RegulationBreakthrough Therapy (United States), Orphan Drug (United States), Orphan Drug (European Union), Orphan Drug (Australia), Priority Review (Australia)

Structure/Sequence

Sequence Code 110850L

Source: *****

Sequence Code 110854H

Source: *****

Clinical Trials associated with Concizumab-mtci

NCT06831734

/ Not yet recruitingNot Applicable

Special Use-results Surveillance on Use of Alhemo in Patients With Haemophilia A or Haemophilia B Without Inhibitors A Multi-centre, Open-label, Non-interventional Post-marketing Study to Investigate the Long-term Safety and Clinical Parameters of Treatment With Alhemo in Patients With Haemophilia A or Haemophilia B Without Inhibitors in Routine Clinical Practice Conditions in Japan

The purpose of the study is to investigate the safety and effectiveness of Alhemo in participants under real-world clinical practice in Japan. Total duration of this study is about 6 years. Participants enrolment will be completed in the first 4 years. The observation period of this study will last for about 2 years for each enrolled participant.

Start Date31 Mar 2025

Sponsor / Collaborator

Novo Nordisk A/S

NCT06285071

/ Enrolling by invitationNot Applicable

A Multi-centre, Open-label, Single-arm, Non-interventional Post-marketing Study to Investigate Safety and Clinical Parameters of Alhemo® Under Routine Clinical Practice in Japan

The purpose of the study is to investigate the safety and effectiveness of Alhemo® in all participants under real-world clinical practice in Japan. Participants will get Alhemo® as prescribed by the study doctor. The study will last for about 2 years.

Start Date08 Aug 2024

Sponsor / Collaborator

Novo Nordisk A/S

NCT05135559

/ RecruitingPhase 3

Open-label Study Investigating Efficacy, Safety and Pharmacokinetics of Concizumab Prophylaxis in Children Below 12 Years With Haemophilia A or B With or Without Inhibitors

This study will test how well a new medicine called concizumab works for participants who have haemophilia A or B with or without inhibitors. The purpose is to show that concizumab can prevent bleeds and is safe to use.
Participants will have to inject the study medicine every day under the skin with a pen-injector.
The study will last for at least 2 years and up to about 4 years. The length of time the participant will be in the study depends on if the study medicine will be available for purchase in their country.

Start Date24 Mar 2022

Sponsor / Collaborator

Novo Nordisk A/S

100 Clinical Results associated with Concizumab-mtci

100 Translational Medicine associated with Concizumab-mtci

100 Patents (Medical) associated with Concizumab-mtci

Literatures (Medical) associated with Concizumab-mtci

01 Apr 2025·The Journal of the Association of Physicians of India

Revolutionizing Treatment Strategies through Inhibition of Tissue Factor Pathway Inhibitor: A Promising Therapeutic Approach for Hemophilia Management.

Review

Author: Shaikh, Bilal Ahmed ; Aggarwal, Sunita ; Seth, Tulika ; Nigam, Rajendra K ; Jain, Ankur ; Baveja, Avriti ; Atri, Sudhir ; Sharma, Vishnu ; Digra, Sanjeev Kumar ; Vyas, Bhadresh ; Gupta, Naresh

Hemophilia, an X-linked genetic bleeding disorder, is caused by the deficiency of coagulation factors VIII (hemophilia A) or IX (hemophilia B). Regular replacement therapy with the missing clotting factor is an effective standard-of-care treatment. However, it comes with a significant fallout of frequent intravenous dosing with poor compliance, the risk of inhibitor development, and a substantial treatment burden. Research has progressed from missing clotting factors and factor VIII mimetics to the most recent rebalancing therapy that suppresses tissue factor pathway inhibitor (TFPI). Thrombin generation is restricted by TFPI, which inhibits the tissue factor-mediated activation of factor VII. This promising therapeutic approach rebalances hemostasis by inhibiting TFPI, a critical regulator of the extrinsic coagulation pathway, thereby increasing thrombin generation. Novel monoclonal antibodies (concizumab and marstacimab) enhance thrombin generation by blocking TFPI to restore hemostasis. Clinical trials have demonstrated good clinical efficacy and safety of these anti-TFPI, besides their convenient subcutaneous administration using pen devices. These innovative therapies have the potential to enhance the quality of life (QoL) of people with hemophilia. This review provides a comprehensive overview of the clinical development, therapeutic potential, challenges, and prospects of anti-TFPI in the management of hemophilia.

