Garadacimab-gxii

ANDEMBRY inhibits the top of the HAE cascade by targeting factor XIIa and provides sustained protection from attacks Once-monthly dosing reduced HAE attacks by a median of more than 99 percent and a least squares mean of 89.2 percent, compared to placebo ANDEMBRY approval expands CSL's HAE franchise and underscores the company's legacy of delivering transformational innovations to the HAE community for over four decades KING OF PRUSSIA, Pa., June 16, 2025 /PRNewswire/ -- Global biotechnology leader CSL (ASX:CSL; USOTC:CSLLY) today announced the U.S. Food and Drug Administration (FDA) approved ANDEMBRY® (garadacimab-gxii), the only treatment targeting factor XIIa for prophylactic use to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older. By targeting factor XIIa, a plasma protein that plays a key role in attacks of swelling in people with HAE, ANDEMBRY inhibits the top of the HAE cascade to prevent HAE attacks. ANDEMBRY, the only treatment to offer once-monthly dosing from the start for all patients, is a subcutaneous self-injection delivered in 15 seconds or less via an autoinjector with a citrate-free formula. HAE is a rare, chronic, and potentially life-threatening genetic disorder characterized by recurrent and unpredictable attacks of angioedema. Attacks of HAE are often painful and can affect multiple sites of the body, including the abdomen, larynx, face, and extremities. HAE occurs in about 1 in 50,000 people of any ethnic group. "ANDEMBRY, the first monoclonal antibody discovered and developed entirely by CSL, offers people living with this life-threatening condition long-term control over their disease along with a convenient administration method," said Bill Mezzanotte, MD, Executive Vice President, Head of R&D, CSL. "ANDEMBRY underscores our long-standing and enduring commitment to better the lives of the patients we serve, including those suffering with HAE. I'd like to thank all the physicians, patients and my colleagues who contributed to this exciting milestone for HAE patients and CSL." The approval is supported by data from the pivotal placebo-controlled Phase 3 VANGUARD trial evaluating the efficacy and safety of ANDEMBRY. The pivotal study (The Lancet, April 2023) demonstrated that treatment with ANDEMBRY: Led to 62 percent of ANDEMBRY-treated patients remaining attack-free throughout the treatment period. Reduced HAE attacks by a median of more than 99 percent and a least squares mean of 89.2 percent, compared to placebo. Achieved more than 99 percent median reduction and an 88 percent mean reduction in HAE attacks requiring on-demand therapy, compared to placebo. Attained more than 99 percent median reduction and