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

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.

Scope

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.

Companies mentioned

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 ...

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

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 ...

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

List of Tables

Table 1: Hemophilia A and B: Key Metrics in the Seven Major Markets, 2018–2028

Table 2: Relationship of Bleeding Severity with Clotting Factor Level.

Table ...

Table 1: Hemophilia A and B: Key Metrics in the Seven Major Markets, 2018–2028

Table 2: Relationship of Bleeding Severity with Clotting Factor Level.

Table 3: Comorbidities for Hemophilia

Table 4: 8MM Treatment Guidelines for Hemophilia

Table 5: The Most-Prescribed Hemophilia A and B Branded Drugs in the Global Markets, 2018

Table 6: Country Profile — US

Table 7: Country Profile — 5EU

Table 8: Country Profile — Japan

Table 9: Country Profile — China

Table 10: Leading Replacement Therapies for Hemophilia A and B and Patients with Inhibitors, 2019

Table 11: Marketed Plasma-Derived Replacement Therapies for Hemophilia A Patients, 2019

Table 12: Marketed Plasma-Derived Replacement Therapies for Hemophilia B Patients, 2019

Table 13: TFPI inhibitors – Early-Stage Pipeline, 2019

Table 14: Hemophilia A and B Gene Therapies – Early-Stage Pipeline, 2019

Table 15: Hemophilia A and B Recombinant Replacement Therapies – Early-Stage Pipeline, 2019

Table 16: Bayer’s Hemophilia Portfolio Assessment, 2019

Table 17: CSL Behring’s Hemophilia Recombinant Portfolio Assessment, 2019

Table 18: Novo Nordisk’s Hemophilia Recombinant Portfolio Assessment, 2019

Table 19: Pfizer’s Hemophilia Recombinant Portfolio Assessment, 2019

Table 20: Roche’s Hemophilia Recombinant Portfolio Assessment, 2019

Table 21: Bioverativ/Sobi’s Hemophilia Portfolio Assessment, 2019

Table 22: Takeda’s Hemophilia Recombinant Portfolio Assessment, 2019

Table 23: Hemophilia A and B Market – Global Drivers and Barriers, 2018–2028

Table 24: Key Events Impacting Sales for Hemophilia A and B in the US, 2018–2028

Table 25: Hemophilia A and B Market – US Drivers and Barriers, 2018–2028

Table 26: Key Events Impacting Sales for Hemophilia A and B Therapeutics in the 5EU, 2018–2028

Table 27: Hemophilia A and B Market – 5EU Drivers and Barriers, 2018–2028

Table 28: Key Events Impacting Sales for Hemophilia A and B in Japan, 2018–2028

Table 29: Hemophilia A and B Market – Japan Drivers and Barriers, 2018–2028

Table 30: Key Events Impacting Sales for Hemophilia A and B in China, 2018–2028

Table 31: Hemophilia A and B Market – China Drivers and Barriers, 2018–2028

Table 32: Key Historical and Projected Launch Dates for Hemophilia A and B

Table 33: Key Historical and Projected Patent Expiry Dates for Hemophilia A and B

Table 34: Average Body Weight Across the 8MM

Table 35: Physicians Surveyed

List of Figures

Figure 1: Global Sales Forecast by Country for Hemophilia A and B, 2018–2028

Figure 2: Competitive Assessment of Late-Stage Pipeline Agents for Hemophilia A and ...