01 Feb 2025·JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Examining downstream effects of concizumab in hemophilia A with a mathematical modeling approach

Article

Author: Mast, Alan E ; Miyazawa, Kenji ; Kjalke, Marianne ; Leiderman, Karin ; Wufsus, Adam R ; Wufsus, Adam R. ; Dockal, Michael ; Mast, Alan E.

BACKGROUND:

Tissue factor (TF) pathway inhibitor (TFPI) is an anticoagulant protein that inhibits factor (F)Xa, the TF-FVIIa-FXa complex, and early forms of the prothrombinase complex. Concizumab is a monoclonal antibody that blocks FXa inhibition by TFPI and reduces bleeding in hemophilia.

OBJECTIVES:

To examine how concizumab impacts various reactions of TFPI to restore thrombin generation in hemophilia A using mathematical models.

METHODS:

A compartment model was used to estimate plasma concentrations of free concizumab and its complexes with TFPIα and TFPIβ. Concizumab was integrated into a flow-mediated mathematical model of coagulation, and a small injury was simulated under hemophilia A conditions. Simulations were then analyzed to determine how concizumab's blockade of TFPI anticoagulant activities, specifically the inhibition of FXa in plasma and on platelets, inhibition of TF:FVIIa at the subendothelium, and prior sequestration of plasma TFPIα to the endothelium via TFPIβ, altered thrombin generation.

RESULTS:

Concizumab improved simulated thrombin generation in hemophilia A by simultaneously altering all 3 mechanisms of the TFPI anticoagulant blockade examined. Concizumab sequestered ∼75% of plasma TFPIα through the formation of ternary TFPIα-concizumab-TFPIβ-complexes. For all TF levels, reducing the TFPIα plasma concentration had the largest impact on the lag time, followed by blocking TFPIα inhibition of TF:FVIIa:FXa and subsequently by blocking TFPIα inhibition of FXa in plasma and on the platelet surface.

CONCLUSION:

The effectiveness of concizumab is mediated through the blockade of TFPI anticoagulant activities in plasma and on multiple physiological surfaces. An important and previously unrecognized function of concizumab was the sequestration of plasma TFPIα to the endothelium.

01 Feb 2025·Research and Practice in Thrombosis and Haemostasis

Concizumab prophylaxis in people with hemophilia A or B without inhibitors: patient-reported outcome results from the phase 3 explorer8 study

Article

Author: Findley, Amy ; Eichler, Hermann ; von Mackensen, Sylvia ; Villarreal Martinez, Laura ; Benson, Gary ; Young, Guy ; Apte, Shashikant ; Sathar, Jameela ; Mazini Tavares, Camila M ; Matsushita, Tadashi ; Puggaard Ravn, Morten ; Angchaisuksiri, Pantep

Background:

Patient-reported outcomes (PROs) can provide useful insights into patient perception of concizumab, an anti-tissue factor pathway inhibitor monoclonal antibody intended for once-daily, subcutaneous prophylaxis for hemophilia A (HA) or hemophilia B (HB), with and without inhibitors.

Objectives:

To evaluate PROs from the phase 3 explorer8 study (NCT04082429).

Methods:

Male patients aged ≥12 years with HA/HB without inhibitors were enrolled and randomized 1:2 to no prophylaxis/on-demand treatment (arm 1) or concizumab prophylaxis (arm 2) or allocated to concizumab prophylaxis (arms 3 and 4). PRO questionnaires included the 36-item short-form health survey version 2, Haemophilia Quality of Life Questionnaire for Adults, Hemophilia Treatment Experience Measure, and Haemophilia Patient Preference Questionnaire.

Results:

Estimated treatment difference for change in 36-item short-form health survey version 2 "bodily pain" and "physical functioning" from baseline to week 24 between patients in arms 1 and 2 was 9.5 points (95% CI, 2.4 to 16.7) and 0.3 points (95% CI, -5.1 to 5.6), respectively. Estimated treatment difference at week 24 between patients in arms 1 and 2 was -18.0 points (95% CI, -26.4 to -9.5) for Haemophilia Quality of Life Questionnaire for Adults "total score" and -16.8 points (95% CI, -32.2 to -1.4) for "physical health." Hemophilia Treatment Experience Measure and Haemophilia Patient Preference Questionnaire results favored concizumab prophylaxis over no prophylaxis or previous treatment.

