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Routine Prophylaxis Now Recommended for Adults and Children Aged 6 and Older with Severe VWD PARAMUS, N.J., Dec. 5, 2023 /PRNewswire/ -- Octapharma USA announced the expansion of the U.S. Food and Drug Administration (FDA) approval for wilate®, von Willebrand Factor/Coagulation Factor VIII Complex (Human) Lyophilized Powder for Solution for Intravenous Injection. The approved label now includes routine prophylaxis aimed at reducing the frequency of bleeding episodes in adults and children aged 6 and older with any type of von Willebrand disease (VWD), the most prevalent bleeding disorder in the United States. Wilate® is the first von Willebrand factor (VWF) concentrate indicated for prophylactic treatment across all forms of VWD, marking a significant milestone in the field. Continue Reading Octapharma USA: FDA Grants Expanded Approval to wilate® as the First VWF Concentrate for Prophylaxis in All Types of VWD Post this Octapharma USA announced the expansion of the U.S. Food and Drug Administration approval for wilate®, von Willebrand Factor/Coagulation Factor VIII Complex (Human) Lyophilized Powder for Solution for Intravenous Injection. The approved label now includes routine prophylaxis aimed at reducing the frequency of bleeding episodes in adults and children aged 6 and older with any type of von Willebrand disease (VWD), the most prevalent bleeding disorder in the United States. "Long-term prophylaxis with VWF concentrate, as compared to on-demand treatment for bleeding, is recommended for patients with severe VWD," according to Shveta Gupta, MD, a specialist in pediatric hematology and oncology with The Haley Center for Children's Cancer and Blood Disorders at Orlando Health Arnold Palmer Hospital for Children. "The approval of wilate® for VWD prophylaxis is a welcome new treatment option that can be life-saving for many patients. Increased use of VWF prophylaxis in VWD patients may lead to improved patient care and a reduced burden of disease." WIL-31 Study: A Breakthrough in VWD Treatment The FDA approval is supported by Octapharma's WIL-31 study, a prospective, non-controlled, international, multicenter phase 3 trial that investigated the efficacy and safety of wilate® prophylaxis over 12 months in people aged 6 and older with severe VWD of any type. All WIL-31 patients received on-demand treatment with wilate® during a previous six-month, prospective, observational study (WIL-29). Patients who experienced at least six bleeding episodes (BEs), excluding menstrual bleeds, with at least two of these BEs treated with a VWF-containing product, were eligible to enter WIL-31. Patients in WIL-31 received wilate® prophylaxis two to three times per week at a dose of 20-40 IU/kg, for 12 months. The clinical trial's primary purpose was to investigate whether prophylaxis with wilate® lowered the mean total annualized bleeding rate (ABR) by more than 50% compared to the six months of on-demand treatment. Secondary goals were to measure