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Home > Pharmaceuticals >  Hematology >  Hemophilia A and B – Global Drug Forecast and Market Analysis, 2021-2030

Hemophilia A and B – Global Drug Forecast and Market Analysis, 2021-2030

Hemophilia A and B are rare disorders in coagulation, characterized by the failure of blood to form normal clots after damage to veins and tissue. The patient population is increasing as a result of longer life expectancies and an aging population. Hemophilia A and B is mostly treated with prophylactic recombinant replacement factor therapy. The current market leader is Roche’s Hemlibra however, BioMarin’s gene therapy Roctavian is expected to be the market leader at the end of the forecast period.

The global market is expected to experience growth during the forecast period. Eleven agents are forecast to launch in the forecast period in the 8MM; four gene therapies, three monoclonal antibodies, two replacement factors, one enzyme and one antisense oligonucleotide. Growth will be driven by the launch of gene therapies, continuing preference for prophylactic regiments, and an increasing life expectancy for hemophiliacs. The major barriers for growth in the hemophilia A and B market include the possible market access issues for gene therapies due to high upfront costs and declining prevalent cases in certain countries.

KEY QUESTIONS ANSWERED

11 late-stage pipeline agents are expected to enter the Hemophilia A and B market from 2021 onwards. What impact will these agents have on the market? Which of these drugs will have the highest peak sales, and why?

What are the current unmet needs in Hemophilia A and B, which pipeline agents are positioned to counter these unmet needs? What are the opportunities for R&D?

What is the market outlook in the 8MM from 2020-2030? Considering major patent expiries, launch of new premium priced agents and expected label expansions.

What are the main corporate trends? Who are the current and future players?

Scope

Overview of Hemophilia A and B including epidemiology, pathophysiology, symptoms, diagnosis, and treatment guidelines.

Topline Hemophilia A and B market revenue, annual cost of therapy, and major pipeline product sales in the forecast period.

Key topics covered include current treatment and pipeline therapies, unmet needs and opportunities, and the drivers and barriers affecting Hemophilia A and B therapeutics sales in the 8MM.

Pipeline analysis: Comprehensive data split across different phases, emerging novel trends under development, and detailed analysis of late-stage pipeline drugs (Phase II – III).

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

Key Highlights

Growth will be driven by the launch of gene therapies, continuing preference for prophylactic regiments, and an increasing life expectancy for hemophiliacs

The major barriers for growth in the hemophilia A and B market include the possible market access issues for gene therapies due to high upfront costs and declining prevalent cases in certain countries

BioMarin’s Roctavian is expected to leapfrog Hemlibra’s sales to become the market leader by the end of the forecast period

The most important unmet needs in the Hemophilia A and B market include: the development of more curative treatments, more therapies for hemophilia B patients with inhibitors, decreasing the costs of prophylactic treatment, and introducing more therapies with convenient routes of administration

Reasons to Buy

The report will enable you to:

Develop and design your in-licensing and out-licensing strategies, using a detailed overview of current pipeline products and technologies to identify companies with the most robust pipelines.

Develop business strategies by understanding the trends shaping and driving the global Hemophilia A and B therapeutics market.

Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global Hemophilia A and B 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.

Track drug sales in the global Hemophilia A and B therapeutics market from 2020-2030.

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

Aptevo

Bayer

BioMarin

Catalyst Biosciences

CSL Behring

GC Pharma

Kaifeng Pharmaceutical

KM Biologics

LFB

Novo Nordisk

Pfizer

Octapharma

Roche

Sanofi

SinoCelltech

Spark Therapeutics

Takeda

UniQure

Table of Contents

| Contents

| List of Tables

| List of Figures

| About GlobalData

1 Hemophilia A and B: Executive Summary

1.1 The Hemophilia A and B Market Is Expected to Grow to $14.71B by 2030

1.2 Gene Therapies and Alternative Modulators of the Coagulation Cascade Remain the Main R&D Focus

1.3 Unmet Need for Innovative Agents in Different Patient Populations to be Partially Addressed but with Increasing Cost of Therapy