Conclusion:

PRO data from the phase 3 explorer8 study provided additional support for concizumab prophylaxis compared with no prophylaxis as a treatment option for patients with HA/HB.

News (Medical) associated with Concizumab-mtci

21 Apr 2025

·www.prnewswire.com

Roche's HEMLIBRA Achieves Significant Uptake Across Key Hemophilia A Markets | DelveInsight

HEMLIBRA has strong market potential due to its unique, subcutaneous prophylactic treatment for Hemophilia A, including patients with and without factor VIII inhibitors. With increasing global diagnosis rates and expanding access in emerging markets, HEMLIBRA is poised for continued growth. LAS VEGAS, April 21, 2025 /PRNewswire/ -- DelveInsight's " HEMLIBRA Market Size, Forecast, and Market Insight Report" highlights the details around HEMLIBRA, a bispecific factor IXa- and factor X-directed antibody indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients ages newborn and older with hemophilia A (congenital factor VIII deficiency) with or without factor VIII inhibitors. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of HEMLIBRA. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Chugai/Genentech/Roche's HEMLIBRA (emicizumab) Overview HEMLIBRA is a bispecific antibody that targets both factor IXa and factor X, two key proteins in the body's natural blood clotting process. Bridging these proteins it helps restore clotting function in individuals with hemophilia A. HEMLIBRA is used as a preventive treatment and is administered as a ready-to-use subcutaneous injection. It can be given once a week, every two weeks, or every four weeks—following an initial four-week period of weekly doses. Developed by Chugai Pharmaceutical Co., Ltd., HEMLIBRA is co-developed internationally by Chugai, Roche, and Genentech. In the U.S., Genentech markets the drug under the name HEMLIBRA (emicizumab-kxwh), with "kxwh" assigned per FDA guidance on naming biological products. HEMLIBRA can be prescribed in various dosage regimens. The initial loading dose is 3 mg/kg administered via subcutaneous injection once weekly for the first four weeks. This is followed by one of the following maintenance dosing options: 1.5 mg/kg once every week, 3 mg/kg once every two weeks, or 6 mg/kg once every four weeks The choice of maintenance dose should be guided by the healthcare provider's judgment, taking into account the potential for improved patient adherence with certain regimens. The prophylactic use of bypassing agents should be stopped the day before initiating HEMLIBRA prophylaxis. Factor VIII (FVIII) prophylaxis may be continued during the first week of HEMLIBRA treatment. Learn more about HEMLIBRA projected market size for hemophilia A @ HEMLIBRA Market Potential Hemophilia A is marked by bleeding episodes, though in mild and moderate cases, these initial episodes are often less intense, which can lead to delays in diagnosis as patients may not seek medical attention promptly. Moreover, early detection is frequently hindered by the lack of access to adequate diagnostic facilities. In 2023, the number of treated prevalent Hemophilia A cases across the 7MM was estimated at around 45,000, with projections indicating growth throughout the forecast period, according to DelveInsight. Among hemophilia A patients—whether or not they have inhibitors—those without inhibitors represent a larger portion of the population. The standard treatment approach focuses on replacing the missing clotting factor, FVIII, to support normal clot formation and minimize or prevent bleeding episodes. Initially, FVIII replacement was sourced from whole blood and plasma, but advances in biotechnology led to the development of recombinant FVIII (rFVIII) therapies, significantly enhancing disease management by offering more precise and efficient options. More recently, innovative therapies using novel mechanisms to prevent bleeding are expanding the treatment arsenal. Several approved therapies for hemophilia A include ALTUVIIIO (Sanofi/Sobi), ROCTAVIAN (BioMarin), OBIZUR (Takeda), ALHEMO (Novo Nordisk), HEMLIBRA (Chugai/Genentech/Roche), ESPEROCT (Novo Nordisk), JIVI (Bayer), WILATE (Octapharma), and ADYNOVATE (Takeda), among others. Notably, ROCTAVIAN, a gene therapy developed by BioMarin, represents a major advancement—it offers a one-time treatment by introducing functional FVIII genes into the patient's cells, potentially restoring normal clotting ability and reducing the need for regular injections. With a wholesale acquisition cost of USD 2.9 million, BioMarin has already facilitated reimbursement in Europe and administered treatment to a patient in Germany in Q2 2023 at a discounted price of approximately USD 900,000. Looking ahead, the hemophilia A treatment landscape is expected to evolve significantly from 2024 to 2034, fueled by the introduction of new therapies currently in development. According to DelveInsight, the hemophilia A market across the 7MM is projected to grow from USD 11.1 billion in 2023 at a robust CAGR through 2034. Nonetheless, factors like delayed diagnosis, limited awareness, and high treatment costs may pose barriers to market growth. Discover more about the hemophilia A market in detail @ Hemophilia A Market Report Emerging Competitors of HEMLIBRA Emerging therapies such as SPK-8011 (Roche [Spark Therapeutics]), fitusiran (Sanofi), marstacimab (Pfizer), Mim8 (Novo Nordisk), and others are being developed to provide safe and effective treatment options. Companies like GeneVentiv Therapeutics are also working on advancements like Adeno-associated Virus (AAV)-based universal gene therapy for hemophilia (GENV-HEM [AAV8.Fva]). Fitusiran is a subcutaneously administered small interfering RNA (siRNA) therapy under development for the prophylactic treatment of hemophilia A and B, regardless of the presence of inhibitors. Its mechanism involves reducing levels of antithrombin—a protein that inhibits clot formation—to enhance thrombin generation, restore hemostatic balance, and prevent bleeding episodes. In January 2018, Sanofi and Alnylam Pharmaceuticals restructured their RNAi therapeutics collaboration to better align and accelerate the development and commercialization of select treatments for rare genetic diseases. Through this revised agreement, Sanofi obtained global rights to develop and commercialize fitusiran. Marstacimab (PF-06741086) is a monoclonal IgG1 antibody that targets the Kunitz 2 domain of the Tissue Factor Pathway Inhibitor (TFPI). It is being developed to prevent or reduce bleeding events in patients with severe hemophilia A or B (defined as <1% activity of Factor VIII or IX), whether or not they have inhibitors. The FDA granted Fast Track Designation to marstacimab in September 2019 for its potential use in patients with inhibitors. In December 2023, Pfizer announced that the FDA had accepted its Biologics License Application (BLA) for marstacimab, supported by data from the Phase III BASIS study involving patients without FVIII or FIX inhibitors. BBM-H803 is Belief BioMed's first gene therapy candidate for hemophilia A and its second product to gain Investigational New Drug (IND) approval from China's National Medical Products Administration (NMPA). In December 2022, the U.S. FDA granted Orphan Drug Designation to BBM-H803. To know more about the number of competing drugs in development, visit @ HEMLIBRA Market Positioning Compared to Other Drugs Key Milestones of HEMLIBRA In June 2022, Chugai received approval in Japan for acquired Hemophilia A based on a domestic Phase III AGEHA study. In October 2018, HEMLIBRA was approved by the US FDA for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A without factor VIII inhibitors (FVIII). However, this therapy, which was co-developed by Genentech, Chugai, and Roche, was already FDA-approved in 2017 for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with FVIII inhibitors In April 2018, the US FDA granted breakthrough therapy designation to Genentech's HEMLIBRA for people with hemophilia A without factor VIII inhibitors. In October 2014, the US FDA granted orphan drug designation to HEMLIBRA for the treatment of Hemophilia