Von Willebrand factor/coagulation factor VIII complex(Octapharma AG)

WIL-31 is the largest prospective prophylaxis study in VWD WIL-31 is the only study in VWD with a prospective on-demand run-in study as an intra-individual comparator wilate® prophylaxis is highly effective at reducing bleeding rates in children and adults with all types of VWD vs prior on-demand treatment LACHEN, Switzerland, Jan. 22, 2024 /PRNewswire/ -- Octapharma announced today the publication of the results from the clinical study WIL-31, which investigated the efficacy and safety of prophylaxis with wilate ®, a von Willebrand factor/factor VIII (VWF/FVIII) concentrate, in patients with all types of von Willebrand disease (VWD). The results have been published in the leading medical journal Blood Advances [Sidonio RF Jr et al. "Von Willebrand factor/factor VIII concentrate (wilate®) prophylaxis in children and adults with von Willebrand disease"] The lead author and Principal Investigator of the WIL-31 study, Robert F. Sidonio Jr, M.D. (Medical Director of Hemophilia at the Aflac Cancer & Blood Disorders Center with Children's Healthcare of Atlanta and Associate Professor of Pediatrics at Emory University School of Medicine), commented: "WIL-31 is the largest prospective study to date examining the efficacy and safety of regular prophylaxis in patients with VWD. Importantly, the study included a diverse patient population, including young children and adults, males and females, and all types of VWD. The findings from WIL-31 provide strong evidence to support the use of VWF prophylaxis and have led to the recent addition of wilate ® prophylaxis as a therapeutic indication for VWD in the US, widening its access to the US patient population." The WIL-31 study (NCT04052698) was a prospective, multinational, open-label, non-controlled phase III study assessing wilate ® prophylaxis in preventing bleeds in VWD. Efficacy was assessed in 33 adult and pediatric VWD patients aged 6 years and older from 14 centers in eight countries, with patients receiving wilate ® prophylaxis two or three times weekly for 12 months. All patients had taken part in a prospective 6-month run-in study (WIL-29) during which they received on-demand treatment with any available VWF concentrate, allowing assessment of the efficacy of prophylaxis at an intra-individual level. The primary endpoint of the study, >50% reduction in mean total annualized bleeding rate (ABR) during wilate® prophylaxis compared with prior on-demand treatment, was met. Mean total ABR during wilate® prophylaxis was 5.2 compared with 33.4 during on-demand treatment, representing a decrease of 84%. Mean spontaneous ABR decreased by 87% during wilate® prophylaxis compared with on-demand treatment. Reductions in total and spontaneous bleeding were comparable across age groups, bleeding sites and VWD disease types, and between males and females. The majority of patients were dosed twice weekly and the median weekly dose of wilate® was relatively low (58 IU/kg) compared to the dose range recommended in the prescribing information. Seven women with child-bearing potential were included in the study. This allowed the investigators to gain insights into the effect of wilate® prophylaxis on heavy menstrual bleeding, which is a major problem in women with VWD as there are limited clinical