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Globe and Mail
16-05-2025
- Health
- Globe and Mail
Coagulation Factor Deficiency Market to Witness Significant Innovation and Growth by 2034
The Coagulation Factor Deficiency market is evolving rapidly, fueled by advances in gene therapy, long-acting clotting factors, and better prophylaxis. DelveInsight's report covers epidemiology and treatments for Hemophilia A, B, and rare factor deficiencies (FVII, FXI, FXIII). AAV-based gene therapies are driving new treatment goals and improving outcomes, especially for underserved patients. DelveInsight's ' Coagulation Factor Deficiency Market Report ' delivers detailed analysis of market dynamics, historical and forecasted epidemiology, and emerging therapies across the 7MM (US, EU5, and Japan), highlighting key players and trends driving this rare disease segment. Some of the Key Facts of the Coagulation Factor Deficiency Market Report: • In 2023, the coagulation factor deficiency market in the 7MM was valued at approximately USD 13.5 billion. • The US led the market, contributing around USD 7.2 billion. • There were about 105K prevalent cases across the 7MM, with the US accounting for nearly 34K. • Hemophilia A was the most common subtype among all coagulation factor deficiencies. • Marketed drugs for coagulation factor deficiency include ESPEROCT (Novo Nordisk), JIVI (Bayer), and WILATE (Octapharma). • In March 2025, the FDA approved QFITLIA (fitusiran) for routine prophylaxis to reduce bleeding episodes in patients (12+) with hemophilia A or B, with or without factor VIII or IX inhibitors. • In December 2024, Novo announced that the FDA approved concizumab, its tissue factor pathway inhibitor (TFPI) antagonist, as a once-daily treatment to prevent or reduce bleeding episodes in patients aged 12 and older with hemophilia A or B with inhibitors. • In October 2024, Novo Nordisk received support from European regulators for the approval of its anti-TFPI antibody, Alhemo (concizumab), as a preventive treatment for hemophilia. The company stated that, if approved, this therapy would provide the first subcutaneous prophylactic treatment to be administered once daily for individuals with hemophilia A or B who have inhibitors. • In March 2024, ReciBioPharm signed a collaboration agreement with GeneVentiv Therapeutics, a preclinical gene therapy company, to advance the development of an adeno-associated virus (AAV)-based universal gene therapy for hemophilia, and reportedly the first to treat hemophilia patients with inhibitors. • Emerging coagulation factor deficiency drugs include Concizumab, Fidanacogene Elaparvovec, RG6357 (SPK-8011), Fitusiran, Marstacimab, Giroctocogene Fitelparvovec, NNC0365-3769 A (MIM8), BT524, SerpinPC, STSP-0601, Marzeptacog Alfa, AB023, BAY2599023, OPK88005, and others. • Leading companies in the coagulation factor deficiency market include Novo Nordisk, Pfizer, Spark Therapeutics, Roche, Sanofi (Genzyme), Alnylam Pharmaceuticals, Sangamo Therapeutics, Biotest AG, Centessa Pharmaceuticals (ApcinteX), Staidson Biopharma Inc., GC Biopharma, Aronora, Inc., Bayer, Ultragenyx Pharmaceutical, OPKO Health, Inc., and others. • The rising incidence of coagulation factor deficiencies, along with ongoing advancements in treatment options, is driving the demand for more effective therapies for these conditions. To know in detail about the coagulation factor deficiency market outlook, drug uptake, treatment scenario, and epidemiology trends, click here: Coagulation Factor Deficiency Market Forecast Coagulation Factor Deficiency Overview Coagulation factor deficiencies, also referred to as bleeding or clotting disorders, are a group of inherited or acquired conditions caused by the absence or dysfunction of specific proteins that are essential for blood clotting. These proteins, known as coagulation factors, are vital for halting bleeding by forming clots to close off damaged blood vessels. There are 13 coagulation factors, each numbered from I to XIII, that work together in a complex process to maintain hemostasis. Deficiencies in any of these factors can lead to abnormal bleeding, with severity varying based on the specific factor affected and the extent of the deficiency. Get a free sample of the coagulation factor deficiency market report with key insights and emerging therapies here: Coagulation Factor Deficiency Epidemiology The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends. Coagulation Factor Deficiency Epidemiology Segmentation: The coagulation factor deficiency epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by: • Total diagnosed prevalent cases • Factor-specific prevalent cases Download the report to understand which factors are driving coagulation factor deficiency epidemiology trends @ Coagulation Factor Deficiency Epidemiology Forecast Coagulation Factor Deficiency Drugs Uptake, and Pipeline Development Activities The coagulation factor deficiency drugs uptake section examines the rate at which newly launched or upcoming potential drugs are being adopted in the coagulation factor deficiency market during the study period. This analysis covers drug uptake, patient adoption of therapies, and the sales performance of each drug. Additionally, the