A. Discover how HEMLIBRA is shaping the hemophilia A treatment landscape @ HEMLIBRA Cost HEMLIBRA Market Dynamics HEMLIBRA developed by Roche/Genentech, has significantly reshaped the hemophilia A treatment landscape since its approval. Designed as a bispecific monoclonal antibody, HEMLIBRA mimics the function of factor VIII by bridging activated factor IX and factor X, offering prophylactic treatment for patients with hemophilia A with or without factor VIII inhibitors. Its subcutaneous administration and less frequent dosing schedule (weekly, biweekly, or monthly) offer considerable convenience compared to traditional intravenous factor replacement therapies, making it a preferred option for both patients and caregivers. The market dynamics for HEMLIBRA have been largely influenced by its clinical efficacy, improved patient compliance, and strong safety profile. Since its launch, it has rapidly gained market share, especially among inhibitor patients where treatment options were limited and complex. In non-inhibitor patients as well, the shift away from frequent IV infusions has led to strong adoption rates, allowing HEMLIBRA to cannibalize traditional recombinant factor VIII therapies. Furthermore, its entry has pressured pricing and market share dynamics across the hemophilia therapeutic space, triggering strategic shifts by legacy players like Takeda, Sanofi, and Bayer. However, HEMLIBRA's market leadership is being challenged by the evolving competitive landscape. The emergence of gene therapies, which offer the potential for long-term or even curative outcomes, introduces a new dimension to treatment decisions. While some gene therapies are still in early stages of adoption and face challenges related to durability, safety, and patient eligibility, they represent a looming threat to the long-term dominance of HEMLIBRA, particularly in younger, eligible patient populations. Going forward, the market trajectory for HEMLIBRA will depend on several factors: expanding label indications (such as pediatric use), long-term safety and efficacy data, continued pricing negotiations with payers, and how effectively Roche can defend its position against next-generation therapies. Additionally, with increasing emphasis on patient-centric outcomes and value-based care, HEMLIBRA's ability to demonstrate long-term cost-effectiveness versus both traditional and novel competitors will be key to sustaining its market share. Dive deeper to get more insight into HEMLIBRA's strengths & weaknesses relative to competitors @ HEMLIBRA Market Drug Report Table of Contents Related Reports Hemophilia A Market Hemophilia A Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key hemophilia A companies, including BioMarin Pharmaceutical, Roche (Spark Therapeutics), ApcinteX, Novo Nordisk, Sanofi (Genzyme), Alnylam Pharmaceuticals, Pfizer, Sangamo Therapeutics, Bayer, Ultragenyx Pharmaceutical, among others. Hemophilia A Pipeline Hemophilia A Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key hemophilia B companies, including UniQure Biopharma B.V., CSL Behring, Pfizer, Spark Therapeutics, Genzyme, a Sanofi Company, Alnylam Pharmaceuticals, Novo Nordisk, ApcinteX Ltd, Freeline Therapeutics, Sangamo Therapeutics, among others. Hemophilia B Market Hemophilia B Market Insight, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of market trends, market drivers, market barriers, and key hemophilia B companies such as Gem Pharmaceuticals, Lytix Biopharma, Incyte Corporation, Daiichi Sankyo, NantPharma, Iovance Biotherapeutics, Agenus, Eli Lilly and Company, Adaptimmune, Aadi, AVEO Pharmaceuticals, Amgen, among others. Hemophilia B Pipeline Hemophilia B Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key hemophilia B companies, including Shanghai Vitalgen BioPharma, Belief BioMed, TiumBio, Jiangsu Gensciences, Centessa Pharmaceuticals, Takeda, Sanofi, GC Biopharma Corp, Equilibra Bioscience LLC, Regeneron Pharmaceuticals, ISU Abxis, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Orphan DrugDrug ApprovalPhase 3Fast TrackBreakthrough Therapy

