Open-label Safety Study in Adults and Adolescents With Haemophilia A With and Without FVIII Inhibitors Switching Directly From Emicizumab Prophylaxis to NNC0365-3769 (Mim8) Prophylaxis

This study is looking at how safe it is to switch from emicizumab to Mim8, in people with haemophilia A. Mim8 is a new medicine that is used to prevent bleeding episodes in people with haemophilia A. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). Mim8 will be injected under the skin using a pen-injector either once every week, once every two weeks or once every month. The participants will be trained in using the pen injector. The participants can choose themselves, in collaboration with the study doctor how often they get Mim8 in this study. When the participant will get their first Mim8 injection depends on their current treatment with emicizumab. The participants will get their first Mim8 injection at Visit 2. Participants will have between 6 and 27 Mim8 injections. The total number of injections participants will have depends on their dosing frequency. The study will last for about 6-12 months. While taking part in this study, there are some restrictions about what medicine participant can use. The study doctor will tell the participants more about this. In case the participants experience bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor. Female participants cannot take part if they are pregnant, breast-feeding or plan to get pregnant during the study period.

开始日期2023-06-26

申办/合作机构

Novo Nordisk A/S

NCT05685238

/ Active, not recruiting临床3期

Open-label, Long-term Safety and Efficacy Study of Mim8 in Participants With Haemophilia A With or Without Inhibitors

This study is looking at how Mim8 works in people with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medicine that will be used to avoid bleeding episodes. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). The study will last for up to 5.5 years. The duration of the study depends on when the participant enrolled in this study. The study will end if Mim8 is approved and marketed in participant's country during the study, or the study will end in June 2028, whichever comes first. Participants will get up to 262 injections; the number of injections depends on how often participants will get injections and how long time participants take part in the study. While taking part in this study, there are some restrictions about what medicine participants can use. The study doctor will tell the participants more about this. In case the participants experience bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor. Female participants cannot take part if they are pregnant, breast-feeding or plan to get pregnant during the study period.

开始日期2023-02-13

申办/合作机构

Novo Nordisk A/S

NCT05681845

/ Completed临床1期

A Study to Demonstrate Bioequivalence of a Single Subcutaneous Administration of NNC0365-3769 (Mim8) With the DV3407-C1 Pen Injector Versus a Syringe and Enhanced Cartridge in Healthy Male Participants

Novo Nordisk is developing the study medicine Mim8 for the treatment of haemophilia A. The study aims to show similar levels of Mim8 in blood when using a new pen injector, called DV3407-C1 pen injector, and when using a syringe and cartridge. The new pen injector is intended to facilitate the administration of Mim8 for patients with haemophilia A. The participants will get Mim8 as injection under the skin (subcutaneously) of the belly using the DV3407-C1 pen injector and a needle (hereinafter referred to as pen injector) or using a needle and syringe from a cartridge (hereafter called syringe and cartridge). The participants will receive one injection with Mim8, either with the DV3407-C1 pen injector or with a syringe and cartridge. The study participation will last up to 20 weeks. Only healthy men can take part in the study.

开始日期2023-01-02

申办/合作机构

Novo Nordisk A/S

100 项与 Denecimig 相关的临床结果

登录后查看更多信息

100 项与 Denecimig 相关的转化医学

登录后查看更多信息

100 项与 Denecimig 相关的专利（医药）

登录后查看更多信息

项与 Denecimig 相关的文献（医药）

2025-12-31·mAbs

Antibodies to watch in 2025

Review

作者: Kapoor, Vaishali ; Kaplon, Hélène ; Crescioli, Silvia ; Visweswaraiah, Jyothsna ; Reichert, Janice M. ; Wang, Lin