therapeutics assessment section highlights the drugs with the most rapid uptake, shedding light on the factors driving their widespread use. It also provides a comparative analysis of these drugs based on their market share. The report further delves into the coagulation factor deficiency pipeline development activities, offering key insights into various therapeutic candidates in different stages of development and the major companies behind these innovations. It also covers recent collaborations, acquisitions, mergers, licensing agreements, patent details, and other critical information related to emerging therapies. Coagulation Factor Deficiency Market Strengths • New gene therapies for coagulation factor deficiencies, like Hemophilia A and B, offer potential long-term or curative solutions. • Better awareness and diagnostic tools are enabling earlier detection and more effective management. Coagulation Factor Deficiency Market Weaknesses •Many therapies, particularly gene therapies, remain expensive, limiting accessibility. • Advanced therapies are not widely accessible in lower-income regions due to cost and healthcare limitations. Scope of the Coagulation Factor Deficiency Market Report • Study Period: 2020–2034 • Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan] • Key Coagulation Factor Deficiency Therapies: Concizumab, Fidanacogene Elaparvovec, RG6357 (SPK-8011), Fitusiran, Marstacimab, Giroctocogene Fitelparvovec, NNC0365-3769 A (MIM8), BT524, SerpinPC, STSP-0601, Marzeptacog Alfa, AB023, BAY2599023, OPK88005, and others. • Key Coagulation Factor Deficiency Companies: Novo Nordisk, Pfizer, Spark Therapeutics, Roche, Sanofi (Genzyme), Alnylam Pharmaceuticals, Sangamo Therapeutics, Biotest AG, Centessa Pharmaceuticals (ApcinteX), Staidson Biopharma Inc., GC Biopharma, Aronora, Inc., Bayer, Ultragenyx Pharmaceutical, OPKO Health, Inc., and others. • Coagulation Factor Deficiency Therapeutic Assessment: Coagulation factor deficiency, currently marketed, and coagulation factor deficiency emerging therapies • Coagulation Factor Deficiency Market Dynamics: Coagulation factor deficiency market drivers and Coagulation Factor Deficiency market barriers • Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies • Coagulation Factor Deficiency Unmet Needs, KOL's views, Analyst's views, Coagulation Factor Deficiency Market Access and Reimbursement To learn more about the key players and advancements in the coagulation factor deficiency treatment landscape, visit the Coagulation Factor Deficiency Market Analysis Report Table of Contents 1. Coagulation Factor Deficiency Market Report Introduction 2. Executive Summary for Coagulation Factor Deficiency 3. SWOT analysis of Coagulation Factor Deficiency 4. Coagulation Factor Deficiency Patient Share (%) Overview at a Glance 5. Coagulation Factor Deficiency Market Overview at a Glance 6. Coagulation Factor Deficiency Disease Background and Overview 7. Coagulation Factor Deficiency Epidemiology and Patient Population 8. Country-Specific Patient Population of Coagulation Factor Deficiency 9. Coagulation Factor Deficiency Current Treatment and Medical Practices 10. Coagulation Factor Deficiency Unmet Needs 11. Coagulation Factor Deficiency Emerging Therapies 12. Coagulation Factor Deficiency Market Outlook 13. Country-Wise Coagulation Factor Deficiency Market Analysis (2020–2034) 14. Coagulation Factor Deficiency Market Access and Reimbursement of Therapies 15. Coagulation Factor Deficiency Market Drivers 16. Coagulation Factor Deficiency Market Barriers 17. Coagulation Factor Deficiency Appendix 18. Coagulation Factor Deficiency Report Methodology 19. DelveInsight Capabilities 20. Disclaimer 21. About DelveInsight About DelveInsight DelveInsight is a leading Healthcare 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. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach. Media Contact Company Name: DelveInsight Contact Person: Jatin Vimal Email: Send Email Phone: +14699457679 Address: 304 S. Jones Blvd #2432 City: Las Vegas State: Nevada Country: United States Website:
Yahoo
18-04-2025
- Health
- Yahoo
New plasma donation center opens in Wichita
WICHITA, Kan. (KSNW) – A new plasma donation center opened in Wichita on Thursday. The company, Octapharma Plasma, has over 180 facilities across the nation. The new facility, which opened near Harry and Oliver, will feature updated technology. 'Octapharma Plasma has been at the forefront of providing lifesaving treatments derived from plasma, which are used to treat more than 300 rare, chronic, and life-threatening diseases, including hemophilia and primary immunodeficiency,' Sedgwick County Commissioner Jim Howell said. 'They are the world's leading plasma collection and human protein product manufacturer.' Local leaders say it will bring a $6 million boost to the local economy. 'Octapharma building is more than just a facility, they are strengthening the lifeline for patients and a legacy of care for this community,' Howell said. Thursday also happened to be World Hemophilia Day, and World Primary Immunodeficiency Week is just around the corner. It is recognized from April 22 to 25. Copyright 2025 Nexstar Media, Inc. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed.


