Hemophilia A and B – Global Drug Forecast and Market Analysis to 2028

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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.
GlobalData projects the global hemophilia A and B marketplace to experience a significant growth during the forecast period. GlobalData valued the hemophilia A and B market at $6.9B in 2018 in the 8MM and expects the market to increase to $9.3B in 2028 at a Compound Annual Growth Rate (CAGR) of 2.9%. This growth will be driven primarily by the increasing prophylactic treatment rates among patients, the continued use of recombinant products and the uptake of non-replacement therapies in the 8MM. However, cost constraints in the 8MM are expected to limit the premium pricing opportunities for new products such as late-to-market long-acting factor concentrates and new alternative coagulation promoters, thereby limiting the size of the hemophilia market.
This model covers the market forecast for marketed and late-stage pipeline hemophilia A and B therapeutics. The base year of this model is 2018, and the forecast period is 2018–2028.
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.


Overview of hemophilia A and B, including:

• Disease overview – etiology, pathophysiology, symptoms, prognosis and quality of life

• Epidemiology

• Disease management – diagnosis and treatment

Patient segmentation:

• Mild, Moderate, and Severe

• Hemophilia A and B with and without inhibitors

• Males and Females

• All ages, Pediatrics and Adults

Annualized hemophilia A and B therapeutics market revenue and treatment usage patterns:

• Patient shares and number of patients treated for marketed and pipeline products

• Duration of and, daily and annual cost of therapies per patient

• Product sales by usage and total sales of marketed and pipeline products

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.

Key Highlights

The hemophilia A & B market is expected to grow at a CAGR of 2.9% until 2028, reaching a global value of $9.3B.

GlobalData forecasts a marginal increase in prevalence rates across 8MM with AGR of 0.1% due to increased in prevalence rates across the US, Spain and UK.

Gene therapies will emerge as novel treatments for hemophilia A & B during the forecast period. Particularly, BioMarin’s first-to-market gene therapy, ValRox is likely to perform exceptionally well. Additionally, Roche’s Hemlibra is anticipated to achieve blockbuster status by the end of the forecast period.

Rising life expectancy for hemophilia patients leads to an increase in the size of the treated population.

Expected launches of expensive new drugs such as gene therapies, continued preference for prophylactic regimens, continued patient evolution from plasma-based to recombinant therapies are other significant drivers of market.

Limited scope for price premiums of late-to-market replacement products, high uptake of lower-priced drugs, rise in cost-consciousness and imposing market access barriers for the entry of expensive gene therapies are expected to affect market growth negatively.

Level of unmet needs in hemophilia patients is high but is expected to decrease due to advances in treatments and the introduction of novel therapies.

There are major market opportunities for the development of novel therapies, cheaper therapies for prophylaxis treatment, more effective treatments for hemophilia B patients with inhibitors and more therapies with convenient administration routes.

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.

Alnylam Pharmaceuticals
Alveron Pharma
Aptevo Therapeutics
Beijing Tiantan Biological Products
Cangene Corporation
Catalyst Biosciences
The Chemo-Sero-Therapeutic Research Institute
China Biologic Products
Chengdu Rongsheng Pharmaceutical
Chugai Pharmaceutical
CSL Behring
Emergent BioSolutions
F. Hoffmann-La Roche
Freeline Therapeutics
GC Pharma
Genetics Institute
Hualan Biological Engineering
Ioproducts Laboratory
Japan Blood Products Organization
Kaifeng Pharmaceutical
Laboratoire Français de Fractionnement et de Biotechnologies (LFB)
Novo Nordisk
Okklo Life Sciences
OPKO Biologics
Pharming Group
Sangamo Therapeutics
Sanquin Pharmaceutical Services
Selecta Biosciences
Shanghai Baxter Healthcare
Shenzhen Geno-Immune Medical Institute
Shenzhen Weiwu Guangming Biological Preparations
SK Chemicals
Spark Therapeutics
St. Jude Children's Research Hospital
Swedish Orphan Biovitrum (SOBI)
Takeda Pharmaceutical
Ultragenyx Pharmaceutical

Table of Contents

1 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

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 of Hemophilia A and Hemophilia B

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 Inhibitor Status

5.4.6 Severity Among Inhibitors in Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B

5.4.7 Type of Treatment Among Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B

5.4.8 Diagnosed Prevalent Cases of Acquired Hemophilia

5.5 Epidemiological Forecast for Hemophilia A (2018–2028)

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 (2018–2028)

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 (2018–2028)

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

5.7.6 Severity Among Inhibitors in Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B

5.7.7 Type of Treatment Among the Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B

5.7.8 Diagnosed Prevalent Cases of Acquired Hemophilia

5.8 Discussion

5.8.1 Epidemiological Forecast Insight

5.8.2 Limitations of Analysis

5.8.3 Strengths of Analysis

6 Disease Management

6.1 Diagnosis and Treatment Overview

6.1.1 Treatment of Hemophilia A and B Without Inhibitors

6.1.2 Treatment of Hemophilia A and B Patients with Inhibitors

6.2 US

6.3 5EU

6.4 Japan

6.5 China

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 Alternative Coagulation Promoters

7.2.4 Plasma-Derived FVIII Concentrates

7.2.5 Desmopressin Acetate

7.3 Product Profiles – Hemophilia B

7.3.1 Short-Acting rFIX

7.3.2 Long-Acting rFIX

7.3.3 Plasma-Derived FIX Concentrates

7.4 Product Profiles – Hemophilia A and B with Inhibitors

8 Unmet Needs and Opportunities

8.1 Overview

8.2 Reduction in Risk of Inhibitor Development in Previously Untreated Patients

8.3 More Effective Treatments for Hemophilia B Patients with Inhibitors

8.4 More Therapies with Convenient Administration Routes

8.5 Treatments to Cure the Disease

8.6 Decreasing the Costs Associated with Prophylaxis and Promising Treatments

8.7 Individualized Prophylaxis

9 Pipeline Assessment

9.1 Overview

9.2 Promising Drugs in Clinical Development

9.3 Promising Approaches in Early-Stage Development

9.3.1 Alternative Coagulation Promoters – Tissue Factor Pathway Inhibitors

9.3.2 Gene Therapies

9.3.3 Replacement Strategies

9.3.4 Strategies to Prevent and Manage Inhibitors

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 CSL Behring

10.3.3 Novo Nordisk

10.3.4 Pfizer

10.3.5 Roche

10.3.6 Sanofi

10.3.7 Takeda

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

11.4 Japan

11.4.1 Forecast

11.4.2 Key Events

11.4.3 Drivers and Barriers

11.5 China

11.5.1 Forecast

11.5.2 Key Events

11.5.3 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 – Payers Interviewed for this Report

12.7 Primary Research – Prescriber Survey

12.8 Real-World Data

12.9 About the Authors

12.9.1 Analyst

12.9.2 Therapy Area Director

12.9.3 Epidemiologists

12.9.4 Reviewers

12.9.5 Global Director of Therapy Analysis and Epidemiology

12.9.6 Global Head and EVP of Healthcare Operations and Strategy

12.10 About GlobalData

12.11 Contact Us

12.12 Disclaimer

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