a 90 percent mean reduction in moderate or severe attacks, compared to placebo. The most common adverse reactions in the pivotal trial (incidence ≥7%) are nasopharyngitis and abdominal pain. A published interim analysis (Allergy, Oct 2024) of the ongoing open-label extension study (median ANDEMBRY exposure of 13.8 months) showed that ANDEMBRY has a favorable long-term safety profile and provides sustained reductions in HAE attacks. In the pivotal trial and the open-label extension study, injection-site reactions (e.g., injection-site bruising, injection-site erythema, injection-site hematoma, injection-site pruritus, injection-site urticaria) were reported in 23 (14%) patients. "We've made significant progress in treating hereditary angioedema, yet many patients still experience painful and sometimes life-threatening HAE attacks and require frequent injections to manage them," said Dr. Tim Craig, Professor of Medicine, Pediatrics and Biomedical Sciences at Penn State University. "We now have a new option to manage this condition through a new target, as it allows us for the first time to inhibit the top of the HAE cascade by targeting factor XIIa." "ANDEMBRY, a novel once-monthly subcutaneous treatment that inhibits factor XIIa, is a welcome addition to the HAE treatment landscape," said Anthony J. Castaldo, CEO and Chairman of the Board, US HAE Association and HAE International. "People with HAE now have another choice for lessening the burden associated with this lifelong condition and realizing the community's shared goal of experiencing life to the fullest." This regulatory approval for ANDEMBRY is another crucial step in building toward the global availability of ANDEMBRY, which was recently approved in Australia, the United Kingdon (UK), the European Union (EU), Japan, Switzerland, and United Arab Emirates. CSL Behring will launch ANDEMBRY commercially immediately, with availability before the end of June. Healthcare professionals and patients interested in learning more about ANDEMBRY or accessing the therapy are encouraged to utilize ANDEMBRY ConnectSM, designed to offer comprehensive support and assistance through various programs. For more information, call 844-423-4273 or visit . About HAE HAE is a rare and potentially life-threatening genetic condition that occurs in about 1 in 10,000 to 1 in 50,000 people. HAE is caused by deficient or dysfunctional C1INH, a protein in the blood that helps to control inflammation. Inadequate amounts of properly functioning C1INH can lead to the accumulation of fluid in body tissues, causing considerable swelling referred to as angioedema. HAE attacks can affect many parts of the body, including the face, abdomen, larynx, and