Damoctocog Alfa Pegol

药械追踪No.1 / GSK玛贝兰妥单抗在日本获批用于多发性骨髓瘤2025年5月19日，葛兰素史克（LSE/NYSE：GSK）宣布，其产品Blenrep（玛贝兰妥单抗）联合疗法已获得日本厚生劳动省（MHLW）批准上市，用于治疗复发或难治性多发性骨髓瘤成人患者。该批准基于DREAMM-7和DREAMM-8两项Ⅲ期临床试验的积极结果。这些试验分别评估了Blenrep联合硼替佐米加地塞米松（BVd）以及联合泊马度胺加地塞米松（BPd）的疗效，研究对象为至少接受过一次既往治疗的多发性骨髓瘤患者。Blenrep是一种抗体偶联药物（ADC），由人源化BCMA单克隆抗体与细胞毒性药物甲硫氨酸通过不可切割的连接子结合而成。该药物连接子技术授权自Seagen公司；单克隆抗体则使用了BioWa公司（Kyowa Kirin集团成员）授权的POTELLIGENT技术生产。2025年4月，英国药品和健康产品管理局（MHRA）批准了Blenrep联合疗法，用于治疗至少接受过一次既往治疗的复发或难治性多发性骨髓瘤成人患者。->点击文末阅读原文，解锁完整双语新闻No.2 / 拜耳Jivi获FDA批准拓展适应证至7-12岁A型血友病儿童患者2025年5月20日，拜耳宣布，Jivi已获美国食品药品监督管理局（FDA）批准用于7岁及以上患有A型血友病（先天性凝血因子VIII缺乏症）的儿童患者。Jivi是一种通过重组DNA技术制备的长效凝血因子VIII浓缩剂，此次获批基于Alfa-PROTECT和PROTECT Kids研究的数据。两项研究证实，Jivi在7至12岁严重A型血友病儿童中具有良好的安全性和有效性。此前，Jivi于2018年8月首次获得FDA批准，用于12岁及以上已接受过治疗的A型血友病患者，包括按需治疗和控制出血发作、围手术期出血管理、常规预防以减少出血发作频率。->点击文末阅读原文，解锁完整双语新闻企业动态No.1 / 逾60亿美元！辉瑞获得三生制药双抗SSGJ-707独家许可2025年5月20日，辉瑞（NYSE:PFE）与三生制药集团（1530.HK）达成全球独家许可协议，辉瑞将获得其子公司三生国健（688336.SH）研发的PD-1/VEGF双特异性抗体SSGJ-707在除中国大陆以外的全球独家开发、生产和商业化权利。根据协议条款，三生制药有望获得12.5亿美元首付款，并有资格获得最高48亿美元的开发、监管批准和销售里程碑付款，以及根据授权地区产品销售额计算得到的两位数百分比的梯度销售分成。预计该交易将于第三季度完成，交易完成后，辉瑞将根据双方达成的证券认购协议，向三生制药进行1亿美元的股权投资。SSGJ-707是一款靶向PD-1和VEGF的双特异性抗体，目前正在中国进行多项临床试验，用于治疗非小细胞肺癌、转移性结直肠癌和妇科肿瘤。无论单药还是与化疗联用，SSGJ-707均展示出显著的抗肿瘤活性和良好的安全性，具有同类最优的潜力。目前，SSGJ-707单药一线治疗非小细胞肺癌适应证已进入Ⅲ期临床阶段，联合化疗一线治疗非小细胞肺癌适应证处于临床Ⅱ期阶段、转移性结直肠癌适应证处于临床Ⅱ期阶段，晚期妇科肿瘤适应证处于临床Ⅱ期阶段。->点击文末阅读原文，解锁完整双语新闻No.2 / 8.95亿美元！靖因药业与CRISPR Therapeutics达成siRNA开发合作2025年5月20日，靖因药业与CRISPR Therapeutics（Nasdaq：CRSP）达成战略合作，将共同开发并商业化下一代长效Factor Ⅺ（FⅪ）靶向小干扰RNA（siRNA）疗法SRSD107，用于治疗血栓及血栓栓塞性疾病。根据协议条款，靖因药业将获得CRISPR Therapeutics支付的9500万美元现金及现金等价物作为首付款，并有资格获得超8亿美元的预付款和里程碑付款。双方将对半分摊成本和利润，共同开发SRSD107。此外，协议还授予CRISPR Therapeutics在未来独家引进最多两个额外siRNA项目的优先授权权利。CRISPR将负责该产品在美国的商业化，靖因药业则负责大中华区市场。SRSD107是一款新一代的长效siRNA疗法，旨在选择性抑制凝血因子Ⅺ（FⅪ）。FⅪ靶点在病理性血栓形成中起关键作用，但对正常止血功能的影响较小。通过靶向FⅪ，SRSD107有望在降低血栓事件发生的同时，显著减少出血风险，展现出有别与凝血因子Ⅹ活性（FⅩa）抑制剂的治疗优势。SRSD107的潜在适应证广泛，包括房颤、静脉血栓栓塞症（VTE）、肿瘤相关性血栓、接受血液透析的终末期肾病患者，以及因出血风险限制现有治疗手段的重大骨科手术人群。目前，SRSD107已完成两项Ⅰ期临床试验，结果显示单次给药安全性且耐受性良好。相关研究成果已在2025年美国心脏病学会（ACC）年会和2024年美国血液学会（ASH）年会上发布。SRSD107的Ⅱ期临床试验正在启动，旨在评估其在膝关节置换术患者中预防静脉血栓栓塞症（VTE）的安全性和有效性。->点击文末阅读原文，解锁完整双语新闻No.3 / 2.56亿美元！再生元收购23andMe资产，Lemonaid Health业务除外2025年5月19日，再生元（Nasdaq：REGN）宣布，已成功中标23andMe Holding Co.（Nasdaq：ME）的破产拍卖，将收购该公司几乎所有资产。再生元计划以2.56亿美元的价格收购23andMe的个人基因组服务（PGS）、全面健康和研究服务业务线，以及其生物样本库及相关资产，并确保23andMe的所有消费级基因组服务不间断地继续开展。该交易预计将在2025年第三季度完成，但需获得破产法院和监管机构的批准以及其他惯例的交割条件。23andMe将作为再生元的全资直接或间接子公司运营，并继续作为个人基因组服务提供商开展业务。Regeneron的收购不包括23andMe的Lemonaid Health业务。Lemonaid Health是一个在线按需获取医疗和药房服务的虚拟平台。2021年10月22日，23andMe宣布与Lemonaid Health, Inc.达成最终合并协议，以4亿美元收购Lemonaid Health，为23andMe的消费者业务增加了Lemonaid Health的创新远程医疗和处方药配送服务。2025年1月，据媒体报道，23andMe正在考虑出售Lemonaid Health业务。23andMe成立于2006年，2021年上市，市值巅峰曾达到60亿美元。成立至今，23andMe从未盈利过，截至2024年12月31日，23andMe的现金和现金等价物只剩7940万美元。2025年3月，23andMe申请破产保护，此前曾尝试私有化和寻求收购，但都未能成功。->点击文末阅读原文，解锁完整双语新闻No.4 / 默沙东/第一三共在研ADC I-DXd启动食管鳞癌III期临试2025年5月20日，默沙东（NYSE：MRK）与合作伙伴第一三共（TSE：4568）联合开发的潜在同类首创B7-H3靶向抗体偶联药物（ADC）Ifinatamab Deruxtecan（简称I-DXd）已启动III期IDeate-Esophageal01研究。IDeate-Esophageal01研究旨在评估I-DXd对比研究者选择的化疗方案，在含铂方案和免疫检查点抑制剂经治后疾病进展的不可切除晚期或转移性食管鳞状细胞癌（ESCC）患者中的疗效与安全性。