data on effective treatments. Although the data are based on a small sample size, they suggest that wilate® prophylaxis has a positive impact in reducing heavy menstrual bleeding, with 71% (n=5) patients not experiencing heavy menstrual bleeding during wilate® prophylaxis. Prophylaxis with wilate® was well tolerated across VWD types, in children and adults, and in males and females. During the 12 months of wilate® prophylaxis, there were no serious adverse events related to wilate® and no thrombotic events or FVIII accumulation. "In contrast to haemophilia A, prophylaxis is not standard of care in VWD and less than 10% of patients with severe VWD receive prophylaxis", said Sigurd Knaub, SVP CR&D Haematology. "This is due in part to the scarcity of data from prospective trials. The results of the WIL-31 study help to fill this gap, providing strong evidence for the benefits of a prophylactic strategy in patients with severe VWD. Increasing the adoption of prophylaxis in VWD is expected to lead to improved outcomes and reduced disease burden for people suffering from VWD." Octapharma would like to thank all participating centers, as well as the patients and their caregivers, for their contribution to the study. Further publications on other analyses from the WIL-31 study are planned. About wilate® wilate® is a high-purity human von Willebrand factor/factor VIII (VWF/FVIII) concentrate, that undergoes two virus inactivation steps during its production1. No albumin is added as a stabiliser1. The purification processes result in a 1:1 ratio of VWF to FVIII that is similar to normal plasma1. wilate® contains a VWF triplet structure and content of large high molecular weight multimers similar to normal human plasma1. wilate® is available in 500 IU and 1,000 IU presentations2. wilate® is indicated for the prevention and treatment of haemorrhage or surgical bleeding in von Willebrand disease (VWD), when desmopressin (DDAVP) alone is ineffective or contra-indicated, and for the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency)2. The indication for prophylaxis in the US is detailed in the updated prescribing information available here: wilate Full Prescribing Information (wilateusa.com) About Octapharma Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein manufacturers in the world, developing and producing human proteins from human plasma and human cell lines. Octapharma employs more than 11,000 people worldwide to support the treatment of patients in 118 countries with products across three therapeutic areas: Immunotherapy, Hematology, and Critical Care. Octapharma has seven R&D sites and five state-of-the-art manufacturing facilities in Austria, France, Germany and Sweden, and operates more than 190 plasma donation centers across Europe and the US. Octapharma has 40 years of experience in patient care. Octapharma press releases are specifically for health specialist/medical media and are not for consumer press. Contact: Ivana Spotakova Global Communications Manager Ivana.Spotakova@octapharma.com +41 79 3474607 References Stadler M et al. Biologicals 2006; 34:281–8. wilate® Summary of Product Characteristics 2023. Logo :