Globe and Mail
28-01-2025
- Business
- Globe and Mail
Hemophilia A FDA Approvals, Clinical Trials, Pipeline Insights, Drugs and Companies
DelveInsight's, 'Hemophilia A Pipeline Insight' report provides comprehensive insights about 40+ companies and 40+ pipeline drugs in Hemophilia A pipeline landscape. It covers the Hemophilia A pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Hemophilia A therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space. Explore the comprehensive insights by DelveInsight and stay ahead in understanding the Hemophilia A Treatment Landscape. Click here to read more @ Hemophilia A Pipeline Outlook Key Takeaways from the Hemophilia A Pipeline Report In January 2025:- CSL Behring:- A Phase 3, Open-label, Multicenter, Pharmacokinetics, Efficacy, and Safety Study of a Recombinant Single-chain Factor VIII (rVIII-SingleChain) in Chinese Previously Treated Patients (PTPs) With Hemophilia A. For bridging the available global clinical data of rVIII-SingleChain, with the Chinese population, the aim of this study in China is to investigate the pharmacokinetics (PK) of rVIII-SingleChain after an initial and repeat dose and to assess efficacy and safety during 2 to 3 times weekly prophylaxis treatment with rVIII-SingleChain in male Chinese PTPs with severe hemophilia A (FVIII activity less than [<] 1%). In January 2025:- Octapharma:-Female patients with mild to moderate haemophilia A will often need FVIII concentrates to provide haemostatic cover during major surgery. This prospective, open-label, non-controlled, single-arm, multinational, multicentre study aims to evaluate the overall perioperative haemostatic efficacy of Nuwiq in women/girls over 12 with haemophilia A undergoing major surgery requiring FVIII treatment. In January 2025:- Bayer:- A Phase 1/2 Open-label Safety and Dose-finding Study of BAY2599023 (DTX201), an Adeno-associated Virus (AAV) hu37-mediated Gene Transfer of B-domain Deleted Human Factor VIII, in Adults With Severe Hemophilia A. By replacing the defective gene with a healthy copy the human body may produce clotting factor on its own. Hemophilia A is a bleeding disorder in which the human body does not have enough clotting factor VIII, a protein that controls bleeding. Researcher want to find the optimal dose of BAY 2599023 (DTX201) so that the body may produce enough clotting factor on its own. DelveInsight's Hemophilia A pipeline report depicts a robust space with 40+ active players working to develop 40+ pipeline therapies for Hemophilia A treatment. The leading Hemophilia A Companies such as Hoffmann-La Roche, Chugai Pharmaceutical, Shire, Pfizer, BioMarin Pharmaceutical, Sinocelltech Ltd., Bayer, Ultragenix Pharmaceutical, Spark Therapeutics, Octapharma, ApcinteX Ltd., Chia Tai Tianqing Pharmaceutical Group Co., Ltd., Expression Therapeutics LLC, CSL Behring, and others. Promising Hemophilia A Pipeline Therapies such as Emicizumab, rFVIIa, aPCC, BAX 888, OBIZUR, BIIB031 (rFVIIIFc), Recombinant Human Coagulation FVIII, and others. Discover groundbreaking developments in Hemophilia A therapies! Gain in-depth knowledge of key Hemophilia A clinical trials, emerging drugs, and market opportunities @ Hemophilia A Clinical Trials Assessment Hemophilia A Emerging Drugs Profile AGN-193408: Allergan Allergan is conducting a multicenter, open-label, dose escalation (Cohort 1) to masked, randomized, parallel-group (Cohort 2) study to evaluate the safety and efficacy of AGN-193408 SR in participants with open-angle glaucoma or ocular hypertension. It is an implant containing preservative-free AGN-193408 dispersed in a biodegradable polymer matrix. OCTA101: Octapharma Octapharma is evaluating OCTA101 (a human-cl rhFVIII and recombinant human von Willebrand Factor fragment dimer) under a Phase 1/2 study, which will be a dose escalation study in adults in 5 cohorts, with the main purpose to assess the safety of subcutaneous injection of OCTA101 (in previously treated adult