31 Mar 2025

·www.pharmaceutical-technology.com

Sanofi’s Qfitlia approved by FDA as first haemophilia therapy for all patients

Sanofi’s monthly subcutaneous treatment reduced annualised bleeding rates by 90%. Image credit: Shutterstock / LCV. Sanofi has won US Food and Drug Administration (FDA) approval for haemophilia treatment Qfitlia (fitusiran), as the company aims to use the drug’s versatility to set it apart from rivals in a crowded market. Qfitlia is approved for adults and adolescents with haemophilia A or B with or without factor VIII or IX inhibitors. The ability for Qfitlia to be used regardless of inhibitor status gives an advantage over other approved therapies. Inhibitors are antibodies that some haemophilia patients develop that prevent factor replacement therapies from working properly. Sanofi’s drug is also the first antithrombin-lowering therapy for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with the disease. Haemophilia A and haemophilia B are genetic bleeding disorders caused by a dysfunction or deficiency of coagulation factor VIII or IX. Qfitlia does not replace the missing clotting factor, instead, it acts by reducing the amount of a protein called antithrombin, leading to an increase in thrombin, an enzyme critical for blood clotting. Qfitlia uses small-interfering RNA technology, which enables low treatment frequency, subcutaneous dosing, and low-volume injections. Qfitlia has demonstrated efficacy across the different types of haemophilia in three completed Phase III trials: ATLAS-INH (NCT03417102), ATLAS-A/B (NCT03417245), and ATLAS-PPX (NCT03549871). Compared to patients taking on-demand clotting factor concentrates and bypassing agents, Sanofi’s monthly subcutaneous treatment reduced annualised bleeding rates by 90%. The most common adverse events (AEs) with the drug were viral and bacterial infection. Qfitlia does come with a boxed warning for thrombotic events and gallbladder disease, along with warnings about liver toxicity. The drug had previously gone through clinical holds – one in 2017 was due to a patient dying from a blood clot and a later one in 2020 was due to a non-fatal thrombotic event. The thrombotic risk was reduced after a revised scheme was identified by Sanofi. An ongoing Phase III open-label extension (OLE) study (NCT03754790) from the ATLAS programme is evaluating a revised dosing regimen, with Sanofi targeting as few as six injections per year. Market landscape Qfitlia’s monthly regimen already gives it an advantage over Novo Nordisk’s daily Alhemo (concizumab) and Pfizer’s weekly Hympavzi (marstacimab) , two of the most recent drugs approved by the FDA for haemophilia. Qfitlia’s edge over rivals is compounded due to inhibitor status versatility – Alhemo, for example, can only be used for the disease with inhibitors while Hympavzi can be used without. Pfizer recently shelved its other haemophilia drug, Beqvez (fidanacogene elaparvovec), citing low demand and interest. National Bleeding Disorders Foundation president and CEO Phil Gattone said: “Current treatment options can make people with haemophilia feel they need to choose between effective bleed control and convenient dosing schedules, leading to trade-offs when it comes to disease management. Qfitlia takes a novel approach to providing protection for people living with haemophilia while reducing the frequency of dosing for patients and their families.” The global market for haemophilia drug sales in the US is expected to be $7.2bn by 2030, according to GlobalData’s Pharma Intelligence Center. Roche’s Hemlibra (emicizumab), indicated for inhibitors and non-inhibitors but only for haemophilia A patients, is currently the best-selling drug in the field, generating $4.9bn globally in 2024. GlobalData is the parent company of Pharmaceutical Technology .

