Glomerulonephritis, Membranous

Felzartamab is being developed to treat antibody-mediated rejection in kidney transplant recipients. Credit: Gorodenkoff / Shutterstock.com. The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Human Immunology Biosciences’ (HI-Bio) investigational therapeutic felzartamab, to treat antibody-mediated rejection (AMR) in kidney transplant recipients. The designation is a significant step in the development of felzartamab, a monoclonal antibody targeting cluster of differentiation 38 (CD38), which is involved in the production of pathogenic antibodies. It selectively depletes CD38+ plasma cells. Clinical trials demonstrated its potential to improve outcomes in diseases caused by these pathogenic antibodies. HI-Bio is developing felzartamab for immune-mediated diseases including AMR, IgA nephropathy, lupus nephritis and primary membranous nephropathy (PMN). The FDA has previously awarded felzartamab both breakthrough therapy and orphan drug statuses for PMN treatment. See Also: Italfarmaco secures FDA approval for Duchenne muscular dystrophy drug Lisata clinches rare disease status for osteosarcoma drug HI-Bio acquired exclusive global rights to felzartamab, excluding Greater China, through a licensing agreement with MorphoSys in 2022. The FDA’s ODD provides HI-Bio with development incentives such as tax credits for qualified clinical studies, a waiver of FDA application fees and the potential for seven-year market exclusivity upon regulatory approval. AMR is a leading cause of kidney transplant failure with no current effective treatment. The condition is associated with donor-specific antibody production by plasma cells and the infiltration of natural killer cells, which contribute to microvascular inflammation. HI-Bio chief medical officer Uptal Patel said: “Following the FDA’s granting of breakthrough therapy designation for felzartamab in primary membranous nephropathy, we are encouraged to receive orphan drug designation for felzartamab for antibody-mediated rejection. “We are confident in the clinical progress of our anti-CD38 cellular depletion strategy, which to date, has resulted in proof-of-concept data in multiple severe immune-mediated diseases, including antibody-mediated rejection, IgA nephropathy and primary membranous nephropathy.”

SOUTH SAN FRANCISCO, Calif., March 21, 2024 /PRNewswire/ -- Human Immunology Biosciences (HI-Bio™), a clinical-stage biotechnology company developing targeted therapies for patients with severe immune-mediated diseases (IMDs), today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for felzartamab for the treatment of antibody-mediated rejection (AMR) in kidney transplant recipients. "Following the FDA's granting of Breakthrough Therapy Designation for felzartamab in primary membranous nephropathy, we are encouraged to receive Orphan Drug Designation for felzartamab for antibody-mediated rejection," said Uptal Patel, M.D., Chief Medical Officer at HI-Bio. "Along with our academic collaborators, we look forward to submitting clinical data from the ongoing study of felzartamab in antibody-mediated rejection to a medical conference this year. We are confident in the clinical progress of our anti-CD38 cellular depletion strategy, which to date, has resulted in proof-of-concept data in multiple severe immune-mediated diseases including antibody-mediated rejection, IgA nephropathy and primary membranous nephropathy." The FDA's Orphan Drug Designation program is designed to advance the development of drugs and biologics intended to treat a rare disease or condition that affects fewer than 200,000 people in the United States. Orphan Drug Designation qualifies HI-Bio for certain development incentives, including tax credits for qualified clinical trials, exemption of FDA application fees and up to seven-year market exclusivity upon regulatory approval. About Antibody-Mediated Rejection (AMR) in Kidney Transplant Recipients Antibody-mediated rejection (AMR) is a major cause of kidney transplant failure, with late AMR affecting approximately 23,000 patients total in the U.S. There is no effective treatment for AMR and patient options are highly limited. Donor-specific antibody (DSA) production by plasma cells, and tissue infiltration of Natural Killer (NK) cells presumed to be involved in DSA-dependent microvascular inflammation, are both linked to AMR. Observations that both plasma cells and NK cells express high levels of CD38 have motivated the approach of targeting CD38 to deplete these cell populations to address AMR. About Felzartamab Felzartamab is an investigational therapeutic human monoclonal antibody directed against CD38, a protein expressed on mature plasma cells. Felzartamab has been shown in clinical studies to selectively deplete CD38+ plasma cells, which may allow applications that ultimately improve clinical outcomes in a broad range of diseases driven by pathogenic antibodies. HI-Bio is focused on developing felzartamab in a number of immune-mediated diseases, including antibody-mediated rejection (AMR), IgA nephropathy (IgAN), lupus nephritis (LN) and primary membranous nephropathy (PMN). Felzartamab has received Breakthrough Therapy designation and Orphan Drug Designation from the FDA for development in the treatment of PMN, and Orphan Drug Designation in the treatment of AMR in kidney transplant recipients. HI-Bio in-licensed felzartamab from MorphoSys in 2022 and holds exclusive worldwide rights for felzartamab with the exception of Greater China. Felzartamab is an investigational therapeutic candidate that has not yet been approved by any regulatory authority. About HI-Bio Human Immunology Biosciences, Inc. (HI-Bio™) is a clinical-stage biotechnology company focused on discovering and developing precision medicines for people suffering from immune-mediated diseases (IMDs). HI-Bio is leading clinical immunology into its next chapter by targeting cellular drivers of disease. To learn more about HI-Bio, visit us at or follow us on LinkedIn and X.

