Hemophilia A and B: Could This Ease to Speed Relief?

A qualitative research study accomplished by HTF MI titled “Hemophilia A and B - Global Drug Forecast and Market covers detailed Product / Industry Scope, current and future market size scenario and elaborates outlook and status to 2025” provides primary data, studies and vendor briefings. The market Study is segmented by key regions along with country level break-up which is accelerating the marketization and by products type, application/end-users. The research study provides estimates for Hemophilia A and B - Drug Forecast and forecast till 2025. Some of the Major Players Included in the study are Alnylam Pharmaceuticals, Alveron Pharma, Aprilbio, Aptevo Therapeutics, Bakusuaruta, Baxalta, Baxter, Bayer, Beijing Tiantan Biological Products, Biogen, BioMarin, BioReliance, Biotest, Bioverativ, Cangene Corporation, Catalyst Biosciences, The Chemo-Sero-Therapeutic Research Institute, China Biologic Products, Chengdu Rongsheng Pharmaceutical, Chugai Pharmaceutical, CSL Behring, Emergent BioSolutions, EpiVax, F. Hoffmann-La Roche, Freeline Therapeutics, GC Pharma, Genentech, Genetics Institute, Grifols, Hualan Biological Engineering, Idogen, Immuno, Ioproducts Laboratory, Japan Blood Products Organization, Kaifeng Pharmaceutical, Kedrion, Kymab, Laboratoire Français de Fractionnement et de Biotechnologies (LFB), Novo Nordisk, Octapharma, Okklo Life Sciences, OPKO Biologics, Pfizer, Pharming Group, RegenxBio, Sanofi, Sangamo Therapeutics, Sanquin Pharmaceutical Services, Selecta Biosciences, Shanghai Baxter Healthcare, Shenzhen Geno-Immune Medical Institute, Shenzhen Weiwu Guangming Biological Preparations, Shire, SK Chemicals, Spark Therapeutics, St. Jude Children's Research Hospital, Swedish Orphan Biovitrum (SOBI), Takeda Pharmaceutical, Ultragenyx Pharmaceutical, UniQure, Wyeth.

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Summary

Hemophilia A and B are rare, genetic, X-linked deficiencies in the blood clotting factors VIII (FVIII) and IX (FIX), respectively. Patients can be categorized as mild, moderate or severe depending on the percentage of circulating FVIII or FIX in their blood. Patients can be treated either on-demand or prophylactically using plasma-derived or recombinant replacement factors, the current standard of care.
Although these therapies are effective, they are associated with a great treatment burden. There is a significant unmet need for new therapies that can reduce the great treatment burden associated with intravenous administration, the frequency of prophylactic infusions, and the risk of developing neutralizing antibodies, or inhibitors, against replacement factors. Overall, the late-stage pipeline indicates an increase in the development of alternative coagulation promoters (ACPs) and gene therapies to further target these areas of significant unmet need. During the forecast window, Roche entered the market with the first monoclonal antibody targeting hemophilia A patients both with and without inhibitors, which addresses the unmet needs of frequency and route of administration. Sanofi are also to exploring a non-replacement approach and will enter the market with the ribonucleic acid (RNA) therapeutic, Fitusiran (previously Alnylam's), further ensuring cross-segment efficacy in patients both with and without inhibitors. The approval of these new products has begun to have a drastic effect on the hemophilia A and B treatment landscapes which is expected to continue through the forecast window. During this forecast window, several pharmaceutical companies, including BioMarin, uniQure and Pfizer/Roche (Spark Therapeutics), will also be launching new gene therapies that aim to cure, or at least reduce the severity of, hemophilia A and B and diminish inhibitors. If approved, gene therapies will pose a threat to existing hemophilia therapies.