Phase 3Drug ApprovalClinical Result

28 Mar 2025

·www.fiercepharma.com

Sanofi's versatile Qfitlia scores FDA nod to enter crowded hemophilia market

The FDA has signed off on another hemophilia treatment, Sanofi’s fitusiran, which sets itself apart as the only treatment for all types of hemophilia. Over the last three years, the FDA has approved six new hemophilia drugs, including three gene therapies.Into this crowded treatment landscape comes another new medicine as the FDA has signed off on Sanofi’s Qfitlia (fitusiran), which sets itself apart as the only treatment for all types of hemophilia.Not only is Qfitlia for those with hemophilia A and B, but unlike most treatments for the disorder, it also can be used by patients regardless of their inhibitor status.With hemophilia patients, “inhibitors” are antibodies that counter the effectiveness of clotting factor replacement therapies. Some hemophilia A patients may develop factor VIII inhibitors, while factor IX inhibitors may arise in some hemophilia B patients.“It’s a really exciting time for people living with hemophilia as it relates to the emergence of new therapies,” Craig Benson, M.D., Sanofi’s hemophilia program leader, said in an interview with Fierce Pharma. “We’re obviously excited about fitusiran because it’s the first therapy in the United States that can be used by all patients.”Qfitlia can be administered under the skin starting once every two months, and its dose and frequency can be adjusted using a companion diagnostic test. A Sanofi spokesperson said that the average annual wholesale acquisition cost for the medicine for the majority of patients will be $642,000.“Today’s approval of Qfitlia is significant for patients with hemophilia because it can be administered less frequently than other existing options,” said Tanya Wroblewski, M.D., deputy director of the FDA’s division of non-malignant hematology, said in a statement Friday.As a small interfering RNA (siRNA) drug, Qfitlia mimics a natural cellular process that regulates gene expression. By suppressing antithrombin (AT), a protein that inhibits blood clotting, Qfitlia promotes thrombin generation, which helps prevent bleeding episodes that plague those with hemophilia.“It’s a different pathway in the coagulation system to rebalance hemostasis,” Benson said. “Our coagulation system is a delicate balance, for all of us, between allowing our blood to stay slippery and smooth and flow, versus more of a clot.”In its annual Drugs to Watch report, Clarivate has referred (PDF) to Qfitlia as a “potentially transformative therapy,” and has tabbed its sales potential at $1 billion by 2030. In phase 3 trials, Qfitlia has shown relatively equal effectiveness in patients with each of the four classifications of hemophilia. It also has performed well across hemophilia patients who altogether underwent 60 major surgeries.Two phase 3 trials backed the approval, both showing that prophylactic use of Qfitlia could reduce annualized bleeding rate by 90% compared with the control arm. In both studies, patients received nine months of treatment.In the ATLAS A/B trial, which enrolled 120 non-inhibitor patients with hemophilia A or B who had been previously treated with on-demand clotting factor concentrates, 51% of those who were given a monthly prophylactic dose of Qfitlia had no annual bleeds versus 5% of those who received on-demand clotting factors.In the ATLAS-INH trial, which enrolled 57 severe hemophilia A and B inhibitor patients, 66% on Qfitlia had no annualized bleeding episodes versus 5% of those in the control group who received an on-demand bypassing agent.Qfitlia is the second RNA interference drug to score a highly anticipated FDA nod in recent days. Last week, the agency signed off on Alnylam’s Amvuttra to treat transthyretin amyloid cardiomyopathy (ATTR-CM) patients.Sanofi originally partnered on the development of both Amvuttra and Qfitlia. But in 2018, the companies agreed that Alnylam would take full control of Amvuttra while Sanofi would do the same for Qfitlia.Both companies struggled to develop fitusiran as it was hit with clinical holds in 2017 and 2020. The first came after a patient died from a blood clot in a phase 2 study. Eventually, Sanofi found a dose that has reduced thromboembolic events. Sizing up the competitionDespite entering a competitive market with a limited patient population, Qfitlia has a chance to carve out a niche as it has much to offer compared to current factor VIII and factor IX replacement treatments, which are administered intravenously once or twice a week and present the risk of infection.“The unique advantage of fitusiran—and siRNA technology—is that there’s no need for an IV. It’s a very small-volume subcutaneous injection,” Benson said. “Because it works on the RNA level, it only has to be dosed every other month.”Qfitlia also holds a convenience edge over two of the most recently approved hemophilia drugs—Novo Nordisk’s Alhemo and Pfizer’s Hympavzi.Though both can be used by hemophilia A and B patients and both are delivered subcutaneously, Alhemo is dosed daily and Hympavzi is dosed weekly. In addition, Alhemo is limited to inhibitors and Hympavzi is only for non-inhibitors.With sales of $4.9 billion in 2024, Roche’s hemophilia A treatment Hemlibra is the dominant drug in the indication. It is delivered subcutaneously and can be used by inhibitors and non-inhibitors.“(Fitusiran) can be kept at room temperature, and the volume is very low. This is the one drug that, from a convenience aspect alone, is actually better than Hemlibra,” a hematologist told Clarivate in its anonymous survey. Sanofi is not new to the market. It partnered with Sobi on hemophilia A treatment Elocate, which was approved in 2014 and had a solid run before Hemlibra took over as the market leader. In 2023, Sanofi and Sobi scored an FDA nod for Elocate follow-on Altuviiio, a prophylactic and rescue medication which has a longer half-life than other factor XIII replacement therapies and is poised to challenge Hemlibra. Analysts from ODDO BHF project the Altuviio’s sales will reach 1.3 billion euros ($1.4 billion) by 2029. There are roughly 30,000 people in the U.S. with hemophilia, including approximately a quarter with type B. As an X chromosome-linked condition, it primarily affects males. If untreated, bleeding episodes in the joints can lead to a severe form of hemophilic arthropathy, which may require surgery to repair or replace damaged joints.

Clinical ResultDrug ApprovalPhase 3Phase 2Acquisition

100 Deals associated with Concizumab-mtci

External Link

KEGG	Wiki	ATC	Drug Bank
D11847	-	-	DB12820

R&D Status

10 top approved records.