The commercial development of antibody therapeutics is a global enterprise involving thousands of biopharmaceutical firms and supporting service organizations. To date, their combined efforts have resulted in over 200 marketed antibody therapeutics and a pipeline of nearly 1,400 investigational product candidates that are undergoing evaluation in clinical studies as treatments for a wide variety of diseases. Here, we discuss key events in antibody therapeutics development that occurred during 2024 and forecast key events related to the late-stage clinical pipeline that may occur in 2025. In particular, we report on 21 antibody therapeutics granted a first approval in at least one country or region during 2024, including bispecific antibodies tarlatamab (IMDELLTRA®), zanidatamab (Ziihera®), zenocutuzumab (BIZENGRI®), odronextamab (Ordspono®), ivonescimab (®), and antibody-drug conjugate (ADC) sacituzumab tirumotecan (®). We also discuss 30 investigational antibody therapeutics for which marketing applications were undergoing review by at least one regulatory agency, as of our last update on December 9, 2024, including ADCs datopotamab deruxtecan, telisotuzumab vedotin, patritumab deruxtecan, trastuzumab botidotin, becotatug vedotin, and trastuzumab rezetecan. Of 178 antibody therapeutics we include in the late-stage pipeline, we summarize key data for 18 for which marketing applications may be submitted by the end of 2025, such as bi- or multispecific antibodies denecimig, sonelokimab, erfonrilimab, and anbenitamab. Key trends in the development and approval of antibody formats such as bispecifics and ADCs, as well as clinical-phase transition and global approval success rates for these antibody formats, are reported.

2025-09-01·EUROPEAN JOURNAL OF PHARMACEUTICAL SCIENCES

Model-informed drug development of Mim8 – a next-generation bispecific antibody for treatment of haemophilia A

Article

作者: Kreilgaard, Mads ; Persson, Paula ; Matytsina, Irina

BACKGROUND:

Haemophilia A is a congenital bleeding disorder caused by deficiency of clotting factor VIII (FVIII). Mim8 (denecimig) is a next-generation activated FVIII mimetic bispecific antibody under phase 3 investigation for subcutaneous management of haemophilia A with or without FVIII inhibitors. Mim8 uses tiered dosing, which is a novel dosing approach in haemophilia where patients receive a fixed dose according to which body weight range they fall into and their chosen dosing frequency, reducing the need for dose calculations and potentially reducing treatment wastage.

OBJECTIVES:

This study aimed to establish the early population pharmacokinetics (PK) and PK/pharmacodynamic (PD) properties of Mim8 using both biomarker and clinical endpoint data. Models were used to inform drug-development decisions using trial simulations and transform the dosing strategy in haemophilia to a novel simplified treatment option using tiered dosing in paediatric and adult populations.

PATIENTS/METHODS:

Data from 129 participants exposed to Mim8 in the phase 1 PK trial (NCT0512747) and the FRONTIER1 trial (NCT04204408) were used to develop population PK and PK/PD models for thrombin generation and treated bleeds. The models were used for trial simulations to optimise dosing regimens evaluating Mim8 exposure, biomarker and clinical endpoint targets on a population level (paediatric and adolescent/adult) and on a typical subject level for the range of body weight (BW) in the trial. Clinical endpoint targets were set to: (1) at least 70 % of patients without treated bleeds in a 6-month trial on a population level and >95 % reduction in relative bleed risk on a typical subject level across all weight ranges and dosing frequencies, and (2) sustained peak Mim8 exposure below highest exposure observed in phase 1/2 trials.

RESULTS:

The population PK of Mim8 were best described with a structural two-compartment model with baseline BW as the most significant covariate for Mim8 exposure. Both peak thrombin response and Mim8 effect on bleed risk were best described with a direct link PK/PD model with an E_max response. Model-estimated EC₅₀ was 2.03 µg/mL for peak thrombin and 0.07 µg/mL for bleed risk. Using trial simulations, tiered dosing in three weight ranges (<15, 15-<45, and ≥45 kg) was identified to reach the clinical endpoint targets and maximum desired exposure for all three dosing frequencies: once every week, once every 2 weeks, and once every month. This was done with an initial one-time loading dose establishing steady-state conditions within the first day, followed by maintenance doses at the desired dosing frequency. Loading and maintenance doses for the nine combinations of weight ranges and dosing frequencies were covered by five dose presentations. Ongoing phase 3 trials have been based on the dosing strategy presented here.

