Marstacimab-hncq

Novo Nordisk finally won US approval for its hemophilia drug Alhemo following the product’s rejection by the FDA last year. The once-daily shot may now be used to reduce the frequency of bleeding episodes or prevent them entirely in adults and children aged 12 and up with hemophilia A or B with inhibitors. The company didn’t provide a US price for the drug. Friday’s approval gives the Danish pharma some good news to end the year — though not enough to erase the damage done by its underwhelming CagriSema readout , which emerged earlier on Friday. The shares gained about 5% in early trading Monday, after losing almost a fifth of their value Friday following the CagriSema data release. Alhemo, previously known as concizumab, is an anti-tissue factor pathway inhibitor (TFPI). By blocking TFPI, Alhemo raises levels of thrombin, a protein that helps blood clot in the presence of inhibitors. Inhibitors are antibodies patients can develop as part of their immune system; they bind to and neutralize clotting factors such as factor VIII or factor IX. In its pivotal trial, Alhemo cut the frequency of treated spontaneous and traumatic bleeds by 86% compared with patients who had no preventive treatment. Novo’s new drug is given subcutaneously using a pen device, available in three doses. The company claims this is the first subcutaneous treatment of its kind for this population, with “many” of the other treatments being intravenous. Pfizer’s Hympavzi, approved in the US in October , has the same mechanism and is also delivered subcutaneously using a pen injector, though on a weekly rather than daily schedule. However, Hympavzi is only approved for hemophilia A and B patients without inhibitors. According to Novo, an estimated 30% of severe hemophilia A patients and 5-10% of severe hemophilia B patients will develop inhibitors, so theoretically Alhemo could reach a wider population than Pfizer’s product. But Pfizer has plans to fight back: data on Hympavzi in a cohort of patients with inhibitors should arrive in 2025. These results could form the basis of a label expansion that would put Pfizer’s drug on equal regulatory footing with Alhemo but with an obvious convenience advantage. The big beast here is Roche’s weekly shot Hemlibra, which had sales of around $4.6 billion last year. This is approved for hemophilia A patients with or without inhibitors, but cannot treat the B form of the disease. Novo is aiming to take Hemlibra on not with Alhemo but with another asset, Mim8, which passed its Phase 3 test in May. Here, Novo may have the convenience edge, since Mim8 can be given once a month.

▎Armstrong 2024年12月20日，诺和诺德TFPI抗体获得FDA批准上市，用于治疗12岁以上人群的A型或B型血友病，商品名为Alhemo，这也是全球第2款TFPI抗体。 两个月前的10月11日，辉瑞宣布TFPI抗体Marstacimab获得FDA批准上市，用于治疗A型和B型血友病，商品名为Hympavz。诺和诺德Concizumab一度是进展最快的TFPI抗体，但由于血栓事件导致III期临床一度被暂停，被辉瑞Marstacimab反超。 TFPI通过抑制FVIIa-TF-FXa复合物削弱外源性凝血途径，TFPI抗体则阻断TFPI的活性从而可被用于治疗血友病。 总结 国内方面，康宁杰瑞KN057为首款TFPI抗体，目前处于三期临床阶段，安源医药AP017则为第二个国产TFPI抗体，目前处于一期临床阶段。血友病的治疗以凝血因子替代治疗、FIX/FX双抗等为主，TFPI抗体的成功为血友病患者提供了全新治疗选择。 Armstrong技术全梳理系列 GPRC5D靶点全梳理； CD40靶点全梳理； CD47靶点全梳理； 补体靶向药物技术全梳理； 补体药物：眼科治疗的重要方向； Claudin 6靶点全梳理； Claudin 18.2靶点全梳理； 靶点冷暖，行业自知； 中国大分子新药研发格局； 被炮轰的“me too”； 佐剂百年史； 胰岛素百年传奇； CUSBEA：风雨四十载； 中国新药研发的焦虑； 中国生物医药企业的研发竞争； 中国双抗竞争格局； 中国ADC竞争格局； 中国双抗技术全梳理； 中国ADC技术全梳理； Ambrx技术全梳理； Vir Biotech技术全梳理； Immune-Onc技术全梳理； 亘喜生物技术全梳理； 康哲药业技术全梳理； 科济药业技术全梳理； 恺佧生物技术全梳理； 同宜医药技术全梳理； 百奥赛图技术全梳理； 腾盛博药技术全梳理； 创胜集团技术全梳理； 永泰生物技术全梳理； 中国抗体技术全梳理； 德琪医药技术全梳理； 德琪医药技术全梳理2.0； 和铂医药技术全梳理； 荣昌生物技术全梳理； 再鼎医药技术全梳理； 药明生物技术全梳理； 恒瑞医药技术全梳理； 豪森药业技术全梳理； 正大天晴技术全梳理； 吉凯基因技术全梳理； 基石药业技术全梳理； 百济神州技术全梳理； 百济神州技术全梳理第2版； 信达生物技术全梳理； 信达生物技术全梳理第2版； 中山康方技术全梳理； 复宏汉霖技术全梳理； 先声药业技术全梳理； 君实生物技术全梳理； 嘉和生物技术全梳理； 志道生物技术全梳理； 道尔生物技术全梳理； 尚健生物技术全梳理； 康宁杰瑞技术全梳理； 科望医药技术全梳理； 科望医药技术全梳理2.0； 岸迈生物技术全梳理； 礼进生物技术全梳理； 康桥资本技术全梳理； 余国良的抗体药布局； 荃信生物技术全梳理； 安源医药技术全梳理； 三生国健技术全梳理； 仁会生物技术全梳理； 乐普生物技术全梳理； 同润生物技术全梳理； 宜明昂科技术全梳理； 派格生物技术全梳理； 迈威生物技术全梳理； Momenta技术全梳理； NGM技术全梳理； 普米斯生物技术全梳理； 普米斯生物技术全梳理2.0； 三叶草生物技术全梳理； 贝达药业抗体药全梳理； 泽璟制药抗体药全梳理； 恒瑞医药抗体药全梳理； 齐鲁制药抗体药全梳理； 石药集团抗体药全梳理； 豪森药业抗体药全梳理； 华海药业抗体药全梳理； 科伦药业抗体药全梳理； 百奥泰技术全梳理； 凡恩世技术全梳理。