PharmaPoint: Hemophilia A and B – Global Drug Forecast and Market Analysis to 2026

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Hemophilia A and B are rare recessive X-linked genetic deficiencies in the blood clotting factors VIII and IX, respectively, and are characterized by the failure of blood to form normal clots after damage to veins and tissue. In the 7MM, patients with severe forms of the disease are increasingly treated on a prophylactic basis rather than on demand after bleeds. The frequent prophylactic infusions of rFVIII or rFIX begin from the first one or two years of life, often continuing through adulthood. However, the burden on patients and their families of maintaining the prophylactic treatment schedule is high.
Traditionally, the hemophilia market has been dominated by short-acting rFVIII or FIX concentrates. However, in recent years the hemophilia market has become increasingly competitive due to the approval of extended half-life products that can reduce the burden associated with prophylaxis. These improvements have mainly benefited hemophilia B patients, while a substantial unmet need remains for hemophilia A patients and patients with inhibitors who do not respond to standard treatments.
The hemophilia pipeline activity is strong and includes two candidates, Roche’s emicizumab (ACE-910) and Alnylam’s fitusiran, which do not exploit a replacement strategy but target different effectors of the coagulation cascade and are expected to cause a paradigm shift in the treatment of the disease.
Key Questions Answered
What are the key drivers behind the increasing uptake and market penetration of the long-acting factor concentrates in hemophilia A and B, since their approval in 2014, and what is the trend in the use of long-acting factors in the forecast period?
The hemophilia market is characterized by a number of unmet needs. What are the main unmet needs in this market? Will the pipeline drugs under development fulfil these unmet needs?
What impact will the approval of Roche’s emicizumab have on the hemophilia market? What will the drug’s peak sales be, and why?


Overview of hemophilia, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and disease management.

Annualized hemophilia therapeutics market revenue, cost of therapy per patient, and treatment usage patterns in nine patient segments and two treatment strategies (episodic and prophylactic treatment), forecast from 2016 to 2026.

Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping, and implications for the hemophilia therapeutics market.

Pipeline analysis: comprehensive data assessing emerging trends and mechanisms of action under development for hemophilia. The most promising candidate in Phase III development is profiled.

Analysis of the current and future market competition in the global hemophilia market. Insightful review of the key industry drivers, restraints and, challenges. Each trend is independently researched to provide qualitative analysis of its implications.

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 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 analysing 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.

Aptevo Therapeutics
Catalyst Biosciences
CSL Behring
Dimension Therapeutics
Novo Nordisk
Opko Biologics
Revo Biologics
Sangamo Therapeutics
Spark Therapeutics
Xenetic Biosciences