CONCLUSIONS:

Model-informed drug development was used to design a novel treatment paradigm for patients living with haemophilia A by utilising a tiered-dosing strategy with Mim8, to be evaluated in phase 3 trials.

2025-07-01·THROMBOSIS RESEARCH

In vitro effects of Mim8 and combined Mim8-bypassing therapy on thrombin generation, thromboelastography and fibrin clot ultrastructure

Article

作者: Dargaud, G Yesim ; Nougier, Christophe ; Josset, Laurie ; Lund, Jacob ; Rezigue, Hamdi

INTRODUCTION:

Mim8, a fully human bispecific IgG4 antibody, FVIIIa mimetic, which emerges as a promising prophylactic treatment option for patients with hemophilia A (HA).

AIM:

This study investigates the in vitro hemostatic activity of Mim8 in blood samples from six patients with severe hemophilia A using a thrombin generation assay (TGA) and thromboelastography. The results were compared to those obtained with emicizumab.

MATERIALS AND METHODS:

TG was assessed in both platelet-poor (PPP) and platelet-rich plasma (PRP). Fibrin clots formed during TG were collected, and clot ultrastructure was examined using scanning electron microscopy (SEM). Whole blood samples were analyzed using ROTEM-NATEM.

RESULTS:

Mim8 significantly improved the TG capacity of severe HA plasma samples. Mim8 6 μg/mL demonstrated higher TG capacity compared to emicizumab 50 μg/mL. In PRP samples fortified with Mim8 6 μg/mL and rFVIIa 90 μg/kg together, ETP levels were fully normalized. No synergistic effect was observed when FVIII concentrate was combined with Mim8 at therapeutic doses due to the higher affinity of FVIII for FIXa and FX compared to Mim8. ROTEM-NATEM in whole blood confirmed the TGA results. SEM showed a more robust fibrin clot structure with thinner fibrin fibers in the presence of Mim8 compared to emicizumab.

CONCLUSIONS:

Mim8 significantly improves TG in vitro in both PPP and PRP from patients with severe HA, with ETP levels comparable to those of FVIII at 100 IU/dL. The TGA can effectively monitor the combined treatment with Mim8 and either rFVIIa or APCC, which is not possible with currently available routine laboratory tests.

项与 Denecimig 相关的新闻（医药）

2025-09-29

·PRNewswire

Novo Nordisk submits Biologics License Application (BLA) to FDA for Mim8; an investigational, prophylaxis treatment for people living with hemophilia A with or without inhibitors

