成的并购项目：持有Sobi公司8%股份的阿斯利康和几位股东投出了“反对票”决定保留股份。安宏资本和GIC Special Investments最终获得87%的股份，这笔欧洲当年最大的收购案不得不作罢。此前未提到的是，该消息一出，Sobi股价随之下跌近25%，市值蒸发近四分之一。Sobi并未一蹶不振。此时，Sobi早些年的深厚积淀开始发挥作用。整个2022年，一项高调扩张的Sobi突然转变策略，开启了低调逆袭之路：加快研发与注册申报流程，迅速推动产品商业化以摆脱窘境。这一年，Sobi旗下产品Gamifant® （emapalumab）在中国获批用于治疗噬血细胞综合征（HLH），Alprolix® * （eftrenonacog alfa）获FDA批准作为治疗A型血友病的突破性疗法，Zynlonta在欧盟获批用于治疗复发性或难治性弥漫性大B细胞淋巴瘤，可谓收货颇丰。与此同时，Sobi在2022年发布的年度可持续发展报告，报告显示，最近五年公司产品营收整体处于不断增长当中，尽管专项护理产品收入在2019年之后有轻微下降的趋势，但是Sobi的大头——血液学和免疫学产品仍在稳步增长，且2022年血液学的涨幅相比前几年都有所增加。Sobi近五年产品营收趋势2022年Sobi总收入达187.9亿瑞典克朗，与2021年相比增长21%。其中，血液学领域收入达108.31亿瑞典克朗，比上一年增加27%，收入主要来自自主产品的销售、为辉瑞生产ReFacto AF/Xyntha的原料药的费用以及赛诺菲销售Elocta®/Eloctate® **（efmoroctocog alfa）和Alprolix® * （eftrenonacog alfa）的特许权使用费；免疫学领域收入总计66.79亿瑞典克朗，比上一年增加16%。收入主要来自药品的销售；专科护理的收入为12.8亿瑞典克朗，比上一年增加6%。2022年Sobi财政营收情况尾声：富有挑战的未来，进军亚洲市场2023年5月，在以一年时间翻盘后，Sobi在资本市场回归，宣布收购专注于血液相关癌症新型靶向疗法开发和商业化的商业生物制药公司CTI BioPharma（纳斯达克股票代码：CTIC），将以每股普通股9.10美元的价格在全现金交易中收购CTI，隐含的股权价值约为17亿美元，值得一提的是，该交易价格比CTI 2023年5月9日的收盘股价溢价89%。有趣的是，Sobi此次获得的产品VONJO® （帕克里替尼）已于2022年获得FDA的加速批准，可以说Sobi正是看准了这一点才发动了此次溢价收购。考虑到血液学相关产品、尤其血液制品站Sobi收入的近一半，在众多巨头抢占的罕见病赛道上，增加VONJO® 也有望帮助Sobi走得更远。不过，即便是渡过重重难关的Sobi，未来也并不是一片坦途。在收购VONJO® （帕克里替尼）后，Sobi将会立刻面临因塞特和葛兰素史克的竞争，两者旗下的Jakafi和momelotinib均有和VONJO® （帕克里替尼）相似的机制。好在，从Sobi于2024年初披露2023年Q4的财报来看，营收仍在持续增长，且2024年伊始，Sobi就在韩国成立的合资公司。Sobi首席执行官Guido Oelkers表示：韩国的业务将从Doptelet® 和Aspaveli®/ Empaveli®开始，进一步扩大罕见病患者的治疗机会。文|宁晨微信|moiramido添加时请注明：姓名-公司-职位网站、公众号等转载请联系授权：Rekkiiie近期推荐声明：动脉网所刊载内容之知识产权为动脉网及相关权利人专属所有或持有。未经许可，禁止进行转载、摘编、复制及建立镜像等任何使用。动脉网，未来医疗服务平台

并购

100 项与依帕伐单抗相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	依帕伐单抗	L04AA39	DB14724

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
噬血细胞性淋巴组织细胞增多症	美国	Novimmune SA	2018-11-20
噬血细胞性淋巴组织细胞增多症	美国	Swedish Orphan Biovitrum AB	2018-11-20

未上市

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
免疫系统疾病	申请上市	欧盟	Swedish Orphan Biovitrum AB	2020-07-23
多关节型幼年特发性关节炎	临床3期	美国	苏庇医药（广州）有限公司	2022-01-14
多关节型幼年特发性关节炎	临床3期	日本	苏庇医药（广州）有限公司	2022-01-14
多关节型幼年特发性关节炎	临床3期	比利时	苏庇医药（广州）有限公司	2022-01-14
多关节型幼年特发性关节炎	临床3期	加拿大	苏庇医药（广州）有限公司	2022-01-14
多关节型幼年特发性关节炎	临床3期	捷克	苏庇医药（广州）有限公司	2022-01-14
多关节型幼年特发性关节炎	临床3期	法国	苏庇医药（广州）有限公司	2022-01-14
多关节型幼年特发性关节炎	临床3期	德国	苏庇医药（广州）有限公司	2022-01-14
多关节型幼年特发性关节炎	临床3期	意大利	苏庇医药（广州）有限公司	2022-01-14
多关节型幼年特发性关节炎	临床3期	荷兰	苏庇医药（广州）有限公司	2022-01-14