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Indication	Country/Location	Organization	Date
Hemophilia	Japan	Novo Nordisk Pharma Ltd.	25 Sep 2023
Hemophilia A	Australia	Novo Nordisk Pharmaceuticals Pty Ltd.	05 Jul 2023
Hemophilia B	Canada	Novo Nordisk Canada, Inc.	10 Mar 2023

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04083781 (explorer7, CTgov)	Phase 3	Hemophilia B \| Hemophilia A	134	concizumab (Arm 1: Previous on Demand: No Prophylaxis)	luauvhlqrv(mnlbyrcfuy) = ngatoczrqb mtbzmkksms (aoixyymovj, ftapvpejah - pxkdkekeig) View more	-	27 Apr 2025
				Concizumab (Arm 2: Previous on Demand: Concizumab Prophylaxis (PPX))	luauvhlqrv(mnlbyrcfuy) = emexivrqcp mtbzmkksms (aoixyymovj, vejqdgsqsb - mqogkwanuz) View more
NCT04083781 (explorer7, FDA_CDER) Manual	Phase 3	Hemophilia B \| Hemophilia A	133	Alhemo	opgfvisiwx(nxgawwyjdh) = kbbbfukgpc xgigpnqyww (brkdmtxqrw, 1.01 - 2.87)	Positive	20 Dec 2024
				no prophylaxis	opgfvisiwx(nxgawwyjdh) = ijuzjsrcnr xgigpnqyww (brkdmtxqrw, 7.03 - 19.86)
explorer7 and explorer8 (ASH2023)	Phase 3	Hemophilia	278	Concizumab prophylaxis	rsuysbamui(mxvlpokmlo) = nrehqxylam wofxarvmrl (zxchuuoyyl )	-	09 Dec 2023
ISTH2023	Phase 3	-	-	Concizumab	eksgoztblt(ctxfayfvch) = icxmzovbfs vpzqcdmedc (geybqpvnry )	-	24 Jun 2023
				Concizumab	eksgoztblt(ctxfayfvch) = lpqlzvhbfp vpzqcdmedc (geybqpvnry, 21)
ISTH2023	Not Applicable	Hemophilia A	80	Concizumab pen-injector	ibzkqwatns(fmnftiwmyy) = utzkdiwcpi ahetkwncdn (ouxhztszjc, 91 - 100) View more	-	24 Jun 2023
NCT04083781 (explorer7, ASH 12022 \| ASH 22022) Manual	Phase 3	Hemophilia B \| Hemophilia A	25	Concizumab (Concizumab prophylaxis)	jpudrftjzp(lcliwjxwcu) = oiwypidvbi pxqaxcubvv (yoscvfqilj, -23.7 to -10.3)	Positive	15 Nov 2022
				Concizumab (non-concizumab prophylaxis treatment)	jpudrftjzp(lcliwjxwcu) = ffvfqnltuo pxqaxcubvv (yoscvfqilj, -9.4 to 15.3)
NCT04083781 (explorer7, GlobeNewswire) Manual	Phase 3	Hemophilia B	133	Concizumab	fdntgkpprp(vyuzsekxyc) = rconhodgha bowtywyxtc (ynbtinyqwa ) View more	Positive	10 Jul 2022
				no prophylaxis	ydepvkpxei(qpdlqobzya) = oyfhourydb ovmcwfzguo (tqwxlmmjoj )
NCT03196284 \| NCT03196297 (explore5 \| explore4, Pubmed \| Blood Adv) Manual	Phase 2	Hemophilia B	-	concizumab (explore4)	httzqkyosh(sdtwomitpc) = twtysirzwk tuweeclvgh (tvyrtcajrz, 3.2 - 7.2) View more	Positive	15 Mar 2022
				concizumab (explore5)	httzqkyosh(sdtwomitpc) = tzasqjpfbz tuweeclvgh (tvyrtcajrz, 4.1 - 9.9) View more
NCT02490787 (Explorer™3, Pubmed \| J Thromb Haemost) Manual	Phase 1	Hemophilia A	24	concizumab	bodjosrhng(bwkcsbskty) = No serious adverse events kzdleijnck (mqaeydtpqr )	Positive	01 Nov 2018
				Placebo

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