patients with severe hemophilia A. The study also aims to assess the pharmacokinetics (PK) characteristics, dose proportionality, and subcutaneous bioavailability of OCTA101 compared with intravenous administration of Nuwiq (Human-cl rh FVIII), in order to define the prophylactic treatment (dose and injection interval) that would result in protective trough levels of FVIII:C for future Phase 3 studies. By Data Monitoring Committee recommendation, patients enrolled in cohorts 1, 2 and 3 will proceed to 3-month prophylactic treatment to receive daily dosing of OCTA101 for 3 months. Valoctocogene roxaparvovec: BioMarin Pharmaceuticals Valoctocogene roxaparvovec, is an investigational gene therapy in clinical trials for the treatment of Hemophilia A and has not been determined to be safe or effective. Valoctocogene roxaparvovec is administered as a single infusion. The ongoing clinical trials will determine if the new gene will enable the body to produce factor VIII. Following infusion, clinical trial subjects are being evaluated to determine the safety profile, changes in Factor VIII activity levels, changes in factor replacement usage, changes in reported bleeds requiring factor replacement, and quality of life measures, among other endpoints. Hemophilia A Companies Hoffmann-La Roche, Chugai Pharmaceutical, Shire, Pfizer, BioMarin Pharmaceutical, Sinocelltech Ltd., Bayer, Ultragenix Pharmaceutical, Spark Therapeutics, Octapharma, ApcinteX Ltd., Chia Tai Tianqing Pharmaceutical Group Co., Ltd., Expression Therapeutics LLC, CSL Behring, and others. Hemophilia A pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as Oral Intravenous Subcutaneous Molecule Type Hemophilia A Products have been categorized under various Molecule types such as Small molecules Gene Therapies Bispecific antibodies Recombinant proteins Fusion Proteins Coagulants Blood coagulation factor replacements Transform your understanding of the Hemophilia A Pipeline! See the latest progress in drug development and clinical research @ Hemophilia A Market Drivers and Barriers, and Future Perspectives Scope of the Hemophilia A Pipeline Report Coverage- Global Hemophilia A Companies- Hoffmann-La Roche, Chugai Pharmaceutical, Shire, Pfizer, BioMarin Pharmaceutical, Sinocelltech Ltd., Bayer, Ultragenix pharmaceutical, Spark Therapeutics, Octapharma, ApcinteX Ltd., Chia Tai Tianqing Pharmaceutical Group Co., Ltd., Expression Therapeutics LLC, CSL Behring, and others. Hemophilia A Pipeline Therapies- Emicizumab, rFVIIa, aPCC, BAX 888, OBIZUR, BIIB031 (rFVIIIFc), Recombinant Human Coagulation FVIII, and others. Hemophilia A Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination Hemophilia A Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Stay Ahead in Genetic Disorders Research–Access the Full Hemophilia A Pipeline Analysis Today! @ Hemophilia A Drugs and Companies Table of Content 1. Introduction 2. Executive Summary 3. Hemophilia A: Overview 4. Pipeline Therapeutics 5. Therapeutic Assessment 6. Hemophilia A - DelveInsight's Analytical Perspective 7. In-depth Commercial Assessment 8. Hemophilia A Collaboration Deals 9. Late Stage Products (Pre-registration) 10. Valoctocogene roxaparvovec: BioMarin Pharmaceutical 11. Late Stage Products (Phase III) 12. Giroctocogene fitelparvovec: Pfizer/ Sangamo Therapeutics 13. Mid Stage Products (Phase II) 14. NNC0365-3769 A (Mim8): Novo Nordisk A/S 15. Early Stage Products (Phase I/II) 16. BAX 888: Baxalta / Shire 17. BAY2599023: Bayer / Ultragenix pharmaceutical 18. Early Stage Products (Phase I) 19. Gene therapy: Expression Therapeutics, LLC 20. Drug profiles in the detailed report….. 21. Hemophilia A- Market Drivers and Barriers 22. Appendix About Us DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve. Media Contact Company Name: DelveInsight Business Research LLP Contact Person: Yash Bhardwaj Email: Send Email Phone: 9650213330 Address: 304 S. 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