3月16日，苏州邦器生物技术有限公司（以下简称“邦器生物”）在第二十一届中国国际检验医学暨输血仪器试剂博览会（CACLP）隆重发布全自动流式荧光发光免疫分析仪BQ-TF6000。通过自研光路和液路系统、微球系统突破，BQ-TF6000实现了流式荧光检测平台的技术革新，以全自动、超高速、高通量、多联检，开启自免＆过敏领域的多联检新时代。CACLP邦器生物新品发布会现场免疫反应是机体识别“自我”与“非我”、产生免疫应答，清除异己抗原或者诱导免疫耐受，从而维持自身内环境稳定的过程。但当免疫系统过度应答或者错误识别无害蛋白（自身抗原），则会引起过敏反应或自身免疫疾病。自身免疫性疾病可以发生于某一器官，也可以发生于全身各处，症状因抗原种类的不同而不同。系统性红斑狼疮、结节性多动脉炎、硬皮病、皮肌炎、混合性结缔组织病，自身免疫性肝病、自身免疫性脑炎、特发性膜性肾病等疾病，都曾被视为“不明原因”的“疑难杂症”“罕见病”。随着诊断技术的发展，该类疾病才被确定为“自身免疫性疾病”。随着广泛临床需求崛起、检验设备更新需求来临、基层医疗机构降本增效需求持续扩大，国内自免诊断市场正在经历快速崛起的窗口期。在响应市场需求、实现产品更高的可及性上，国产化自免诊断厂商已经崛起，逐步搭建起国产化的产业价值链。国内自免、过敏检测市场崛起，基层市场空间广阔目前，自免疾病诊断正在经历一段快速发展的时期，国产化厂商入局、多种方法学平台迭代正在重塑自免、过敏诊断市场。一方面，国产替代正进入快速增长期。为了实现关键领域的技术自主可控，政府加大了对国产体外诊断平台研发的投入和支持，连续发布多条高端科学仪器国产替代政策，进一步限制进口设备的采购，为国产替代提供了良好的政策环境。《中华人民共和国政府采购法》也有明确规定，国产药品和医疗器械能够满足要求的，政府采购项目原则上须采购国产产品，逐步提高公立医疗机构国产设备配置水平。另一方面，自免疾病诊断的高成本、长时间倒逼国产厂商迭代。基层发育不足、临床需求发展成为新的增长点。自免疾病诊断的临床特点之一是识别和诊断自免疾病的过程十分耗时。没有单一检测指标可以有效和准确地诊断自身免疫性疾病，因而需要多种检测方法和多项指标结果共同验证。更实际问题在于，自身免疫程序的较低渗透性使得此类测试周转时间更长，医疗成本不断上升。深层次而言，这间接导致了国内自免诊断市场的显著难题——长期以来，大众认识不足和医疗机构关注不足是自免疾病受到忽视的重要原因，尤其是广泛的基层医疗机构。由于自免患者症状往往不危重，可能导致医疗工作者不重视，患者也拥有充分的治疗窗口时间前往上级医院就诊。更重要的是，多数二级及以下医院尚不具备诊断自免疾病的能力——进口诊断设备昂贵、早期诊断设备自动化程度低、开机成本高、专业人员储备不足等均为限制因素。低诊断水平也导致了自免疾病的低确诊率，自免患者往往被迫分流至肾内科、肝病科、血液内科、内分泌科等进行诊疗，致使中国还存在着庞大的潜在患者群体。2019年，卫健委发文要求和鼓励医院建设风湿免疫科，全国的三级医院必须建风湿科室且配备10张床位，同时医院应具有独立的检验科支持风湿免疫疾病的常规检查。类风湿关节炎、痛风性关节炎、过敏性哮喘等常见疾病均被包含至风湿免疫科。如果把“自免疾病”替换为“风湿病”，国内市场空间的广阔更加显而易见。自免疾病患病率增加、人们对于自免疾病的认识增长、生活质量追求提升，继续寻求新业务增长点的基层医疗机构开始看到自免疾病的临床机会。自动化、高性能的国产替代渐成主旋律就现实情况而言，医保控费、医改、集采等大环境下，二级及以下医疗机构对性价比的要求更为突出。与此同时，三级医院的仪器迭代需求也已提上日程，进一步打开了国产替代市场。一大趋势是，仪器和检验过程的全自动化，包括解放人力；确保检测安全性；压缩报告产出时间，提高效率；标准化、保证重复性和结果可靠性。以免疫印迹法为例，早期的诊断试剂需要按照说明书手工操作，效率低下、判读准确性受多种干扰因素影响。后迭代为需要人工辅助、人工监控的半自动化检测平台，精度和判读周期均有待提升。