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.
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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.
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