If approved, Mim8 (denecimig) would offer the flexibility of treatment with a single-use, prefilled disposable pen injector with dosing options including once every month, once every two weeks, and once every week for people living with hemophilia A In hemophilia A, denecimig is designed to help the body form blood clots more effectively and was studied as part of the FRONTIER program across different dosing frequencies, age groups, severities, and with or without inhibitors1 Novo Nordisk is advancing its long-standing commitment to people living with hemophilia A, a potentially life-threatening condition BAGSVÆRD, Denmark and PLAINSBORO, N.J., Sept. 29, 2025 /PRNewswire/ -- Today, Novo Nordisk announced Mim8 (denecimig) has been submitted for review to the US Food and Drug Administration (FDA) through a Biologics License Application (BLA), a formal request to evaluate a biologic medicine. Denecimig is an investigational, next-generation bispecific antibody Factor VIIIa (FVIIIa) mimetic, designed as a preventive bleed routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A (congenital FVIIIa deficiency), with or without inhibitors.2 If approved, denecimig will be the first FVIIIa mimetic with flexible once every month, once every two weeks, and once every week dosing in a pre-filled, single-use pen, catering to the diverse needs of people living with hemophilia A, with or without inhibitors.1 "It is important that we continue to drive scientific advances in hemophilia A, and the FRONTIER program supports the potential of denecimig as a prophylaxis bleed treatment option for a range of patients across dosing frequencies, age groups, and condition severity, regardless of inhibitor status," said Anna Windle, Senior Vice President, Clinical Development, Medical & Regulatory Affairs at Novo Nordisk. "A cornerstone of hemophilia treatment is to help prevent bleeding episodes, and this submission is another step forward in our long-standing mission to help improve care. If approved, denecimig's single-dose prefilled injector pen and flexible dosing schedule may better fit into the lives of people living with this challenging condition." The FDA submission is grounded in the results from the FRONTIER study program, a comprehensive program of studies designed to establish the efficacy and safety profile of denecimig as a prophylaxis treatment to prevent or reduce the frequency of bleeding episodes in people with hemophilia A, with or without inhibitors.1,3-6 About Mim8 (denecimig) Mim8 (denecimig) is an investigational FVIIIa mimetic bispecific antibody designed with the aim to deliver once every month, once every two weeks, or once every week prophylaxis for people living with hemophilia A, with or without inhibitors.3-6 Denecimig, which is administered under the skin, "mimics" the role of FVIIIa by bridging Factor IXa and Factor X.7 This action replaces FVIIIa function, which helps restore the body's thrombin generation capacity, helping blood to clot.8 The use of denecimig in people living with hemophilia A is investigational and not approved by any regulatory authorities worldwide. About Hemophilia A Hemophilia is a rare inherited bleeding disorder that impairs the body's ability to make blood clots, a process needed to stop bleeding.9 According to the World Federation of Hemophilia, it is estimated to affect approximately 836,000 people worldwide and hemophilia A is estimated to account for 80-85% of all hemophilia cases.10 There are different types of hemophilia, which are characterized by the type of clotting factor protein that is defective or missing.6 Hemophilia A is caused by a missing or defective clotting Factor VIII (FVIII).9 Some people with hemophilia A may develop inhibitors, an immune system response to the clotting factors used in replacement therapy, which can cause treatment to become ineffective.11 It has been estimated that approximately 30% of people living with hemophilia A have inhibitors.11 About the FRONTIER Clinical Program The FRONTIER clinical program includes FRONTIER1-5 and investigates denecimig as a preventive bleed treatment across pediatric and adult populations with hemophilia A, with or without inhibitors. 