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

CTR20222253 / Active, not recruiting临床4期

一项描述依马利尤单抗用于治疗原发性噬血细胞性淋巴组织细胞增多症中国经治患者的安全性和有效性的开放性、单臂、多中心、上市后研究

本研究主要目的是收集依马利尤单抗在经治pHLH中国患者中的安全性数据。本研究次要目的是收集依马利尤单抗在经治pHLH中国患者中的有效性数据。

开始日期2023-01-29

申办/合作机构

Patheon Italia SpA

[+2]

EUCTR2009-018146-38-FR / Unknown statusN/A

Multicenter randomized controlled study comparing the efficacy and safety of natural Multi-subtype interferon alpha (Multiferon) in association with ribavirin versus retreatment with pegylated interferon-alpha 2a and ribavirin in non-responding genotype 1 hepatitis C patients. - Must Have

开始日期2010-05-28

申办/合作机构

Swedish Orphan Biovitrum International AB

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0 项与 Biovitrum AB 相关的专利（医药）

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项与 Biovitrum AB 相关的文献（医药）

2024-07-01·ADVANCES IN THERAPY

florio® HAEMO: A Longitudinal Survey of Patient Preference, Adherence and Wearable Functionality in Central Europe

Article

作者: Boban, Ana ; Batorova, Angelika ; Máchal, Jan ; Zapotocka, Ester ; Zombori, Marianna ; Zupan, Irena Preloznik ; Laguna, Pawel ; Puras, Gediminas ; Bilic, Ernest ; Kotnik, Barbara Faganel ; Nemes, Laszlo ; Ettingshausen, Carmen Escuriola ; Hrdlickova, Radomira

INTRODUCTION:

florio^® HAEMO is a hemophilia treatment monitoring application (app) offering activity tracking and wearable device connectivity. Its use might support everyday activities for people with hemophilia. The aim of this study was to evaluate user satisfaction, long-term usage and the impact on data entry when pairing a wearable with a hemophilia monitoring app.

METHODS:

This is a follow-up of a two-part user survey conducted in Central Europe. People with hemophilia and parents/caregivers of children with hemophilia using florio HAEMO and who completed part one were invited to complete a second online questionnaire at least 4 months later.

RESULTS:

Fifty participants (83.3%) who completed part one of the survey continued to use the florio HAEMO app and completed part two. Of 14 participants who chose to use the app with a wearable, more than half (57.1%) were aged between 13 and 25 years. Overall, the results demonstrated that florio HAEMO is very easy or rather easy to use, especially for individuals pairing the app with a wearable. Most people using a wearable indicated that florio HAEMO was very or rather important in bringing certainty to daily activities (85.7%). Notably, 14 of 36 (38.9%) non-wearable users indicated that they would prefer to pair the app with a wearable in the future.

CONCLUSIONS:

Adherence to the florio HAEMO app is maintained over an extended period of use. Pairing the app with a wearable might enable easier access to app features, increase data entry motivation and provide more certainty about daily activities for people with hemophilia.

2023-08-01·Research and practice in thrombosis and haemostasis

Recombinant factor IX Fc for major surgery in hemophilia B: factor IX plasma activity levels and effective hemostasis

Article

作者: Shapiro, Amy D ; Ozelo, Margareth C ; Ahlin, Helena ; Chambost, Hervé ; Santagostino, Elena ; Casiano, Sandra ; Falk, Aletta

Background:

Major surgical procedures are associated with significant bleeding risk and infectious complications in patients with hemophilia, which may be minimized by factor replacement. Monitoring perioperative factor levels guides dosing to maintain adequate levels for hemostatic control.

Objectives:

We report prospectively collected post hoc surgical data in patients with hemophilia B who underwent major surgery with extended half-life recombinant factor IX Fc fusion protein (rFIXFc) in phase 3 studies (B-LONG/Kids B-LONG and B-YOND).

Methods:

Achieved FIX plasma levels were described for those who underwent major surgeries with ≥1 peak and/or predose FIX assessment available on the day of surgery (Day 0 [D0]) from the central laboratory. Dosing, injection frequency, adverse events, and hemostatic responses were assessed. Two representative cases were described further including blood loss, transfusions, and concomitant medication assessment.