extremities. Patients who have abdominal attacks of HAE can experience extreme pain, diarrhea, nausea, and vomiting caused by swelling of the intestinal wall. HAE attacks that involve the face or throat can result in airway closure, asphyxiation and, if left untreated, death. About ANDEMBRY ANDEMBRY is a novel monoclonal antibody inhibiting factor XIIa (anti-FXIIa mAb) that has completed the Phase 3 pivotal study as a new type of once-monthly subcutaneous prophylactic treatment for attacks related to HAE, a form of bradykinin-mediated angioedema. ANDEMBRY is CSL's first homegrown recombinant monoclonal antibody to gain FDA approval. It was discovered and optimized by scientists at CSL's Bio21-based research site, with formulation and manufacturing for the clinical programs completed at the CSL Broadmeadows Biotech Manufacturing Facility. ANDEMBRY uniquely inhibits the plasma protein, FXIIa. FXII is the first protein activated in the HAE pathway, initiating the cascade of events leading to an HAE attack. By targeting activated FXII (FXIIa), ANDEMBRY inhibits this cascade at the top as compared to other HAE therapies that target downstream mediators. About the VANGUARD Trial The multicenter, randomized, double-blind, parallel-group VANGUARD trial evaluated the efficacy and safety of ANDEMBRY, an investigational first-in-class monoclonal antibody, as a prophylactic treatment for patients with hereditary angioedema. Patients aged 12 years and older with HAE type I or II underwent screening and a run-in study period to verify a baseline attack rate. Patients were randomized 3:2 to receive a loading dose of 400 mg followed by 200 mg of ANDEMBRY monthly (n=39) or volume matched placebo monthly (n=25) subcutaneously. After the six-month treatment period, patients were given the opportunity to continue into the open-label extension study, which is currently ongoing. The ongoing open-label extension of the Phase 3 VANGUARD study is evaluating the long-term safety and efficacy of ANDEMBRY (200 mg monthly) for the prophylactic treatment of hereditary angioedema attacks. IMPORTANT SAFETY INFORMATION What is ANDEMBRY? ANDEMBRY® (garadacimab-gxii) injection, for subcutaneous use, is a prescription medication used to prevent attacks of hereditary angioedema (HAE) in people 12 years and older. It is not known if ANDEMBRY is safe and effective in children under 12 years of age. What should I tell my healthcare provider before using ANDEMBRY? Before using ANDEMBRY, tell your healthcare provider about any medical condition you may have, especially if you are pregnant, planning to become pregnant, breastfeeding, or planning to breastfeed. It is not known if ANDEMBRY