业内人士表示，仍有70%以上在院临床检验设备为手工方法和半自动化检测。邦器生物即为典型的坚持设备+试剂科技创新的国产厂商。2022年，邦器生物推出首款自研产品全自动免疫印迹仪BQ-BM100。从原始管上机到结果判读的全自动、智能化设计下，检测全程无需人员值守、不同项目同时上机，可同时获得5000+结果。通过双模块运行、多项目并行，单次可检测100个样本，将过去3—7天的结果时间缩短到最短1.5小时。在免疫印迹方法上做到高精度、高通量、高速率后，邦器生物又拓展新的方法学检测平台——流式荧光发光免疫分析技术。此次发布的全自动流式荧光发光免疫分析仪BQ-TF6000更往前一步，可支持自动稀释、自动补杯、自动清洗、自动完成反应杯转运等，全程无需人员干预。通过LIS双向通讯，可实时监控试验状态。此外，BQ-TF6000实现了全定量的检测，极大提高了准确性与稳定性。 继续向下追溯，第二大趋势是，检测结果的高精度、高速率。前文提到，没有单一的检验指标可以有效和准确地诊断自免疾病。就自免疾病临床诊断而言，高效的多指标数据可为医生提供诊断依据，极大缩短诊断时长，因此多指标联检也是增效的关键竞争力之一。流式荧光技术（又称多重液相芯片）能在一个反应体系中完成几十种指标的联合检测，仅需一份试剂和耗材即可完成多指标联检，以及多指标任意组合，最多可一管同时准确定量检测200-500种不同的生物分子。同时具有高通量、高灵敏度、并行检测、自动化高、线性范围宽、重复性好、综合成本低等特点。性能上，BQ-TF6000检测通量为恒速120样本/小时，联机最高通量可达9120T/h高通量；可同时加载60+指标的定量检测；CV≤3%；模块化设计、支持多台联机，可联流水线。BQ-TF6000全自动流式荧光检测平台布局多方法学检测平台，以工匠精神打造“国之重器”目前，邦器生物拥有近4000平GMP车间和研发实验室，搭建了海外行业精英领衔的设备+试剂自主研发团队。基于免疫印迹BQ-BM100和流式荧光BQ-TF6000检测平台，邦器生物将逐步推出全套自身免疫及过敏原检测解决方案。紧贴临床需求，邦器生物将构建对于风湿病自身抗体、抗核抗体谱、自身免疫性肝病免疫球蛋白G抗体等多病种、多联检的临床解决方案。新品发布会后，邦器生物创始人陶瑞接受了动脉网独家专访。陶瑞表示“邦器一词源于《周礼》‘衅邦器，及军器’，科技创新、工匠精神始终是邦器生物的源动力，邦器生物一直以合规、安全、高效、稳定的理念，打磨每一款产品，为医学检验提供更加快速、稳定、精确的数据，为临床提供明确的诊断依据，不断发展，科技兴邦，器械助医。”从成立之初，邦器生物就确定了立足主流检测方法学、布局多种方法学检测平台、逐步拓展至其他多联检检测领域的商业战略。陶瑞表示，面向未来，邦器生物一方面将逐步布局自免&过敏领域的多种方法学检测平台，为各级医院提供多元化、高性能的自免＆过敏检测平台矩阵，惠及更多患者。另一方面，邦器生物将逐步拓展完善流式荧光检测平台，推出系列试剂解决方案，并探索流式荧光的更多适应领域，不断拉高企业发展天花板，为邦器生物成为行业龙头企业打下坚实的基础。写在最后自身免疫疾病已经成为除心血管疾病和癌症外的第三大慢性疾病，据《柳叶刀》数据，常见的自身免疫疾病患病人数均逐年上升。当生命科学被写入《政府工作报告》、纳入具有生产力跃迁意义的“新质生产力”范畴，自主创新、自主研发正日益成为高壁垒、高精尖技术类医疗企业成功的关键。更广泛意义上，随着邦器生物此类自主研发的行业领军企业崛起，越来越多过去认为是“疑难杂症”的疾病被攻克，逐步走入基层医疗诊断与治疗，也正指向卫生健康事业高质量发展，护佑人民健康，满足美好生活需要的医疗终点。*封面图片来源：123rf文|李秋萩微信|lqq15008979948添加时请注明：姓名-公司-职位网站、公众号等转载请联系授权：Rekkiiie近期推荐声明：动脉网所刊载内容之知识产权为动脉网及相关权利人专属所有或持有。未经许可，禁止进行转载、摘编、复制及建立镜像等任何使用。动脉网，未来医疗服务平台