目前，该研究已完成首例患者给药。IDeate-Esophageal01研究的启动基于I/II期IDeate-PanTumor01研究的积极数据，IDeate-PanTumor01显示，I-DXd在多线经治的ESCC患者中具有显著疗效。->点击文末阅读原文，解锁完整双语新闻全球医疗情报领导者解锁隐藏在数据中的商业潜力 关于 G B I”自从2002年成立以来，GBI始终以技术为驱动，为药企、器械及行业相关服务商提供贯穿生命周期的全球药品市场竞争数据、全球行业资讯、HCPs洞察、全国医疗器械数据等商业信息与洞察，助力企业在进行战略布局和决策时，脱颖而出。历经20余年的深耕细作GBI已成为95%以上跨国药企、国内头部药企、咨询与投资机构等医疗圈灯塔用户值得信赖的长期合作伙伴。联系我们投稿 | 发稿 | 媒体合作▶ olivia.xu@generalbiologic.com数据库 | 咨询服务 | 资讯追踪▶ 点击左下“阅读原文”完成表单填写点击阅读原文，解锁完整双语新闻

WHIPPANY, N.J.--(BUSINESS WIRE)--Bayer announced that the U.S. Food and Drug Administration (FDA) has approved Jivi®, a recombinant DNA-derived, extended half-life factor VIII concentrate, for use in pediatric patients 7 years of age and older with hemophilia A (congenital Factor VIII deficiency). The approval is based on data from the Alfa-PROTECT and PROTECT Kids studies, demonstrating Jivi’s safety and efficacy in children aged 7 to under 12 years with severe hemophilia A. This significant milestone reflects Bayer’s ongoing commitment to providing effective treatment options for the hemophilia community. Jivi was first granted approval by the FDA in August 2018 for use in previously treated adults 12 years of age and older with hemophilia A for: On-demand treatment and control of bleeding episodes Perioperative management of bleeding Routine prophylaxis to reduce the frequency of bleeding episodes. Jivi is not indicated for use in: Children <7 years of age due to a greater risk for hypersensitivity reactions and/or loss of efficacy. Previously untreated patients (PUPs). Treatment of von Willebrand disease. "Bayer remains dedicated to meeting the needs of the hemophilia A community," stated Jessica Charlet, Scientific Director. “The FDA’s approval for pediatric patients 7 to <12 years of age is reflective of Bayer’s commitment to the hemophilia A community and underscores our commitment to supporting families through their treatment journey.” Bayer continues to prioritize support for the hemophilia community, and we are excited to offer Jivi as a treatment option for patients and their families. About Jivi® [antihemophilic factor (recombinant), PEGylated-aucl] Jivi is approved for the routine prophylactic treatment of hemophilia A in previously treated adult and pediatric patients 7 years of age or older. Jivi’s initial recommended dosing for children 7 to under 12 years of age is 60 IU/kg twice weekly. Adjust the dose based on the patient’s clinical response and/or recovery. The recommended initial dosage regimen for adults and adolescents is (30-40 IU/kg) twice weekly with the ability to dose every five days (45-60 IU/kg) and further individually adjust to less or more frequent dosing based on bleeding episodes. Jivi is also approved for on-demand treatment and the perioperative management of bleeding in the same population.1 To learn more about Jivi, visit www.jivi.com. INDICATION JIVI ® is a recombinant DNA-derived, Factor VIII concentrate indicated for use in previously treated adults and pediatric patients 7 years of age and older with