Table of Contents

Table of Contents

1 Table of Contents

1.1 List of Tables

1.2 List of Figures

2 Executive Summary

2.1 Sales for Hemophilia A and B Recombinant Therapies by Region, 2016–2026

2.2 Long-Acting rFVIIIs Have Not Lived Up to Expectations

2.3 Inhibitor Segment to Devalue Post-Alternative Coagulation Promoter Launch

2.4 Roche’s Emicizumab to Radically Impact Hemophilia A and Inhibitor Segments

2.5 Treatment Cost to Remain a Major Unmet Need

2.6 New Comers to Take Over Market Space From Established Companies

2.7 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

5 Epidemiology

5.1 Disease Background

5.2 Risk Factors and Comorbidities

5.3 Global and Historical Trends

5.4 Forecast Methodology

5.4.1 Sources

5.4.2 Forecast Assumptions and Methods

5.4.3 Diagnosed Prevalent Cases

5.4.4 Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B by Severity

5.4.5 Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B by Inhibitors

5.5 Epidemiological Forecast for Hemophilia A (2016–2026)

5.5.1 Diagnosed Prevalent Cases of Hemophilia A

5.5.2 Age-Specific Diagnosed Prevalent Cases of Hemophilia A

5.5.3 Sex-Specific Diagnosed Prevalent Cases of Hemophilia A

5.5.4 Diagnosed Prevalent Cases of Hemophilia A by Severity

5.5.5 Diagnosed Prevalent Cases of Hemophilia A with Inhibitors

5.6 Epidemiological Forecast for Hemophilia B (2016–2026)

5.6.1 Diagnosed Prevalent Cases of Hemophilia B

5.6.2 Age-Specific Diagnosed Prevalent Cases of Hemophilia B

5.6.3 Sex -Specific Diagnosed Prevalent Cases of Hemophilia B

5.6.4 Diagnosed Prevalent Cases of Hemophilia B by Severity

5.6.5 Diagnosed Prevalent Cases of Hemophilia B with Inhibitors

5.7 Epidemiological Forecast for Hemophilia A and Hemophilia B (2016–2026)

5.7.1 Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B

5.7.2 Age-Specific Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B

5.7.3 Sex -Specific Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B

5.7.4 Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B by Severity

5.7.5 Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B with Inhibitors

5.8 Discussion

5.8.1 Epidemiological Forecast Insight

5.8.2 Limitations of the Analysis

5.8.3 Strengths of the Analysis

6 Disease Management

6.1 Treatment Overview

6.1.1 Treatment Guidelines

6.1.2 Clinical Practice

6.2 US

6.2.1 Clinical Practice

6.3 France

6.3.1 Clinical Practice

6.4 Germany

6.4.1 Clinical Practice

6.5 Italy

6.5.1 Clinical Practice

6.6 Spain

6.6.1 Clinical Practice

6.7 UK

6.7.1 Clinical Practice

6.8 Japan

6.8.1 Clinical Practice

7 Competitive Assessment

7.1 Overview

7.2 Product Profiles – Hemophilia A

7.2.1 Short Acting rFVIII

7.2.2 Long Acting rFVIII

7.2.3 Plasma-Derived FVIII Concentrates

7.2.4 Desmopressin Acetate

7.3 Product Profiles – Hemophilia B

7.3.1 Short Acting rFIX

7.3.2 Long Acting FIX

7.3.3 Plasma-Derived FIX Concentrates

7.4 Product Profiles – Hemophilia A and B with Inhibitors

7.4.1 NovoSeven RT (Eptacog Alfa)

7.4.2 Feiba NF (Activated Prothrombin Complex Concentrate)

8 Unmet Needs and Opportunities

8.1 Overview

8.2 Reduction in Risk of Inhibitor Development in Previously Untreated Patients

8.2.1 Unmet Need

8.2.2 Gap Analysis

8.2.3 Opportunity

8.3 More Effective Treatments for Patients with Inhibitors

8.3.1 Unmet Need

8.3.2 Gap Analysis

8.3.3 Opportunity

8.4 Longer-Lasting Agents and More Convenient Administration Routes

8.4.1 Unmet Need

8.4.2 Gap Analysis

8.4.3 Opportunity

8.5 Treatments to Cure the Disease

8.5.1 Unmet Need

8.5.2 Gap Analysis

8.5.3 Opportunity

8.6 Decreasing the Costs Associated with Prophylaxis

8.6.1 Unmet Need

8.6.2 Gap Analysis

8.6.3 Opportunity

8.7 Individualized Prophylaxis

8.7.1 Unmet Need

8.7.2 Gap Analysis

8.7.3 Opportunity

9 Pipeline Assessment

9.1 Overview

9.2 Late-Stage Development Candidates – Hemophilia A

9.2.1 BAY 94-9027 (Damoctocog Alfa Pegol)

9.2.2 N8-GP (Turoctocog Alfa Pegol)

9.2.3 Emicizumab (ACE-910, RG-6013)

9.2.4 Fitusiran (ALN-AT3, SAR-439774)

9.3 Late-Stage Development Candidates – Hemophilia B

9.4 Late-Stage Development Candidates – Hemophilia A and B Patients with Inhibitors

9.4.1 LR-769 (rhFVIIa)

9.4.2 Obizur (Susoctocog Alfa)

9.4.3 CSL-689 (rVIIa-FP)

9.4.4 BAX-817 (rFVIIa BI)

9.5 Promising Approaches in Early-Stage Development

9.5.1 Alternative Coagulation Promoters – Tissue Factor Pathway Inhibitors

9.5.2 Gene Therapies

9.5.3 Replacement Strategies

9.5.4 Strategies to Prevent and Manage Inhibitors

9.5.5 Other Drugs in Development

10 Current and Future Players

10.1 Overview

10.2 Trends in Corporate Strategy

10.3 Company Profiles

10.3.1 Bayer

10.3.2 Bioverativ

10.3.3 CSL Behring

10.3.4 Novo Nordisk

10.3.5 Pfizer

10.3.6 Roche

10.3.7 Shire

11 Market Outlook

11.1 Global Markets

11.1.1 Forecast

11.1.2 Drivers and Barriers – Global Market

11.2 US

11.2.1 Forecast

11.2.2 Key Events

11.2.3 Drivers and Barriers

11.3 5EU

11.3.1 Forecast

11.3.2 Key Events

11.3.3 Drivers and Barriers – 5EU

11.3.4 Drivers and Barriers – France

11.3.5 Drivers and Barriers – Germany

11.3.6 Drivers and Barriers – Italy

11.3.7 Drivers and Barriers – Spain

11.3.8 Drivers and Barriers – UK

11.4 Japan

11.4.1 Forecast

11.4.2 Key Events

11.4.3 Drivers and Barriers

12 Appendix

12.1 Bibliography

12.2 Abbreviations

12.3 Methodology

12.4 Forecasting Methodology

12.4.1 Diagnosed Hemophilia Patients

12.4.2 Percent Drug-Treated Patients

12.4.3 Drugs Included in Each Therapeutic Class

12.4.4 Launch and Patent Expiry Dates

12.4.5 General Pricing Assumptions

12.4.6 Individual Drug Assumptions

12.4.7 Pricing of Pipeline Agents

12.5 Primary Research – KOLs Interviewed for this Report

12.6 Primary Research – Prescriber Survey

12.7 Real-World Data

12.8 About the Authors

12.8.1 Analyst

12.8.2 Reviewer

12.8.3 Therapy Area Director

12.8.4 Epidemiologists

12.8.5 Reviewers

12.8.6 Global Director of Therapy Analysis and Epidemiology

12.8.7 Global Head and EVP of Healthcare Operations and Strategy

12.9 About GlobalData

12.10 Contact Us

12.11 Disclaimer

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