can harm your unborn baby or if ANDEMBRY passes into breastmilk. Talk to your healthcare provider about the best way to feed your baby while using ANDEMBRY. Tell your healthcare provider about all medications you take, including prescription medicines, over-the-counter treatments, vitamins, and herbal supplements. How should I use ANDEMBRY? Use ANDEMBRY exactly as instructed by your healthcare provider. Detailed instructions for use can be found in the patient information section of the full prescribing information. ANDEMBRY is given as an injection under your skin (subcutaneous) by you or a caregiver. Your healthcare provider should show you or your caregiver how to prepare and inject your dose of ANDEMBRY before you inject yourself for the first time. Do not try to inject ANDEMBRY unless you have been trained by your healthcare provider. What are the possible side effects of ANDEMBRY? The most common side effects of ANDEMBRY include: Redness, itchiness, and bruising (injection-site reactions) Stomach (abdominal) pain Runny or stuffy nose, sneezing, watery eyes (nasopharyngitis) Please see full prescribing information for ANDEMBRY, including patient information and instructions for use. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit , or call 1-800-FDA-1088. You can also report side effects to CSL Behring's Pharmacovigilance Department at 1-866-915-6958. About CSL CSL (ASX:CSL; USOTC:CSLLY) is a global biotechnology company with a dynamic portfolio of lifesaving medicines, including those that treat haemophilia and immune deficiencies, vaccines to prevent influenza, and therapies in iron deficiency and nephrology. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL – including our three businesses: CSL Behring, CSL Seqirus and CSL Vifor – provides lifesaving products to patients in more than 100 countries and employs 32,000 people. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest. For more information about CSL, visit . Media Contacts Valerie Bomberger CSL Office: +1 610-291-5388 Mobile: +1 267-280-3829 Email: [email protected] Etanjalie Ayala CSL Behring Mobile: +1 610 297 1069 Email: [email protected] Greg Healy CSL Mobile: +1 610-906-4564 Email: [email protected] In Australia: Kim O'Donohue CSL Mobile: +61 449 884 603 Email: [email protected] Brett Foley CSL Mobile: +61 461 464 708 Email: [email protected] Investors Contact Chris Cooper CSL Mobile: +61 455 022 740 Email: [email protected] SOURCE CSL WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