1.4 Gene Therapies to Dominate the Late-Stage Pipeline of Hemophilia A and B

1.5 What Do Physicians Think?

2 Introduction

2.1 Catalyst

2.2 Related Reports

2.3 Upcoming Reports

3 Disease Overview

3.1 Etiology and Pathophysiology

3.1.1 Etiology

3.1.2 Pathophysiology

4 Epidemiology

4.1 Disease Background

4.2 Risk Factors and Comorbidities

4.3 Global and Historical Trends

4.4 8MM Forecast Methodology

4.4.1 Sources

4.4.2 Sources Not Used

4.4.3 Forecast Assumptions and Methods

4.4.4 Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B

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

4.4.6 Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B by Inhibitor Status

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

4.4.8 Types of Treatment Among Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B

4.4.9 Diagnosed Prevalent Cases of Acquired Hemophilia

4.5 Epidemiological Forecast for Hemophilia A (2020–2030)

4.5.1 Diagnosed Prevalent Cases of Hemophilia A

4.5.2 Age-Specific Diagnosed Prevalent Cases of Hemophilia A

4.5.3 Sex-Specific Diagnosed Prevalent Cases of Hemophilia A

4.5.4 Diagnosed Prevalent Cases of Hemophilia A by Severity

4.5.5 Diagnosed Prevalent Cases of Hemophilia A with Inhibitors

4.6 Epidemiological Forecast for Hemophilia A and Hemophilia B (2020–2030)

4.6.1 Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B

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

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

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

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

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

4.6.7 Types of Treatment Among the Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B

4.6.8 Diagnosed Prevalent Cases of Acquired Hemophilia

4.7 Discussion

4.7.1 Epidemiological Forecast Insight

4.7.2 COVID-19 Impact

4.7.3 Limitations of the Analysis

4.7.4 Strengths of the Analysis

5 Disease Management

5.1 Diagnosis and Treatment Overview

5.1.1 Hemophilia A and B Without Inhibitors

5.1.2 Hemophilia A and B with Inhibitors

5.2 KOL Insights on Disease Management

5.2.1 Hemophilia A and B Without Inhibitors

5.2.2 Hemophilia A and B with Inhibitors

6 Current Treatment Options

6.1 Overview

7 Unmet Needs and Opportunity Assessment

7.1 Overview

7.2 Development of More Curative Treatments

7.3 Reduction in Risk of Inhibitor Development in Previously Untreated Patients

7.4 More Effective Treatments for Hemophilia B Patients with Inhibitors

7.5 Decreasing the Costs Associated with Prophylaxis

7.6 More Therapies with Convenient Administration Routes

8 R&D Strategies

8.1 Overview

8.1.1 Developing Replacement Factor Agents with Longer Half-Lives

8.1.2 Targeting Alternative Modulators of the Coagulation Cascade

8.1.3 Easier Routes of Administration

8.1.4 Sustained Focus on Developing Gene Therapies

8.2 Clinical Trials Design

8.2.1 Choosing a Patient Population

8.2.2 Primary and Surrogate Endpoints

8.2.3 Long-Term Monitoring

9 Pipeline Assessment

9.1 Overview

9.2 Promising Drugs in Clinical Development

10 Pipeline Valuation Analysis

10.1 Overview

10.2 Competitive Assessment

11 Current and Future Players

11.1 Overview

11.2 Deal-Making Trends

12 Market Outlook

12.1 Global Markets

12.1.1 Forecast

12.1.2 Drivers and Barriers – Global Issues

12.2 US

12.2.1 Forecast

12.2.2 Key Events

12.2.3 Drivers and Barriers

12.3 5EU

12.3.1 Forecast

12.3.2 Key Events

12.3.3 Drivers and Barriers

12.4 Japan

12.4.1 Forecast

12.4.2 Key Events

12.4.3 Drivers and Barriers

12.5 China

12.5.1 Forecast

12.5.2 Key Events

12.5.3 Drivers and Barriers

13 Appendix

13.1 Bibliography

13.2 Abbreviations

13.3 Methodology

13.3.1 Forecasting Methodology

13.4 Primary Research – KOLs Interviewed for This Report

13.4.1 KOLs

13.5 Primary Research – Prescriber Survey