Octapharma 将在 2024 年 2 月 6 日至 9 日于德国法兰克福举行的欧洲血友病及相关疾病协会 (EAHAD) 第 17 届年会上公布临床和科学数据，以推进对出血性疾病的理解和治疗 Octapharma 将公布 wilate ® 和 Nuwiq ® 的最新临床和科研成果 顶尖专家将在一场卫星研讨会、一次口头报告和六个海报展示中介绍有关血管性血友病 (VWD) 和 A 型血友病的新发现 展示内容将包括 wilate ® 对血管性血友病 (VWD) 患者的预防疗效和安全性的新数据 瑞士拉亨 2024年1月19日 /美通社/ -- 在 EAHAD 2024 上展示 wilate ® WIL-31 是 VWD 领域最大规模的前瞻性预防研究，其中包含按需进行的磨合研究，用于患者个体内的比较。该研究表明，wilate ® 预防疗法在降低各种类型 VWD 儿童和成人患者的出血率方面疗效卓著，而且适用于所有已知的出血部位。研究结果为 VWD 患者采用常规预防疗法提供了有力证据。 "WIL-31 研究的新数据为在所有类型的 VWD 患者中使用 wilate ® 预防疗法提供了有力证据。这些研究结果促使美国批准 wilate ® 预防用途，为这些患者提供了更多的治疗选择。"– IBU Haematology 高级副总裁兼负责人 Larisa Belyanskaya。 Octapharma 的 卫星研讨会 "转变 VWD 预防范式：聚焦 WIL-31 研究"将深入探讨 WIL-31 研究的发现： Jan Astermark（瑞典）将探讨相较于 A 型血友病，VWD 预防性治疗不足的问题 Robert F. Sidonio Jr. （美国）将阐述 WIL-31 的研究结果何以对 VWD 预防性治疗的现状造成冲击 将以 WIL-31 研究中的三个互动病例介绍 wilate ® 预防疗法的疗效： 由来自亚特兰大的 Robert F. Sidonio Jr. 介绍儿童 VWD 病患 由来自萨格勒布的 Ana Boban 介绍经常流鼻血的成人 由来自德布勒森的 Csongor Kiss 介绍经期大量出血的女性 卫星研讨会将于 欧洲中部时间 2 月 7 日（星期三） 17:30-18:45 在 Panorama Room 2 举行。 Ana Boban 将于 欧洲中部时间 2 月 9 日（星期五） 08:30-10:00 在 SLAM 第 6 分会场 发表题为"使用血浆来源的血管性血友病因子/凝血因子 VIII 浓缩剂进行常规预防，可有效减少血管性血友病儿童和成人患者的鼻出血" (OR04) 的口头 SLAM 报告，阐述预防 VWD 患者频繁流鼻血的重要性以及 wilate ® 在这种情况下的预防效果。 两个海报展示提供对 WIL-31 研究结果的更多看法： PO213 ：在 WIL-31 研究中，在使用血浆来源的血管性血友病因子/凝血因子 VIII 浓缩剂进行预防治疗期间，凝血因子 VIII 和血管性血友病因子没有蓄积 PO194 ：在 WIL-31 研究中，在使用血浆来源的血管性血友病因子/凝血因子 VIII 浓缩剂进行预防治疗期间，使用抑制剂治疗血管性血友病 在 EAHAD 2024 上展示 Nuwiq ® 除了在凝血级联中的作用外，新的证据表明 FVIII 还会影响血小板和内皮细胞的功能。使用重组 FVIII (rFVIII) 浓缩剂进行替代治疗已成为治疗 A 型血友病患者的主要方法，但目前尚不清楚 rFVIII 浓缩剂的改性对血小板和内皮细胞功能的影响是否存在差异。与其他 rFVIII 浓缩剂相比，Nuwiq ® 在体外试验中与活化血小板的结合程度更高，而在另一项研究中，Nuwiq ® 比其他 rFVIII 浓缩剂对内皮细胞功能的影响更大： PO027 ：重组因子 VIII 浓缩剂与血小板的不同结合对血小板功能的影响 PO024 ：研究凝血因子 VIII 在内皮细胞功能中的作用 对于 A 型血友病等罕见疾病，无法在临床研究中直接比较治疗方法。在这种情况下，可以采用间接治疗比较方法来比较不同治疗方法的效果。其中匹配调整间接比较法 (MAIC) 是一种行之有效的间接比较方法。Nuwiq ® 以药代动力学为指导的个性化预防治疗与其他治疗方法的疗效已采用 MAIC 进行比较： PO041 ：在 A 型血友病患者中，对使用 simoctocog alfa 的个性化预防与使用 efanesoctocog alfa 的标准预防进行匹配调整的间接比较 PO042 ：在 A 型血友病患者中，对使用 simoctocog alfa 的个性化预防与使用 emicizumab 的标准预防进行匹配调整的间接比较 "Octapharma 的核心价值观是全力改善患者的生活，支持临床和科学研究项目是我们实现这一承诺的必要手段。我们很高兴能在 EAHAD 2024 上展示这些研究成果。"—Octapharma 董事会成员兼国际业务部负责人 Olaf Walter。 关于 Octapharma Octapharma 公司总部位于瑞士拉亨，是全球最大的人类蛋白质生产商之一，主要开发和生产源于人类血浆和人类细胞系的人类蛋白。 Octapharma 在全球拥有 11,000 多名员工，通过免疫疗法、血液学和重症监护三大治疗领域的产品，为 118 个国家\/地区的患者提供治疗支持。 Octapharma 在奥地利、法国、德国和瑞典拥有七个研发基地和五个先进生产设施，并在欧洲和美国设立 190 多个血浆捐赠中心。Octapharma 在患者护理方面积累了 40 年的丰富经验。 关于 Nuwiq ® Nuwiq ® (simoctocog alfa) 是第 4 代重组因子 VIII (rFVIII) 蛋白，源自人类细胞系，未经化学修饰或与任何其他蛋白融合 1。在培养过程中不含人源性或动物源性添加剂。不含非人类蛋白抗原表位，与血管性血友病因子具有高度亲和性 1。Nuwiq ® 治疗已在九项 1-3 已完成的临床试验中进行了评估，包括 201 名接受过治疗（190 人） 1 、108 名未接受过治疗 2 的重症 A 型血友病患者。Nuwiq ® 提供 250 IU、500 IU、1,000 IU、1,500 IU、2,000 IU、2,500 IU、3,000 IU 和 4,000 IU 八种规格 4。Nuwiq ® 已获准用于治疗和预防各年龄组 A 型血友病（先天性 FVIII 缺乏）患者的出血 4。 关于 wilate ® wilate ® 是一种高纯度人类血管性血友病因子\/凝血因子 VIII (VWF\/FVIII) 浓缩剂，在生产过程中采用双重病毒灭活步骤 5。未添加白蛋白作为稳定剂 5。经过纯化过程，VWF 与 FVIII 的比率达到与普通血浆近似的 1:1 5。wilate ® 含有一种 VWF 三重结构以及与普通人血浆类似的大分子量多聚体含量 5。wilate ®提供 500 IU 和 1000 IU 两种规格。 6wilate ® 在仅使用去氨加压素 (DDAVP) 无效或存在使用禁忌时，用于预防和治疗血管性血友病 (VWD) 患者的大出血或手术出血，同时还用于治疗和预防 A 型血友病（先天性 FVIII 缺乏）患者的出血 6。美国预防性治疗的适应症详见更新后的处方信息： wilate 完整处方信息 (wilateusa.com) Octapharma 新闻稿专供健康专家\/医疗媒体使用，不面向消费者媒体。 参考文献： 1.  Lissitchkov T et al. Ther Adv Hematol 2019; 10:2040620719858471. 2.  Liesner RJ et al.Thromb Haemost 2021; 121:1400-8. 3.  Octapharma AG; Data on file. 4.  Nuwiq ® Summary of Product Characteristics. 5.  Stadler M et al. Biologicals 2006; 34:281-8. 6.  wilate ® Summary of Product Characteristics. 联系方式： Ivana Spotakova +41 79 3474607 Ivana.Spotakova@octapharma. com