hemophilia A (congenital Factor VIII deficiency) for: On-demand treatment and control of bleeding episodes. Perioperative management of bleeding. Routine prophylaxis to reduce the frequency of bleeding episodes. On-demand treatment and control of bleeding episodes. Perioperative management of bleeding. Routine prophylaxis to reduce the frequency of bleeding episodes. Limitations of use JIVI is not indicated for use in: Children <7 years of age due to a greater risk for hypersensitivity reactions and/or loss of efficacy. Previously untreated patients (PUPs). Treatment of von Willebrand disease. Children <7 years of age due to a greater risk for hypersensitivity reactions and/or loss of efficacy. Previously untreated patients (PUPs). Treatment of von Willebrand disease. IMPORTANT SAFETY INFORMATION JIVI is contraindicated in patients who have a history of hypersensitivity reactions to the active substance, polyethylene glycol (PEG), mouse or hamster proteins, or other constituents of the product. Hypersensitivity reactions, including severe allergic reactions, have occurred with JIVI. Monitor patients for hypersensitivity symptoms. Early signs of hypersensitivity reactions, which can progress to anaphylaxis, may include chest or throat tightness, dizziness, mild hypotension and nausea. If hypersensitivity reactions occur, immediately discontinue administration and initiate appropriate treatment. JIVI may contain trace amounts of mouse and hamster proteins. Patients treated with this product may develop hypersensitivity to these non-human mammalian proteins. Hypersensitivity reactions may also be related to antibodies against polyethylene glycol (PEG). Neutralizing antibody (inhibitor) formation has occurred following administration of JIVI. Carefully monitor patients for development of Factor VIII inhibitors, using appropriate clinical observations and laboratory tests. If expected plasma Factor VIII activity levels are not attained or if bleeding is not controlled as expected with administered dose, suspect the presence of an inhibitor (neutralizing antibody). An immune response associated with IgM anti-PEG antibodies, manifested as symptoms of acute hypersensitivity and/or loss of drug effect, has occurred with JIVI administration. In the clinical trials, the IgM anti-PEG antibodies disappeared within 4-6 weeks. No immunoglobulin class switching from IgM to IgG has been observed. A low post-infusion Factor VIII level, in absence of detectable Factor VIII inhibitors, may be due to loss of treatment effect related to high titers of anti-PEG IgM antibodies. In these cases, discontinue JIVI and switch patients to a different anti-hemophilic product. A reduced recovery of Factor VIII after start of JIVI treatment may be due to transient low titers of anti-PEG IgM antibodies. In these cases, increase the dose of JIVI until recovery of Factor VIII returns to expected levels. The most common (incidence ≥5%) adverse reactions in clinical trials in previously treated patients (PTPs) ≥7 years of age were headache, fever, cough, and