Results:

Of 35 major surgeries, 17 (N = 16 subjects) with sufficient FIX measurements were included in this analysis; 13 of 17 surgeries were orthopedic. On D0, a median loading (preoperative) dose of 101.1 International Units (IU)/kg/injection achieved a median peak FIX of 103.3 IU/dL. Across postoperative Days 1 to 3, 4 to 6, and 7 to 14, the median predose levels were 75.1 IU/dL with 1 injection/d, 71.6 IU/dL with 0 to 1 injection/d, and 43.2 IU/dL with 0 to 1 injection/d, respectively. Hemostasis was rated excellent (14 of 16) or good (2 of 16) across surgeries. Both case studies (knee arthroscopy and ankle fusion) illustrate measured FIX levels with rFIXFc.

Conclusion:

The aggregate analysis and representative cases of major surgeries demonstrate that rFIXFc can achieve FIX levels for effective hemostasis during invasive high-risk procedures.

Patient preference and adherence3区 · 医学

A Cross-National Survey of People Living with Hemophilia: Impact on Daily Living and Patient Education in Central Europe

3区 · 医学

ArticleOA

作者: Faganel Kotnik, Barbara ; Puras, Gediminas ; Batorova, Angelika ; Banchev, Atanas ; Kiss, Csongor ; Zupancic-Salek, Silva ; Zapotocka, Ester

BACKGROUND:

Information about the impact of hemophilia on daily living and information preferences for patients and their caregivers in Central Europe has been limited.

METHODS:

This cross-national survey was conducted between April 1 and October 15, 2020 and utilized a self-administered questionnaire to collect data (Typeform™) from people living with hemophilia in Bulgaria, Croatia, Czech Republic, Hungary, Slovakia and Slovenia. The questionnaire included 22 questions regarding difficulties in daily life and preferences for receiving hemophilia-related information. Respondents were stratified into two main groups, people with hemophilia (PwH) or their caregivers (CPwH). Results were analyzed using descriptive statistics.

RESULTS:

Of the 364 respondents, 232 were PwH (63.7%) and 132 were CPwH (36.3%). In total, 70.3% of hemophilia patients/caregivers responded that they are kept sufficiently informed about life with hemophilia, with 68.0%, 59.1% and 56.3% of respondents obtaining information from their physicians, patient associations and via digital media (internet and social media), respectively. However, 97.8% of respondents expressed an interest in additional information, particularly new hemophilia treatment options (62.1%), which in contrast to other topics was indicated most frequently by both patients and caregivers in all six countries. Most frequent difficulties in everyday life with hemophilia were identified as mobility problems (41.8%), unexpected bleeding (38.5%), pain (35.4%), and uncertainty with what they can or cannot do (25.0%). During the 2020 COVID-19 pandemic, 52.5% of respondents reported that they did not experience any major change in daily living with hemophilia.

CONCLUSION:

Based on our Central European survey, hemophilia mostly affects peoples' lives by causing mobility difficulties, unexpected bleeding, pain and uncertainty in daily activities. Although the majority of respondents reported being educated about hemophilia, most PwH and CPwH respondents sought additional information, highlighting the need for continuous personalized patient education to cope with present challenges.

项与 Biovitrum AB 相关的新闻（医药）

2024-10-18

·echemi

Merck's New Therapy Only Reduces Infant RSV Infections by 60.4%, Hospitalization Rate Decline of 84.2% Raises Safety Concerns

Merck announced Thursday that its investigational antibody treatment, clesrovimab, demonstrated a 60.4 percent effectiveness in reducing infections associated with a specific type of respiratory syncytial virus (RSV) in infants. This seasonal injection regimen successfully achieved its primary goal in the mid to late phase of the trial, significantly reducing the incidence of medically care-required lower respiratory tract infections due to RSV in healthy infants under 1 year of age during the first RSV season. Merck further noted that clesrovimab also significantly reduced RSV-related hospitalizations by as much as 84.2% compared to placebo. The rate of side effects caused by the drug during treatment was comparable to that of the placebo group, and there were no deaths directly related to treatment or RSV during the study period. Merck previously announced in July that the vaccine had met the main goal of the study, but did not disclose details at the time. If approved, clesrovimab will compete in the U.S. market with Sanofi's (SASY.PA) and Astrazeneca's (AZN.L) Beyfortus, also an antibody therapy that prevents RSV infections in infants and children and requires a quarterly injection. In a separate late-stage study, Beyfortus was able to reduce the incidence of lower respiratory infections in infants requiring medical care by 74.5%. In addition, Synagis from Swedish orphan drug company Biovitrum (SOBIV.ST) is available as a treatment to prevent RSV, requiring monthly injections. Merck reported on Thursday that clesrovimab was on par with Synagis in terms of safety, a conclusion based on interim results from another late-stage study. Notably, Pfizer's RSV vaccine Abrysvo is already approved for use in pregnant women and is designed to prevent RSV infection in babies during the first six months of life. RSV often causes cold-like symptoms and is one of the leading causes of pneumonia and death in infants and the elderly. In the United States, the virus causes about 177,000 hospitalizations and 14,000 deaths each year.