Key Questions Answered
- Which pipeline agents - replacement factors, alternative coagulation promoters and gene therapies - are the most promising and expected to launch in the 8MM
- What are the forecasted sales of these agents and what will be their impact in the hemophilia A and B market
- What are the main unmet needs in hemophilia A and B, and which pipeline drugs will fulfil these needs, and to what extent
- What are the current research and development (R&D) strategies being explored and how can developers incorporate these methods into their business strategy
- Key Opinions Leader (KOL) and payer insights across the 8MM, relating to the current treatment options and opinions on the late and early stage pipeline agents.

Scope

Scope
Overview of hemophilia A and B, including -
- Disease overview - etiology, pathophysiology, symptoms, prognosis and quality of life
- Epidemiology
- Disease management - diagnosis and treatment

Key topics covered include -
- Competitive assessment of marketed and pipeline products including SWOTs and, clinical and commercial positioning.
- Unmet needs and opportunities including current and future level of attainment and gap analysis
- Pipeline assessment - analysis of commercial and clinical attributes of promising drugs in clinical development and comprehensive analysis of promising approaches and emerging trends in early stage development
- Analysis of the current and future players for the global hemophilia A and B market, including strength of marketed and pipeline products and trends in corporate strategy, profiles of leading companies in the hemophilia market.
- Market outlook of the 8MM including forecast, key events and, market drivers and barriers.

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Reasons to buy

The report will enable you to -
- Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline.
- Develop business strategies by understanding the trends shaping and driving the global hemophilia A and B market.
- Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global hemophilia market in the future.
- Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various competitors.
- Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
- Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.

Companies Mentioned in the Report:
Alnylam Pharmaceuticals, Alveron Pharma, Aprilbio, Aptevo Therapeutics, Bakusuaruta, Baxalta, Baxter, Bayer, Beijing Tiantan Biological Products, Biogen, BioMarin, BioReliance, Biotest, Bioverativ, Cangene Corporation, Catalyst Biosciences, The Chemo-Sero-Therapeutic Research Institute, China Biologic Products, Chengdu Rongsheng Pharmaceutical, Chugai Pharmaceutical, CSL Behring, Emergent BioSolutions, EpiVax, F. Hoffmann-La Roche, Freeline Therapeutics, GC Pharma, Genentech, Genetics Institute, Grifols, Hualan Biological Engineering, Idogen, Immuno, Ioproducts Laboratory, Japan Blood Products Organization, Kaifeng Pharmaceutical, Kedrion, Kymab, Laboratoire Français de Fractionnement et de Biotechnologies (LFB), Novo Nordisk, Octapharma, Okklo Life Sciences, OPKO Biologics, Pfizer, Pharming Group, RegenxBio, Sanofi, Sangamo Therapeutics, Sanquin Pharmaceutical Services, Selecta Biosciences, Shanghai Baxter Healthcare, Shenzhen Geno-Immune Medical Institute, Shenzhen Weiwu Guangming Biological Preparations, Shire, SK Chemicals, Spark Therapeutics, St. Jude Children's Research Hospital, Swedish Orphan Biovitrum (SOBI), Takeda Pharmaceutical, Ultragenyx Pharmaceutical, UniQure, Wyeth.

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Table of Contents

1.1 List of Tables

1.2 List of Figures

2 Executive Summary

2.1 Hemophilia A and B Market Expected to Grow to $9.3B by 2028

2.2 Inhibitor Segment Post-ACP Launch

2.3 ACPs and Gene Therapies to Radically Impact Hemophilia A and B Inhibitor and Non-Inhibitor Segments

2.4 Uncertainty in the Safety, Durability, and Availability of Gene Therapies Renders Curative Hemophilia Treatments a Continuing Unmet Need

2.5 Newcomers to Take Over Market Space from Established Companies

2.6 What Do Physicians Think

3 Introduction

3.1 Catalyst

3.2 Related Reports

3.3 Upcoming Related Reports

4 Disease Overview

4.1 Etiology and Pathophysiology

4.1.1 Etiology

4.1.2 Pathophysiology

4.2 Classification

4.3 Symptoms

4.3.1 Hemophilia A and B

4.3.2 Inhibitors

4.4 Prognosis and Quality of Life

....Continued

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