abdominal pain. For additional important risk and use information, please see the full Prescribing Information. About Alfa-PROTECT Study A multi-center, prospective, single-arm, study to evaluate the safety of Jivi infusions for prophylaxis and treatment of bleeding in previously treated pediatric patients 7 to <12 years of age with severe hemophilia A. The study evaluated the potential risk of hypersensitivity and loss of drug effect associated with an immune response to polyethylene glycol (PEG) during the first 4 exposure days to Jivi. A total of 35 patients with a median age of 8 years (range: 7 to 11) were enrolled and treated prophylactically for a minimum of 50 EDs and 26 weeks. The patients received a treatment regimen of Jivi twice weekly (40-60 IU/kg) at investigator’s discretion. Thirty-two patients completed the treatment phase and were offered continuation in an 18 month extension study. A key secondary endpoint was the annualized bleed rate (ABR) About PROTECT Kids Study A multi-center, prospective, single-arm trial to evaluate the pharmacokinetics, safety, and efficacy of Jivi for prophylaxis and treatment of bleeding in previously treated pediatric patients <12 years of age with severe hemophilia A. The primary efficacy endpoint was the annualized bleed rate (ABR). About Hemophilia A Hemophilia affects approximately 400,000 people around the world and is a largely inherited disorder in which one of the proteins needed to form blood clots is missing or reduced. Hemophilia A is the most common type of hemophilia, in which blood clotting is impaired because there is a lack or defect of coagulation FVIII. Patients repeatedly experience bleeds in muscles, joints or other tissues, which can result in chronic joint damage over time. Injuries can have severe consequences if not treated appropriately, as the blood clots more slowly in hemophilia patients than in healthy individuals. Based on a study that used data collected on patients receiving care in federally funded hemophilia treatment centers during the period 2012–2018, about 33,000 males in the United States are living with the disorder.2 About Bayer Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, “Health for all, Hunger for none,” the company’s products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2024, the Group employed around 93,000 people and had sales of 46.6 billion euros. R&D expenses amounted to 6.2 billion euros. © 2025 Bayer BAYER, the Bayer Cross and Jivi are registered trademarks of Bayer. Forward-Looking Statements This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer’s public reports which are available on the Bayer website at www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to confirm them to future events or developments. References: 1. Jivi® [antihemophilic factor (recombinant), PEGylated-aucl] [Prescribing Information]. Whippany, NJ: Bayer 2025 2. Centers for Disease Control and Prevention. About Hemophilia. Last Reviewed: May 2025. https://www.cdc.gov/hemophilia/about/index.html PP-JIV-US-2365- 1 May 2025