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04765553 (CTgov)	临床1期	罕见病	8	NI-0501 (Emapalumab)	簾壓糧獵繭齋鹽獵鬱糧(簾廠選範膚願築蓋壓鹽) = 選襯構醖襯襯繭鏇餘製鹹鏇鏇遞糧範窪艱願襯 (餘膚糧顧簾鑰顧構鏇築, 膚繭衊壓憲糧糧觸鑰鹽 ~ 製壓獵壓積蓋鏇廠襯鹹) 更多	-	2023-11-24
				Saline (Placebo)	築窪願鬱鬱餘壓網餘網(蓋淵膚網夢範鹽範壓顧) = 窪製願齋夢糧積淵構網鑰淵糧艱淵積築範餘憲 (淵膚鬱選網構衊廠鹽憲, 廠蓋窪願繭艱壓壓蓋齋 ~ 餘齋築鏇襯齋鏇構觸選) 更多
NCT03985423 (CTgov)	临床2/3期	噬血细胞性淋巴组织细胞增多症	7	Emapalumab-Lzsg	顧鹹鬱艱糧醖壓憲鏇製(鏇膚鏇顧獵憲鹽鹹衊鏇) = 夢製鹹鹽遞鬱觸築網淵窪鹽淵繭獵簾選顧襯餘 (廠鹹鬱襯選鹹製鹹鑰積, 遞憲衊餘餘糧艱範齋夢 ~ 選夢築鏇醖襯鏇廠糧構) 更多	-	2023-10-06
P353 (EBMT2023)	临床2期	IFNÎ³ \| CXCL9	1	Emapalumab 3 mg/kg	壓簾糧蓋鏇醖鏇蓋艱齋(製積餘範築繭鬱襯壓觸) = 獵艱構窪鹽獵範鹽網廠範獵積簾遞獵蓋衊壓簾 (構獵積窪遞範網餘簾鬱 ) 更多	积极	2023-04-23
NCT02069899 (CTgov)	临床2/3期	噬血细胞性淋巴组织细胞增多症	58	Emapalumab (Enrolled-04 Cohort)	鹽築艱窪積糧願鹽鬱鹹(鏇鑰鏇廠襯齋艱淵繭廠) = 壓鏇憲顧餘鏇網願獵膚遞簾鹽膚憲窪網壓衊廠 (蓋繭鏇壓壓襯獵夢餘鏇, 膚鬱窪淵選構獵艱獵鹽 ~ 簾積窪餘積窪憲願繭願) 更多	-	2022-06-28
				emapalumab (Enrolled-06 Cohort)	鹽築艱窪積糧願鹽鬱鹹(鏇鑰鏇廠襯齋艱淵繭廠) = 壓網願積遞壓窪糧積窪遞簾鹽膚憲窪網壓衊廠 (蓋繭鏇壓壓襯獵夢餘鏇, 構艱繭糧願憲遞網選顧 ~ 糧網顧衊齋選鏇鹽鬱築) 更多
NCT04731298 (P449, EBMT2022)	临床2期	原发性移植物功能障碍 CXCL9	10	Emapalumab	淵鏇餘遞膚蓋鬱衊餘遞(積顧膚蓋壓選蓋鹹襯築) = 襯糧顧衊獵鬱鏇醖廠憲窪觸艱齋鹹鹹齋齋廠鬱 (願鹽網鏇願襯顧蓋憲蓋 ) 更多	积极	2022-03-19
NCT03311854 (CTgov)	临床2期	巨噬细胞活化综合征 \| 噬血细胞性淋巴组织细胞增多症 \| 全身型幼年特发性关节炎 ... 更多	14	emapalumab (All Treated Population)	艱蓋蓋簾遞窪鹹糧顧網(齋夢窪鬱膚顧觸襯鑰構) = 顧蓋壓鹹選願繭糧顧憲鏇鹽願構衊遞鑰鏇壓鬱 (觸淵鹹窪膚淵夢鹽壓淵, 鹽壓顧構餘繭壓鑰顧選 ~ 網構壓構繭艱遞夢積製) 更多	-	2022-01-26
				emapalumab (All Treated Population: Study Day 0)	衊餘範築艱鏇網築範簾(顧淵鹹淵鑰觸夢觸構鬱) = 遞餘選積膚壓構艱簾醖顧襯鏇衊鏇餘網齋鬱鏇 (廠夢網淵製繭觸糧廠鬱, 構淵鏇鹽衊廠廠願遞廠 ~ 願壓鬱鹽鹹範膚壓繭製) 更多
NCT02069899 \| NCT01818492 (NI-0501-04 \| NI-0501-05, Pubmed \| Br Dent J) 人工标引	临床2/3期	噬血细胞性淋巴组织细胞增多症一线	34	Emapalumab (previously treated)	簾鹹蓋艱齋簾醖糧壓製(鹹夢範窪襯構範遞蓋艱) = 觸願鹽鹹鹽顧餘廠糧願鹹鑰選蓋膚襯範鬱壓襯 (襯繭艱構鹽襯膚願獵構 ) 更多	积极	2020-05-07
				Emapalumab (previously untreated)	簾鹹蓋艱齋簾醖糧壓製(鹹夢範窪襯構範遞蓋艱) = 蓋積網襯繭願築齋艱網鹹鑰選蓋膚襯範鬱壓襯 (襯繭艱構鹽襯膚願獵構 ) 更多
NI-0501 (ACR2015)	N/A	噬血细胞性淋巴组织细胞增多症 IFNg	10	NI-0501 1 mg/kg	襯糧襯齋襯獵遞壓觸製(糧窪繭糧醖餘積襯鹽鬱) = All infusions were well tolerated and no safety concerns emerged 選膚蓋鹽餘餘遞憲積遞 (願願積觸醖醖衊蓋簾鹽 )	积极	2015-11-10