疫苗临床结果上市批准

2024-09-24

·CPHI制药在线

补体药物进军C3肾小球病，诺华伊普可泮纳入优先审评，Pegcetacoplan III期临床成功

关注并星标CPHI制药在线 9月23日，诺华的「盐酸伊普可泮胶囊」被CDE拟纳入优先审评，用于治疗成人C3肾小球病（C3G）。伊普可泮（Iptacopan，Fabhalta）是一款同类首创的补体因子B（FB）抑制剂，通过与补体旁路途径中的FB结合，调节C3的裂解、下游效应物的产生和末端途径的放大，控制C3b介导的血管外溶血和末端补体介导的血管内溶血。目前，伊普可泮在美国已获批两项适应症：阵发性睡眠性血红蛋白尿症（PNH）（2023.12)和IgA肾病(2024.08)。伊普可泮市场表现不错，2024上半年销售额达2800万美元。在国内，伊普可泮于2024年4月被NMPA批准治疗成人PNH，2024年8月在国内申报第二项适应症。C3肾小球病是伊普可泮在国内申报的第三项适应症，也是伊普可泮该适应症首次在全球申请上市。若顺利，伊普可泮有望成为第一个针对C3G替代补体途径的潜在治疗方法。已公布的3期临床试验APPEAR-C3G中期结果显示：与安慰剂加支持治疗相比，接受伊普可泮加支持治疗的C3G患者在6个月时蛋白尿（通过 24 小时尿蛋白与肌酐比值 [UPCR] 测量）减少了35.1% (p=0.0014)，结果具有临床意义和统计学意义。而且，次要终点肾小球滤过率 (eGFR)数据表明，与安慰剂相比，6 个月期间数值改善了 +2.2 mL/min/1.73 m2 (p=0.1945)。安全性方面，伊普可泮具有良好的安全性，没有新的安全信号。此外，伊普可泮还被开发用于治疗IgA肾病、膜性肾病、非典型溶血尿毒综合征、重症肌无力等。 C3肾小球病治疗现状 C3肾小球病（C3G）是一类由于先天性遗传变异及获得性自身抗体介导的补体旁路途径异常活化导致的罕见肾小球疾病，主要包括致密物沉积病（DDD）和 C3 肾小球肾炎（C3 GN）两种类型。据2020年公布的数据，估计全球每100万人中只有1到2例C3G患者。在C3G中，替代补体通路的过度激活会导致 C3 蛋白沉积在肾小球中积聚，引发炎症和肾小球损伤，导致蛋白尿、血尿和肾功能下降。约50%的C3G患者在诊断后10年内进展为肾衰竭，此时他们将需要透析和/或肾移植，超过55%的C3G患者在移植后经历疾病复发。目前，C3G尚无特异性治疗方法，临床上主要以延缓肾脏病进展为目的，如血浆置换、免疫抑制、肾移植和抗C5抗体(依库珠单抗)及对症支持治疗(如肾素-血管紧张素抑制剂、利尿剂、降脂、营养支持等)。多款在研，Pegcetacoplan明年递交C3G上市申请除了伊普可泮，全球还有多款药物被开发用于治疗C3G，详见下表。在研药物绝大多数属于补体药物，但药物类型多样，除了化药，还包括单抗、双抗、环肽、siRNA。研发进度上，大多处于I、II期临床。具体品种来看，Pegcetacoplan、SGB-9768今年在C3G领域取得新进展。其中Pegcetacoplan是Apellis Pharmaceuticals开发的一款补体C3抑制剂，已被FDA批准用于治疗PNH、年龄相关性黄斑变性（AMD）继发地图样萎缩（GA）。Pegcetacoplan市场表现不错，据Apellis财报其2024上半年在美国的销售额约为3.422亿美元。 2024年8月，Pegcetacoplan治疗C3G或原发性免疫复合物介导的膜增生性肾小球肾炎（IC-MPGN）的III期VALIANT研究取得积极结果。该研究是一项多中心、随机、双盲、安慰剂对照临床试验（n=124），旨在评估 Pegcetacoplan（1080mg，每周2次）治疗12岁及以上的C3G或原发性IC-MPGN的疗效和安全性。该研究达到主要终点，即Pegcetacoplan组患者的蛋白尿相比安慰剂组减少了68%（p<0.0001）。而且，所有亚组的趋势一致，包括C3G和IC-MPGN、青少年和成年患者以及自体肾脏和移植后肾脏亚组。此外，Pegcetacoplan还在关键性次要终点上显示出统计学显著性，包括结合蛋白尿减少和估算肾小球滤过率（eGFR）稳定的复合肾功能终点，以及蛋白尿与基线相比减少至少50%的患者比例。安全性方面，Pegcetacoplan在研究中表现出良好的安全性和耐受性，与既往情况一致。基于上述研究积极结果，Apellis Pharmaceuticals计划于2025年初向FDA提交Pegcetacoplan的补充新药申请（sNDA），苏庇医药计划于2025年向欧洲药品管理局（EMA）提交该药物的上市申请。 SGB-9768是圣因生物开发的一种靶向补体C3蛋白的siRNA-GalNAc结合物，其采用圣因生物独特创新的新一代LEAD™ GalNAc技术递送到肝脏细胞，通过RNAi抑制肝脏C3的合成。临床前研究数据显示，SGB-9768能有效并持续地减少C3合成。此外，SGB-9768可实现每3个月或6个月给药一次的频率，具有治疗频率较低、患者依从性好、药效持久的优势。 2024年6月，SGB-9768在国内获批临床试验，用于治疗补体介导的肾脏疾病，包括成人IgA肾病、C3G、免疫复合物介导的膜增生性肾小球肾炎患者。总结作为一种罕见肾小球疾病，C3肾小球病（C3G）治疗缺乏特异性治疗药物。鉴于补体系统异常或过度活化在C3G、IgA肾病等自免疾病中发挥重要作用，多款补体药物被开发用于治疗C3G。其中诺华补体因子B（FB）靶向药伊普可泮在国内率先递交治疗C3G的新适应症上市申请，有望成为首个治疗C3G的补体药物。此外，补体C3靶向药Pegcetacoplan治疗C3G的III期临床试验也已取得积极结果，预计明年将递交治疗C3G的新药补充申请。参考资料： 1.《潜在重磅疗法达到3期临床终点，明年初提交监管申请》.药明康德.2024年08月10日 2.《靶向补体C3，圣因生物RNAi疗法获批临床》.医药观澜.2024年06月25日 END 来源：CPHI制药在线声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。投稿邮箱：Kelly.Xiao@imsinoexpo.com ▼更多制药资讯，请关注CPHI制药在线▼ 点击阅读原文，进入智药研习社~