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Therapeutics，布局基因治疗；同年还以29亿美元收购MorphoSys，增强血液肿瘤领域优势。百时美施贵宝2024年营收：483亿美元去年，百时美施贵宝的多款创新疗法获得监管突破与临床应用拓展。在血液肿瘤领域，公司3月获得CD19靶向CAR-T疗法Breyanzi加速批准，用于治疗复发/难治性慢性淋巴细胞白血病和小淋巴细胞淋巴瘤（SLL），并于5月扩展至滤泡性淋巴瘤（FL）和套细胞淋巴瘤（MCL）适应症；PD-1抑制剂Opdivo则在3月获批联合化疗用于尿路上皮癌一线治疗，10月进一步扩展至可切除NSCLC围手术期治疗，其皮下注射剂型Opdivo Qvantig更于年底获批，将给药时间缩短至5分钟。靶向BCMA的CAR-T疗法Abecma则于4月获批用于多发性骨髓瘤，在临床试验中，该疗法将无进展生存期提高至标准疗法的三倍以上；酪氨酸激酶抑制剂（TKI）Augtyro和KRAS G12C抑制剂Krazati分别在6月获加速批准用于实体瘤和结直肠癌治疗；神经精神领域则迎来突破——Cobenfy成为近十年来首个获批治疗精神分裂症的新机制药物。研发合作方面，公司通过战略合作强化管线布局：年初与VantAI合作开发分子胶新靶点，与Immunocore合作探索TCR双特异疗法IMC-F106C联合Opdivo治疗黑色素瘤的疗效和安全性；10月携手Prime Medicine开发下一代T细胞疗法；年底则与BioArcti达成全球许可协议，推进靶向焦谷氨酸修饰β淀粉样蛋白的抗体项目开发。礼来2024年营收：450亿美元2024年，礼来在多个疾病领域实现关键监管突破：5月，RET激酶抑制剂Retevmo获批用于治疗2岁及以上RET基因变异相关实体瘤及甲状腺癌患儿，成为首款覆盖12岁以下RET突变儿童的靶向疗法，6月，该药加速批准转为完全批准用于治疗RET融合阳性甲状腺癌全年龄段患者。7月，靶向β淀粉样蛋白的单抗Kisunla获批治疗早期阿尔茨海默病成人患者；9月，IL-13抑制剂Ebglyss获准用于治疗12岁以上、体重≥40公斤且局部治疗无效的中重度特应性皮炎患者；年底，重磅GLP-1/GIP受体双重激动剂Zepbound获批改善中重度阻塞性睡眠呼吸暂停肥胖患者的呼吸症状。在合作布局上，礼来通过多项战略合作强化管线：2024年初与Isomorphic Labs合作开发基于AlphaFold技术的小分子疗法；7月斥资32亿美元收购Morphic Holding，获得炎症性肠病口服整合素疗法MORF-057；9月与HAYA Therapeutics达成10亿美元的多年协议，利用其RNA引导基因调控平台开发代谢疾病疗法；10月扩展与KeyBioscience的合作，聚焦胰淀素/降钙素双重激动剂（DACRA）在肥胖及相关疾病中的应用。赛诺菲2024年营收：444.6亿美元2024年，赛诺菲与再生元共同开发的重磅抗炎药Dupixent斩获多项关键适应症：年初获FDA批准用于1-11岁嗜酸性粒细胞性食管炎（EoE）儿童患者；年中获美国FDA和欧洲药品管理局批准作为慢性阻塞性肺病（COPD）患者的附加维持治疗，并在随后获FDA批准作为慢性鼻窦炎伴鼻息肉（CRSwNP）青少年患者的辅助维持治疗。此外，IL-6拮抗剂Kevzara获批治疗活动性多关节型幼年特发性关节炎（pJIA）患者，抗CD38抗体Sarclisa则联合VRd方案成为不适合自体干细胞移植的新确诊多发性骨髓瘤成人患者的一线疗法。战略合作方面，赛诺菲1月以17亿美元收购Inhibrx，获得α-1抗胰蛋白酶缺乏症疗法INBRX-101；同月与Synthekine合作开发IL-10受体激动剂布局炎症性疾病；9月与RadioMedix和Orano Med达成3.2亿欧元独家许可协议，开发基于同位素铅的放射性配体疗法；10月追加3亿欧元股权投资，加速与Orano Med在罕见癌症及下一代RLT领域的合作。诺和诺德2024年营收：约421亿美元2024年，诺和诺德重磅产品Wegovy（semaglutide，司美格鲁肽）注射液斩获心血管保护新适应症，成为获FDA批准用于辅助患有心血管疾病及肥胖或超重的成年人预防重大心血管事件的首款减重药物。在2024年欧洲肥胖大会（ECO）上，该公司公布了Wegovy的长期减重效果数据，其疗效可维持长达4年。诺和诺德突破性皮下注射血友病疗法Alhemo获FDA批准，是针对伴抑制物血友病A或B患者的同类疗法中的首款皮下注射治疗方案。该公司还达成多项超5亿美元的研发合作，聚焦利用表观遗传调控开发肥胖管理潜在新疗法、针对代谢功能障碍相关脂肪性肝炎（MASH）的小分子药物、分子胶降解剂的开发，以及利用肝外脂质纳米颗粒（LNP）递送技术推进针对心脏代谢性疾病和罕见病的创新基因药物（genetic medicine）的开发。GSK2024年营收：约401亿美元GSK的PD-1抑制剂Jemperli在局部晚期错配修复缺陷（dMMR）/高微卫星不稳定性（MSI-H）直肠癌患者中展现出前所未有的疗效，并获得FDA授予的突破性疗法认定。近期，该公司的抗生素gepotidacin获FDA批准上市，是近30年来首个治疗单纯性尿路感染的新型口服抗生素。疫苗方面，GSK的带状疱疹疫苗Shingrix的长期随访数据显示，其对50岁以上成人的保护效力持续超10年。2024年，GSK达成了多项超5亿美元的收购和合作协议，覆盖呼吸道疾病、自身免疫疾病、肿瘤及疫苗领域，包括收购Aiolos Bio公司以获得用于治疗哮喘的胸腺基质淋巴细胞生成素（TSLP）靶向单抗AIO-001，收购用于治疗B细胞驱动的自身免疫疾病的T细胞接合器（TCE）CMG1A46，引进映恩生物（Duality Biologics）的抗癌抗体偶联药物DB-1324，以及深化用于治疗传染病的mRNA疫苗的开发合作。安进2024年营收：约334亿美元2024年6月，美国FDA批准安进双特异性抗体疗法Blincyto（blinatumomab），用于年龄一个月或以上、CD19阳性、费城染色体阴性B细胞前体急性淋巴细胞白血病（B-ALL）患者的巩固阶段治疗，无论患者的可测量残留病灶状态如何。在一项3期临床试验中，接受诱导后巩固治疗的B-ALL患者的5年总生存率超80%。近期，该公司的抗CD19抗体Uplizna获FDA批准扩展适应症，用于治疗免疫球蛋白G4相关疾病（IgG4-RD）成人患者。新闻稿指出，这是治疗这一患者群体的首个获批药物。此外，安进的在研减重疗法MariTide于2024年年末公布了积极的2期研究结果，已进入3期开发阶段。在没有2型糖尿病的肥胖或超重人群中，MariTide在第52周实现了平均体重减轻约20%，且未出现体重减轻平台期。