1,3-5,12,13 FRONTIER2, FRONTIER3 and FRONTIER4 formed the basis of the denecimig BLA submission. FRONTIER2 evaluated denecimig treatment once every month and once every week in adults and adolescents 12 years of age and older; FRONTIER3 evaluated denecimig treatment once every month and once every week in children below the age of 12; and FRONTIER4 was an open label extension trial evaluating the efficacy of denecimig once every two weeks (Q2W) as well as investigating the long-term safety of denecimig across all dosing regimens (once every month, once every two weeks, and once every week) in subjects with hemophilia A, with or without inhibitors.3-5 Novo Nordisk is a leading global healthcare company founded in 1923 and headquartered in Denmark. Our purpose is to drive change to defeat serious chronic diseases built upon our heritage in diabetes. We do so by pioneering scientific breakthroughs, expanding access to our medicines, and working to prevent and ultimately cure disease. Novo Nordisk employs about 78,400 people in 80 countries and markets its products in around 170 countries. For more information, visit novonordisk.com, Facebook, Instagram, X, LinkedIn, and YouTube. Contacts for further information References Novo Nordisk Data on File. Bowyer AE, Hickey K, Kitchen S, Ezban M. A next generation FVIII mimetic bispecific antibody, Mim8, the impact on non-factor VIII related haemostasis assays. Haemophilia. 2023;29(6):1633-1637. ClinicalTrials.gov. A Research Study Investigating Mim8 in Adults and Adolescents With Haemophilia A With or Without Inhibitors. Last accessed September 2025. Available at . ClinicalTrials.gov. A Research Study Looking at Mim8 in Children With Haemophilia A With or Without Inhibitors. Last accessed September 2025. Available at . ClinicalTrials.gov. A Research Study Looking at Long-term Treatment With Mim8 in People With Haemophilia A (FRONTIER 4). Last accessed September 2025. Available at . Østergaard H, Lund J, Greisen PJ, et al. A factor VIIIa-mimetic bispecific antibody, Mim8, ameliorates bleeding upon severe vascular challenge in hemophilia A mice. Blood. 2021;138(14):1258-1268. Lentz S, Chowedary P, Gil L, et al. FRONTIER1: a partially randomized phase 2 study assessing the safety, pharmacokinetics, and pharmacodynamics of Mim8, a factor VIIIa mimetic. J Thromb Haemost. 2024;22(4): 990-1000. U.S. National Library of Medicine. F8 gene. MedlinePlus Genetics. Last accessed August 2025. Available at . MedlinePlus. Hemophilia. Last accessed September 2025. Available at . World Federation of Hemophilia. World Federation of Hemophilia Report on the Annual Global Survey 2023. Last accessed September 2025. Available at . Kim JY, You CW. The prevalence and risk factors of inhibitor development of FVIII in previously treated patients with hemophilia A. Blood Res. 2019;54:204-209. ClinicalTrials.gov. A Research Study Investigating Mim8 in People With Haemophilia A (FRONTIER1). Last accessed September 2025. Available at . ClinicalTrials.gov. A Research Study Looking at How Safe it is to Switch From Emicizumab to Mim8 in People With Haemophilia A (FRONTIER 5). Last accessed September 2025. Available at . Novo Nordisk is a registered trademark of Novo Nordisk A/S. All other trademarks, registered or unregistered, are the property of their respective owners. © 2025 Novo Nordisk All rights reserved. US25HRBD00364 September 2025 Logo - WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床2期临床结果