优先审批临床3期申请上市

2024-08-15

·Insight数据库

月度分析丨2024 年 7 月全球新药月度报告-分析篇（附下载通道）

据 Insight 数据库统计，7 月全球获批上市 75 款新药（其中包括 5 款生物类似药和 21 款改良新药），申请上市 78 款新药（其中包括 19 款改良新和 8 款生物类似药）。具体清单见「全球新药月报-数据篇」，可进入下载平台内免费下载！ 7 月份全球新药热点资讯 FDA 批准礼来新药 Donanemab：阿尔茨海默病治疗领域取得重大突破；苏庇医药滚动提交 SEL-212 的生物制品许可申请，难治性痛风迎来新选择； NewAmsterdam 口服小分子 CETP 抑制剂 Obicetrapib 结果披露，有望改变 HeFH 治疗格局；礼来斥资 32 亿美元并购 Morphic，持续加强 IBD 管线；基于真实可溯源的全球医药数据，丁香园 Insight 数据库每月推出「全球新药月报」，以期帮助业内人士通盘把握每月全球范围内的新药开发情况。报告内容节选试阅

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药物(靶点)	适应症	全球最高研发状态

尼替西农 ( 4HPPD )	酪氨酸血症更多	批准上市
安纳白介素 ( IL1R1 )	成人斯蒂尔病更多	批准上市
马来酸阿伐曲泊帕 ( TPO receptor )	免疫性血小板减少症更多	批准上市
Pegcetacoplan ( C3 )	阵发性睡眠性血红蛋白尿症更多	批准上市
依马利尤单抗 ( IFNγ )	多关节型幼年特发性关节炎更多	临床2/3期