武田2024年营收：约309亿美元2024年4月，美国FDA批准了武田的Entyvio皮下注射制剂，用于中度至重度活动性克罗恩病人患者在接受Entyvio静脉注射制剂诱导治疗后的维持治疗，进一步巩固了其在炎症性肠病领域的布局。FDA还批准了该公司的Eohilia（布地奈德口服混悬液）上市，治疗11岁以上嗜酸性粒细胞性食管炎（EoE）患者。新闻稿指出，这是FDA批准治疗EoE的首款口服疗法。近期，武田的在研多肽疗法rusfertide作为真性红细胞增多症（PV）标准治疗附加方案的3期研究达到主要终点，80%的患者不再需要接受放血治疗。2024年，田达成数起超10亿美元的战略合作和许可协议，囊获多款创新疗法的开发权益，包括Kumquat Biosciences公司的一种新型免疫调节癌症小分子抑制剂、AC Immune公司的阿尔茨海默病疗法ACI-24.060、用于肿瘤学、神经科学和炎症领域多个靶点的新型分子胶降解剂，以及血液疾病创新疗法elritercept。勃林格殷格翰2024年营收：约290亿美元近期，勃林格殷格翰为其用于治疗NSCLC的小分子药物zongertinib（BI 1810631）递交了新药申请（NDA），该疗法获得了优先审评资格，有望于今年获批。新闻稿指出，如果获批，zongertinib将成为针对携带HER2突变、既往接受过全身治疗的不可切除或转移性NSCLC成人患者的首个口服靶向疗法。该公司用于治疗渐进性肺纤维化（PPF）的小分子抑制剂nerandomilast已达3期临床试验终点，即将递交上市申请。2024年6月，勃林格殷格翰公布了其胰高血糖素/GLP-1受体双重激动剂survodutide（BI 456906）的积极2期临床试验结果，并即将开展针对MASH的3期临床试验。在这项2期研究中，多达64.5%的中晚期（F2和F3期）肝纤维化成人患者的肝纤维化得到改善，且MASH未恶化。2024年，勃林格殷格翰达成多起超5亿美元的收购和合作协议，包括针对MASH的RNAi疗法、针对精神分裂症的小分子疗法、用于治疗癌症的细胞周期蛋白抑制剂和创新蛋白酪氨酸磷酸酶N1和N2（PTPN1和PTPN2）小分子抑制剂。吉利德科学2024年营收：约286亿美元2024年8月，美国FDA加速批准了吉利德科学斥资43亿美元收购的小分子疗法Livdelzi与熊去氧胆酸（UDCA）联合用于治疗对UDCA应答不足的原发性胆汁性肝硬化（PBC）成人患者，或作为单药治疗对UDCA不耐受的患者，但不建议患有或发展为失代偿性肝硬化的患者使用Livdelzi。根据新闻稿，Livdelzi是在碱性磷酸酶（ALP）正常化、关键生物标志物和瘙痒控制方面，与安慰剂相比表现出统计学显著改善的首个疗法。2025年2月，该疗法获欧盟批准，用于相同的适应症。此外，该公司的突破性长效HIV疗法lenacapavir已获得FDA授予的优先审评资格，预计在今年6月19日之前完成审评。该疗法的上市申请在欧洲也获得了加速审评。Lenacapavir是一种“first-in-class”长效HIV病毒衣壳抑制剂，可以干扰HIV病毒衣壳蛋白的组装和拆卸，在病毒生命周期的多个阶段发挥作用。在两项3期临床试验中，近100%接受每半年一次lenacapavir注射的受试者没有出现HIV感染。拜耳2024年营收：约260亿美元2024年3月，拜耳达成了一项3.1亿美元的合作，获得了在欧洲商业化BridgeBio Pharma公司用于治疗伴有心肌病的转甲状腺素蛋白介导的淀粉样变性（ATTR-CM）的在研疗法acoramidis的独家许可。该疗法目前已在美国、欧洲和日本获批上市，用于治疗ATTR-CM。该公司超8亿美元收购的双重神经激肽-1，3（NK-1，3）受体拮抗剂elinzanetant达3期临床主要终点，公司已向FDA递交NDA，寻求批准elinzanetant用于治疗与更年期相关的中度至重度血管运动症状（VMS，也称为潮热）。德国默克2024年营收：约191亿美元德国默克过去一年通过与多家新锐公司达成合作和授权交易，扩展研发管线。2024年3月，该公司与C4 Therapeutics（C4T）公司签订一项许可和合作协议，合作开发针对C4T在其内部发现管线中推进的两个关键致癌蛋白靶点的靶向蛋白降解剂。2024年6月，德国默克与Biolojic Design公司达成多靶点药物发现合作。该合作将利用Biolojic Design的AI驱动发现平台来设计用于治疗癌症和免疫疾病的治疗性抗体。梯瓦2024年营收：约165亿美元近期，梯瓦公司公布了其与赛诺菲共同开发的在研TL1A靶向单抗duvakitug在2b期临床试验中获得的积极结果。结果显示，接受duvakitug治疗的两大炎症性肠病——溃疡性结肠炎和克罗恩病的患者在第14周时均获得显著缓解。两家公司预计在今年下半年启动3期临床试验。此外，该公司用于治疗精神分裂症的长效缓释皮下注射疗法TEV-‘749已达3期临床试验主要终点。这款疗法利用名为SteadyTeq的缓释技术，让活性药物olanzapine能够在每月一次给药的治疗方案下通过持续释放维持治疗剂量。在一项近期公布的患者和医疗保健专业人员态度和体验调查研究中，超过92%的患者对TEV-‘749的起始治疗方案、给药方案和药物本身感到满意或非常满意。CSL2024年营收：约152亿美元CSL所开发的基因疗法Hemgenix在2022年11月与2023年2月分别获美国FDA与欧盟批准，用于对中度至重度血友病B患者进行一次性治疗。这款疗法在2023年荣获有“医药界诺贝尔奖”之称盖伦奖的最佳孤儿药/罕见病产品奖。此外，欧盟委员会已于今年2月批准“first-in-class”疗法Andembry上市，用于预防12岁及以上成年和青少年患者的遗传性血管性水肿（HAE）发作。新闻稿指出，它是首款只需每月一针、靶向血浆蛋白因子XIIa的HAE发作预防疗法。美国FDA目前正在针对该疗法进行审评。参考资料：[1] The top 20 pharma companies by 2024 revenue. Retrieved April 21, 2025, from https://www.fiercepharma.com/special-reports/top-20-pharma-companies-2024-revenue免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新