2025-09-10

·PRNewswire

Precision BioLogic Leads the Way in Hemophilia Testing

Proven CRYO check ™ Chromogenic Factor VIII assay now offers expanded utility in anticipation of emerging treatments HALIFAX, NS, Sept. 10, 2025 /PRNewswire/ - Precision BioLogic Inc., a leading developer of hemostasis diagnostic products, is pleased to announce the expanded utility of its CRYOcheck Chromogenic Factor VIII assay in anticipation of emerging hemophilia treatments such as Mim8, a next generation bispecific antibody for the treatment of hemophilia A. Precision BioLogic originally developed CRYOcheck Chromogenic Factor VIII in response to a growing demand for chromogenic assays1. Launched in 2020, it was the first commercially available, FDA-cleared chromogenic factor VIII (FVIII) assay designed for use on automated instruments and was readily welcomed by clinical labs to help identify FVIII deficiency and aid in the management of hemophilia A. And now Precision BioLogic is again leading the way in bringing novel, cleared solutions to clinical labs conducting hemophilia testing. "We took a great assay and made it even better," explains Paul Empey, President & CEO of Precision BioLogic. "We want to ensure labs are ready and able to deliver accurate results when new treatments become available. And we want to make doing that as easy as possible for labs." As new hemophilia drugs become available, it is crucial that labs can accurately report the level of those drugs in human plasma as part of managing treatment. While next generation bispecific antibody treatments such as Mim8 show great promise in enhancing quality of life for people with hemophilia A, studies2 show that many existing assays are sensitive to Mim8 and therefore not suitable for monitoring native FVIII or conventional FVIII replacement activities in plasma in the presence of Mim8. The updated CRYOcheck Chromogenic Factor VIII meets the current needs of hemophilia testing labs while anticipating future requirements. In data submitted to the FDA as part of its recent 510(k) submission, Precision BioLogic proved that therapeutic levels of Emicizumab, a first-generation bispecific antibody treatment, and expected therapeutic levels of Mim8 do not interfere with the assay's performance. Furthermore, Precision BioLogic demonstrated the assay's ability to accurately evaluate the potency of leading FVIII replacement products. To ensure continuity of testing for labs, the assay continues to be supported on all current automated coagulation analyzers, with a limit of quantification of 0.5% FVIII activity and a test range of 0–200% FVIII using one standard curve (depending on the instrument in use). It is formatted to meet the needs of any size laboratory, increasing efficiency, reducing wastage, and ensuring accuracy of results. Assay components are frozen, allowing for fast and easy preparation. The test is cleared for sale in the United States, Canada, European Union, United Kingdom, Australia, and New Zealand, and will be commercially available later this year. Precision BioLogic has resources in place to help labs transition to the new kit smoothly and easily. Labs will be able to switch to the new kit immediately or may choose to transition later. Precision BioLogic's Hemophilia Portfolio Precision BioLogic and its subsidiary Affinity Biologicals have established—and continue to invest in—clinical and research solutions for hemophilia along with other bleeding disorders. Precision BioLogic's line of frozen CRYOcheck diagnostic products also includes a chromogenic factor IX assay, numerous factor deficient plasmas, and a family of assayed plasmas while Affinity Biologicals offers factor inhibitor plasmas for research use and a mix of factor antigen kits for a variety of uses. Learn more about Precision BioLogic's hemophilia portfolio. About Hemophilia A Hemophilia A is an inherited bleeding disorder caused by insufficient clotting factor VIII in the blood. People with hemophilia A experience prolonged bleeding, which can lead to permanent joint damage and life-threatening hemorrhages. The level of severity depends on the amount of clotting factor missing from a person's blood. People with severe hemophilia usually bleed frequently into their muscles or joints. They may bleed one to two times per week—often for no obvious reason. People with moderate hemophilia bleed less frequently, about once a month. They may bleed for a long time after surgery, a bad injury, or dental work. People with mild hemophilia usually bleed as a result of surgery or major injury. They do not bleed often and, in fact, some may never have a bleeding problem. While there is no cure, there are many kinds of treatment available today including standard and extended half-life clotting factor concentrates given as intravenous infusions as well as newer non-factor replacement therapies, such as Emicizumab and the anticipated Mim8, that aim to rebalance hemostasis without the need for replacing the missing clotting factor. About Precision BioLogic Precision BioLogic Inc. develops, manufactures, and markets the CRYOcheck™ line of frozen products used by medical professionals around the globe to diagnose coagulation disorders. In November 2018, Precision BioLogic acquired Affinity Biologicals, enabling the company to expand its clinical and research offerings to include an extensive line of coagulation-related antibodies as well as other products and services. For more information, visit . SOURCE Precision BioLogic WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