Figure 1: Global Sales Forecast by Country for Hemophilia A and B, 2018–2028

Figure 2: Competitive Assessment of Late-Stage Pipeline Agents for Hemophilia A and B, 2018–2028

Figure 3: Key Company Portfolio Positioning in Hemophilia A and B, 2018–2028

Figure 4: 8MM, Diagnosed Prevalence of Hemophilia A (%), All Ages, 2018

Figure 5: 8MM, Diagnosed Prevalence of Hemophilia B (%), All Ages, 2018

Figure 6: 8MM, Sources Used, Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B

Figure 7: 8MM, Sources Used, Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B, by Severity

Figure 8: 8MM, Sources Used, Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B with Inhibitors

Figure 9: 8MM, Sources Used, Severity Among Inhibitors in Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B

Figure 10: 8MM, Sources Used, Type of Treatment Among Hemophilia A and Hemophilia B Patients

Figure 11: 8MM, Sources Used, Diagnosed Prevalent Cases of Acquired Hemophilia

Figure 12: 8MM, Diagnosed Prevalent Cases of Hemophilia A, All Ages, Both Sexes, N, 2018

Figure 13: 8MM, Age-Specific Diagnosed Prevalent Cases of Hemophilia A, Both Sexes, N, 2018

Figure 14: 8MM, Sex-Specific Diagnosed Prevalent Cases of Hemophilia A, All Ages, N, 2018

Figure 15: 8MM, Diagnosed Prevalent Cases of Hemophilia A by Severity, Both Sexes, All Ages, N, 2018

Figure 16: 8MM, Diagnosed Prevalent Cases of Hemophilia A with Inhibitors, Both Sexes, All Ages, N, 2018

Figure 17: 8MM, Diagnosed Prevalent Cases of Hemophilia B, All Ages, Both Sexes, 2018

Figure 18: 8MM, Age-Specific Diagnosed Prevalent Cases of Hemophilia B, Both Sexes, 2018

Figure 19: 8MM, Sex-Specific Diagnosed Prevalent Cases of Hemophilia B, All Ages, N, 2018

Figure 20: 8MM, Diagnosed Prevalent Cases of Hemophilia B by Severity, Both Sexes, All Ages, N, 2018

Figure 21: 8MM, Diagnosed Prevalent Cases of Hemophilia B with Inhibitors, Both Sexes, All Ages, N, 2018

Figure 22: 8MM, Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B, Both Sexes, N, 2018

Figure 23: 8MM, Age-Specific Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B, Both Sexes, N, 2018

Figure 24: 8MM, Sex-Specific Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B, All Ages, N, 2018

Figure 25: 8MM, Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B by Severity, Both Sexes, All Ages, N, 2018

Figure 26: 8MM, Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B with Inhibitors, Both Sexes, All Ages, N, 2018

Figure 27: 8MM, Severity Among Inhibitors in Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B, Both Sexes, All Ages, N, 2018

Figure 28: 8MM, Type of Treatment Among the Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B, Both Sexes, All Ages, N, 2018

Figure 29: 8MM, Diagnosed Prevalent Cases of Acquired Hemophilia, All Ages, Both Sexes, N, 2018

Figure 30 : Flowchart of the Diagnosis and Management of Hemophilia Patients, With and Without Inhibitors

Figure 31: Unmet Needs and Opportunities in Hemophilia A and B

Figure 32: Competitive Assessment of Late-Stage Pipeline Agents, 2018–2028

Figure 33: Overview of the Development Pipeline in Hemophilia, 2018–2028

Figure 34: Hemophilia A and B – Phase III Pipeline, 2019

Figure 35: Company Portfolio Analysis in Hemophilia A and B, 2018–2028

Figure 36: Global Sales for Hemophilia A and B by Drug Class, 2018–2028

Figure 37: Global Sales Forecast by Country for Hemophilia A and B, 2018–2028

Figure 38: Global Sales Forecast by Patient Segment, 2018–2028

Figure 39: Sales for Hemophilia A and B Therapeutics in the US by Drug Class, 2018–2028

Figure 40: Sales for Hemophilia A and B Therapeutics in the 5EU by Drug Class, 2018–2028

Figure 41: Sales for Hemophilia A and B Therapeutics in Japan by Drug Class, 2018–2028

Figure 42: Sales for Hemophilia A and B Therapeutics in China by Drug Class, 2018–2028

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