spontaneous ABR and treatment-emergent adverse events.1 Researchers reported an 84% reduction in the mean total ABR compared with on-demand treatment during the prior study. The median spontaneous ABR decreased by 95%. Importantly, no serious drug-related adverse events or thrombotic events were observed during the study. Addressing the Prevalence of VWD in the U.S. Population Von Willebrand disease affects up to 1% of the U.S. population, equating to about 3.3 million individuals. The disease, occurring equally among men and women, is often noticed more by women due to heavy or abnormal bleeding during menstrual periods and after childbirth. VWD includes three major types – Type 1, Type 2, and Type 3, the most severe form. The expanded wilate® label provides a treatment option for a significant patient population.2 "Wilate® prophylaxis for adults and children aged 6 and older with VWD promises to be life-changing for many patients," stated Octapharma USA President Flemming Nielsen. "Patients have been forced to live with far too many bleeding episodes while receiving on-demand treatment. Octapharma is pleased to offer patients a new therapy option that can greatly improve their quality of life." Factor My Way: Empowering VWD Patients In tandem with this approval, Octapharma USA extends its commitment to patient care through the Factor My Way patient support program. Tailored for those living with VWD and hemophilia A, this free membership program provides access to caregivers, educational programs, a resource library, and more. For additional information, please visit FactorMyWay.com. About wilate ® Wilate®, von Willebrand Factor/Coagulation Factor VIII Complex (Human) Lyophilized Powder for Solution for Intravenous Injection is indicated in children and adults with von Willebrand disease for: On-demand treatment and control of bleeding episodes Perioperative management of bleeding Routine prophylaxis to reduce the frequency of bleeding episodes Wilate® is indicated for routine prophylaxis in children 6 years of age and older and adults with von Willebrand disease. Wilate® is indicated in adolescents and adults with hemophilia A for: Routine prophylaxis to reduce the frequency of bleeding episodes On-demand treatment and control of bleeding episodes CONTRAINDICATIONS Do not use in patients with known hypersensitivity reactions, including anaphylactic or severe systemic reaction, to human plasma-derived products, any ingredient in the formulation, or components of the container. WARNINGS AND PRECAUTIONS Anaphylaxis and severe hypersensitivity reactions are possible. Thromboembolic events may occur. Monitor plasma levels of FVIII activity. Development of neutralizing antibodies to FVIII and to VWF, especially in VWD type 3 patients, may occur. Wilate® is made from human plasma and carries the risk of transmitting infectious agents. For complete prescribing information, please visit wilateusa.com/pi. About the Octapharma Group 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: Hematology, Immunotherapy 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. The company's American subsidiary, Octapharma USA, is located in Paramus, N.J. For more information, please visit octapharmausa.com. REFERENCES 1 – Robert F. Sidonio, Jr., Ana Boban, Leonid Dubey, Adlette Inati, Csongor Kiss, Toshko Lissitchkov, Dzmitry Novik, Elina Peteva, Ali T. Taher, Margarita Arkadevna Timofeeva, Kateryna V. Vilchevska, Vladimir Vdovin, Sylvia Werner, Sigurd Knaub, Claudia Khayat; Efficacy and Safety of Prophylaxis with a Plasma-Derived Von Willebrand Factor/ Factor VIII Concentrate in Previously Treated Patients with Von Willebrand Disease. Blood 2022; 140 (Supplement 1): 8438–8439. doi: . 2 – Centers for Disease Control and Prevention, What is von Willebrand Disease. SOURCE Octapharma

//  针对血友病的基因治疗，是武田在该疾病领域中的又一块重要的拼图。越来越频繁的授权交易，预示着跨国药企与中国新药公司之间的合作已逐渐进入深水区。一周前，BMS宣布与联拓生物达成一项治疗肥厚型心肌病药物的交易。实际上，今年以来，AZ、GSK 、强生、罗氏等多家跨国药企纷纷与中国新药公司开展合作。随着一批本土创新企业的崛起与成熟，跨国药企看到了扎根中国，乃至领跑全球的新机遇。作为罕见病领域的全球领先者，武田制药也一直在积极寻找中国的合作伙伴。最近，武田中国与本土创新型企业信念医药（Belief BioMed，BBM）达成合作协议，信念医药授权武田中国负责治疗B型血友病的创新基因疗法BBM-H901注射液在中国大陆、中国香港和中国澳门的商业化。根据协议，武田将负责该产品在中国的市场策略、销售策略以及医学事务等方面的商业化工作。基因疗法  罕见病患者的新希望对于普通人来说，罕见病可能只是某部影视作品中的一颗催泪弹，但对于全球3亿的罕见病患者而言，疾病的痛苦与负担终身相伴。正因如此，罕见病领域的创新疗法一直被寄予厚望。2018年，第一款RNAi疗法上市，让该疗法在罕见病领域的治疗备受期待。就在今年9月，FDA批准了一款RNAi疗法，用于治疗9岁及以上儿童和成人1型原发性高草酸尿症(PH1)。基因治疗是罕见病患者的另一道曙光。近年来，罕见病基因治疗的研发热度持续攀升，2022年，FDA共批准了3款治疗罕见病的基因疗法。分别用于治疗β-地中海贫血、肾上腺脑白质营养不良和血友病。值得关注的是，全球范围内，针对血友病基因疗法的研发，正在开启该疾病领域的新篇章。血友病作为一种遗传性凝血功能障碍疾病，分为A和B两种亚型。其中B型血友病是由于血液中缺乏凝血因子Ⅸ(FIX)所致，患者绝大部分为男性，其特点为反复的、自发性的关节、肌肉和软组织出血，如长时间反复出血，还可能导致患者关节畸形、肌肉萎缩甚至死亡。目前，预防和按需使用凝血因子替代疗法是血友病的标准治疗方案。尽管这种治疗方式可以让疾病得到一定程度的缓解，但终身反复注射，不仅严重影响患者的生活质量，也带来了巨大的治疗负担。随着基因疗法的不断成熟，血友病患者看到了新的希望。与传统治疗方式相比，理论上，基因治疗药物具有“一次给药，长期有效”的优势，可以让血友病患者体内凝血因子水平长期维持在临床治愈水平，同时能减少注射次数以及规避使用血液产品带来的感染风险。加快落地  让更多中国患者获益此次武田中国与信念医药达成授权合作的BBM-H901注射液，是一款由信念医药自主研发的腺相关病毒（AAV）基因治疗药物，也是国内第一个获批进入注册临床试验的、治疗B型血友病的AAV基因治疗药物，于2022年先后登上国际权威期刊《柳叶刀-血液病学》和《新英格兰医学杂志》，已被国家药监局药品审评中心纳入“突破性治疗品种”名单。已发表临床研究数据显示，BBM-H901注射液具有良好的安全性和有效性，AAV基因治疗药物输注后患者体内凝血因子水平提高显著，并长期稳定，且在临床期间未发现明显的不良反应，患者的年化出血率（ABR）显著降低。《柳叶刀-血液病学》曾刊登的临床研究结果表明，18岁以上、FIX残留水平≤2IU/ dl的血友病B患者接受BBM-H901注射液治疗后，中位58周随访显示出“3个0”的临床获益：患者中位年化出血率从12次减少至0次，中位靶关节数从1.5个减少至0个，中位凝血因子IX药物年输注次数从53.5次减少至0次。据统计，目前我国注册登记的B型血友病患者约3800人，这些患者仍然背负着沉重的治疗负担和长久的疾病困扰。中国在B型血友病领域的治疗仍有着未被满足的需求。显然，此番武田中国与信念医药的合作，有望加速基因疗法在中国的落地，帮助更多患者回归正常生活。武田中国表示，公司商业化方面有非常成熟的经验，一旦药物获得国家药品监督管理局（NMPA）批准，将利用自身在罕见病领域的商业化规模和专业知识，将该创新疗法带给中国患者。同时，双方将结合各自优势和资源，推动B型血友病基因疗法在中国的普及，帮助更多B型血友病患者长期获益于领先的基因疗法，提升罕见病创新药的可及性。拓展产品组合  持续深耕中国作为全球罕见病领域的代表性企业，多年来，武田加速推动了多款罕见病创新药物进入中国。包括治疗I型戈谢病的维葡瑞、全球首个针对遗传性血管性水肿（HAE）预防治疗的达泽优和用于治疗急性发作的飞泽优、治疗法布雷病的瑞普佳等。血友病是武田在罕见血液疾病领域的重要组成部分，公司持续引入先进预防性治疗与个体化治疗方案，包括用于血友病预防治疗的百因止（基因重组凝血因子Ⅷ）、血友病个体化治疗管理软件myPKFiT。此外，用于治疗获得性血友病A的Obizur和治疗血管性血友病的Vonvendi也已在中国递交上市申请。而针对血友病的基因治疗，可以说是武田在该疾病领域中的又一块重要的拼图。与信念医药的合作，将进一步丰富武田在罕见病领域的产品组合，也让其在罕见病创新疗法上的突破，站上了新的高度。武田相信，其在罕见病领域的专业知识和丰富经验，将能助力中国本土创新的开花结果，同时也将在未来推动更多创新方案在中国落地，为中国的创新市场注入更多活力，最终在实现患者获益，也为“健康中国2030”贡献武田力量。编辑 | 姚嘉yao.jia@PharmaDJ.com 总第1990期访问研发客网站可浏览更多文章www.PharmaDJ.com