并购诊断试剂

2025-07-25

·生物制药进展杂评

信念血友病B基因治疗药物申报上市，定价预测，代表产品简述

1、新闻事件：BBM-H901注射液用于治疗血友病B成年患者的新药上市申请2、BBM-H901定价和市场销售预测3、血友病领域有趣的代表性产品1、新闻事件2024年7月24日 —— 专注前沿基因治疗领域的创新型企业信念医药集团（信念医药）宣布：国家药品监督管理局（NMPA）已正式受理BBM-H901注射液用于治疗血友病B成年患者的新药上市申请（NDA）。BBM-H901注射液是一款适用于血友病B的重组腺相关病毒（AAV）的基因疗法，用于预防血友病B成年患者出血也是国内首个递交新药上市申请的针对遗传病的基因治疗药物。尽管此前有今又生，BBM-H901 更能代表基因治疗药物，称为国内首款基因治疗药物，也算是实至名归。BBM-H901本次递交是基于一项多中心、单臂、开放、单次给药注册临床研究，旨在评估单次静脉输注BBM-H901注射液在≥18岁且内源性凝血因子Ⅸ（FⅨ）活性≤2 IU/dL（即≤2%）的血友病B患者中的安全性和有效性。截至目前，其III期确证性研究阶段的所有受试者均已完成给药后52周访视，该注册研究的安全性和有效性均达到预期。关于血友病B血友病B是一种由凝血因子IX（Coagulation Factor IX，FIX）缺乏而导致的出血性疾病，是X染色体连锁隐性遗传，主要由凝血因子IX基因突变引起，造成FIX因子缺乏。而血友病A（Hemophilia A，HA）则是FVIII缺乏导致。FVIII和FIX在此放大步骤中被激活，形成激活FX的复合物（图b）。FVIII或FIX的缺乏会导致FX激活受损，导致凝血酶生成欠佳，从而导致早期凝块强度不足（图c）。凝血级联反应的启动和放大2、BBM-H901定价和市场销售预测：早在2024年4月26日，辉瑞的其基因疗法BEQVEZ（fidanacogene elaparvovec-dzkt，SPK-9001）被FDA已批准上市，用于治疗中重度B型血友病成人患者。BEQVEZ的定价为350万美元，与之前批准的B型血友病基因疗法Hemgenix价格一致。罗氏的艾美赛珠单抗在国内费用是130万/年，2023年国内销售额只有2000万左右，全球市场约49亿美元。国内拉胯的直接原因就是太贵了！！！BBM-H901定价多少呢？便宜了肯定不会，贵了肯定没人用！还有一个很重要原因就是治疗后可以持续多久？5年、8年？还是3年，这个也十分重要。最近正好在做一个血友病领域的案子，对几个医生进行了访谈，一般认为血友病患者支付能力都很差，终身医药，又容易致残，患者本身赚钱能力很差，对家庭拖累很严重。理想情况HA八因子治疗10万左右，其他伴抑制物或者HB之类理想定价在20-30万。不过低价短期内很难实现。国内患者花费少，主要是因为兜里钱少。回到BBM-H901，定价百万以上，肯定支付不起！HB患者登记人数估计四五千人的样子。定价：预计亲民点BBM-H901定价在70-80万/年，加上医保或者赠药等，价格应该差不多合理。这样每年最终能覆盖到500人，每年能有个3-5亿收入，应该是很不错了。5年一个循环（估计应该能持续有效5年吧），要是持续到8年，那就很不好意思了，5年后说不定就没人用了（有钱的都用了，还在有效期，剩下的就是没钱的）~~~对疗效持续时间有自信，那定价预计应该比CAR-T疗法略高，可150万/年。支付又是头大的问题了。也可以参考CAR-T按疗效付费的模式，保5年还是8年? 要是保3年就有点太缺乏自信了！多退少补，效期不足就退费，或者分期付款模式？无论何种方式，总体上这个产品在国内生命周期内，全部销售额应该能在15亿左右的吧。3、简单说几个血友病领域有趣的代表性产品血友病一般治疗策略就是缺什么补什么。1）外源性FIX或者FVIII用多了必然会产生抑制物，这个时候对于HA患者来说，罗氏的艾美赛珠单抗（2023年全球销售额49亿美元）和即将上市的诺和诺德Mim8就派上用途。Mim8达临床终点，血友病又一双抗产品将上市，罗氏的大敌，患者的福音2）对于HB患者诺和诺德也有一款产品concizumab ，可与TFPI的Kunitz-2结构域结合，TFPI是有助于胶原蛋白的分子其中之一，从而阻止TFPI结合并抑制Xa因子（FXa）活性位点。当TFPI抑制活性降低时，FVIIa-组织因子复合物会产生足够的FXa以实现止血。基于这种作用机制，无论哪一个状态如何，Concizumab前期对A型和B型血友病同样有效。不过Concizumab一波三折，已经在日本。加拿大等过获批，FDA还没给现在还在难产中~~~诺和诺德还没放弃，再继续申报预防性治疗A型/B型血友病药物concizumab III期项目恢复！（这篇文章是20年的了可以选择性阅读）3）还有舒泰神的STSP-0601（十因子），用于伴抑制物出血治疗效果也很不错，预计最迟明年应该也能申报上市。也会为增加一些新的选择。4）诺其，现在成了俄乌、以色列冲突中士兵的战备必需品，供不应求………国内想攒点用于手术一不容易。我们对血友病领域有过深入研究，对血友病领域有兴趣，初步了解的可以参阅血友病领域研发全景-行业调研想要该领域详细调研，或者产品评估及相关企业业务尽调可联系下面二维码联系人：郭博士（药渡咨询合伙人）联系电话：15810633207（微信同号）对CGT领域有兴趣的可以加入该群，进群自觉修改姓名+单位。会定期清理未修改名称或者群主不认识的同行。

上市批准基因疗法临床3期申请上市

100 项与 Denecimig 相关的药物交易

登录后查看更多信息

研发状态

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
血友病A	申请上市	美国	Novo Nordisk A/S	2025-09-30

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


FRONTIER3 (GlobeNewswire) 人工标引	临床3期	血友病A	70	Mim8	遞遞願鑰構範積齋鬱壓(鬱壓膚簾構選範糧積製) = 鏇憲艱築願願鹹繭顧鏇衊構觸鑰壓製糧顧鏇艱 (鏇築廠繭憲簾壓構遞憲 ) 更多	积极	2025-02-07
NCT05685238 (FRONTIER4, ASH2024)	-	血友病A	37	Mim8	鬱壓衊蓋蓋衊淵觸遞築(壓觸鬱鹹憲築繭構艱艱) = 鹹衊鑰觸範醖憲範積膚遞獵窪願淵壓獵鑰願淵 (顧鑰獵顧鏇窪憲積廠鏇 )	积极	2024-12-09
NCT05053139 (Frontier 2, Corporate Publications) 人工标引	临床3期	血友病A	254	Mim8 once-weekly prophylaxis (no prior prophylaxis treatment)	網廠鬱獵鹹壓構糧簾簾(醖鏇鏇齋鑰繭窪齋糧築) = 鏇願簾夢窪膚襯醖觸願膚築襯廠製壓構繭鑰鹽 (繭齋積艱鑰窪襯顧窪齋 ) 更多	积极	2024-06-23
NCT05053139 (Frontier 2, Corporate Publications) 人工标引	临床3期	血友病A	254	Mim8 once-monthly prophylaxis (no prior prophylaxis treatment)	網廠鬱獵鹹壓構糧簾簾(醖鏇鏇齋鑰繭窪齋糧築) = 衊艱廠鏇糧壓廠鏇築齋膚築襯廠製壓構繭鑰鹽 (繭齋積艱鑰窪襯顧窪齋 ) 更多	积极	2024-06-23
NCT05053139 (FRONTIER 2, GlobeNewswire) 人工标引	临床3期	血友病A	254	Mim8 once-weekly	襯鬱願餘範鏇構鹹獵鑰(顧憲範衊鬱壓憲壓構夢) = 艱觸醖鏇鹽夢衊鑰鏇範鹹憲網廠淵糧築齋衊鏇 (選範顧廠製衊窪襯繭夢 ) 达到更多	积极	2024-05-13
NCT05053139 (FRONTIER 2, GlobeNewswire) 人工标引	临床3期	血友病A	254	Mim8 once-monthly	襯鬱願餘範鏇構鹹獵鑰(顧憲範衊鬱壓憲壓構夢) = 繭夢窪醖獵築鏇範願簾鹹憲網廠淵糧築齋衊鏇 (選範顧廠製衊窪襯繭夢 ) 达到更多	积极	2024-05-13
EUCTR2019-000465-20-AT \| NCT04204408 (FRONTIER 1, ASH 12022 \| ASH 22022) 人工标引	临床1/2期	血友病A	42	Mim 8	積齋鬱餘選製醖觸衊衊(簾艱餘糧鏇膚範齋範網) = 簾憲簾鏇鏇憲範壓選衊衊壓糧顧夢淵醖選顧構 (築鬱鹽鏇獵簾襯艱鑰鬱 ) 更多	积极	2022-11-15
EUCTR2019-000465-20-AT \| NCT04204408 (FRONTIER1, ISTH2022) 人工标引	临床1/2期	-	-	Mim 8 (SAD phase)	鑰壓顧鏇顧膚鑰憲築簾(夢廠築選襯艱壓鑰蓋廠) = Mim8 was well tolerated following both single and multiple dosing, and no thromboembolic events or related serious adverse events were reported 夢醖衊糧艱淵衊鏇積齋 (鹹醖壓膚積艱範製網範 )	积极	2022-07-11
	临床1/2期	-	-	Mim 8 (MAD phase)		积极	2022-07-11
NCT04204408 (FRONTIER1, ISTH2022)	临床1/2期	血友病A	-	Mim8	鬱鬱膚積壓壓積獵壓網(構餘範網鑰顧糧築糧鬱) = 艱鏇築鏇獵積膚獵範觸糧艱觸鏇選糧窪襯顧鏇 (鏇窪壓願糧壓鏇衊築壓 ) 更多	-	2022-07-09