13.6 About the Authors

13.6.1 Analyst

13.6.2 Therapy Area Director

13.6.3 Epidemiologist

13.6.4 Managing Epidemiologist

13.6.5 Global Director of Therapy Analysis and Epidemiology

13.6.6 Global Head and EVP of Healthcare Operations and Strategy

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List of Tables

Table 1: Hemophilia A and B: Key Metrics in the 8MM

Table 2: Severity Classifications of Hemophilia

Table 3: Relationship of Bleeding Severity with Clotting Factor Level

Table 4: Comorbidities for Hemophilia

Table 5: Treatment Guidelines for Hemophilia A and B

Table 6: Top 10 Deals by Value, 2017–2021

Table 7: Hemophilia A and B Market – Global Drivers and Barriers, 2020–2030

Table 8: Key Events Impacting Sales for Hemophilia A and B in the US, 2020–2030

Table 9: Hemophilia A and B Market – Drivers and Barriers in the US, 2020–2030

Table 10: Key Events Impacting Sales for Hemophilia A and B in the 5EU, 2020–2030

Table 11: Hemophilia A and B Market – Drivers and Barriers in the 5EU, 2020–2030

Table 12: Key Events Impacting Sales for Hemophilia A and B in Japan, 2020–2030

Table 13: Hemophilia A and B Market – Drivers and Barriers in Japan, 2020–2030

Table 14: Key Events Impacting Sales for Hemophilia A and B in China, 2020–2030

Table 15: Hemophilia A and B Market – Drivers and Barriers in China, 2020–2030

Table 16: High-Prescribing Physicians (non-KOLs) Surveyed, By Country

List of Figures

Figure 1: Global Sales Forecast by Country for Hemophilia A and B in 2020 and 2030

Figure 2: Analysis of the Company Portfolio Gap in Hemophilia A and B During the Forecast Period

Figure 3: Competitive Assessment of the Late-Stage Pipeline Agents that GlobalData Expects to Be Licensed for the Treatment of Hemophilia A and B During the Forecast Period

Figure 4: The Cascade of Events that Lead to the Formation of Blood Clots

Figure 5: Common Areas of Bleeding and Classes of Therapy

Figure 6: 8MM, Diagnosed Prevalence of Hemophilia A (%), Both Sexes, All Ages

Figure 7: 8MM, Diagnosed Prevalence of Hemophilia B (%), Both Sexes, All Ages

Figure 8: 8MM, Sources Used to Forecast the Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B

Figure 9: 8MM, Sources Used to Forecast the Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B, by Severity

Figure 10: 8MM, Sources Used to Forecast the Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B, by Inhibitors

Figure 11: 8MM, Sources Used to Forecast the Severity Among Inhibitors in Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B

Figure 12: 8MM, Sources Used to Forecast the Types of Treatment Among Hemophilia A and Hemophilia B Patients

Figure 13: 8MM, Sources Used to Forecast the Diagnosed Prevalent Cases of Acquired Hemophilia

Figure 14: 8MM, Diagnosed Prevalent Cases of Hemophilia A, N, Both Sexes, All Ages, 2020

Figure 15: 8MM, Diagnosed Prevalent Cases of Hemophilia A by Age, N, Both Sexes

Figure 16: 8MM, Diagnosed Prevalent Cases of Hemophilia A by Sex, N, All Ages, 2020

Figure 17: 8MM, Diagnosed Prevalent Cases of Hemophilia A by Severity, N, Both Sexes, All Ages, 2020

Figure 18: 8MM, Diagnosed Prevalent Cases of Hemophilia A with Inhibitors, N, Both Sexes, All Ages, 2020

Figure 19: 8MM, Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B, N, Both Sexes, All Ages, 2020

Figure 20: 8MM, Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B by Age, N, Both Sexes, 2020

Figure 21: 8MM, Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B by Sex, N, All Ages, 2020

Figure 22: 8MM, Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B by Severity, N, Both Sexes, All Ages, 2020

Figure 23: 8MM, Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B with Inhibitors, N, Both Sexes, All Ages, 2020

Figure 24: 8MM, Severity Among Inhibitors in Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B, Both Sexes, All Ages, N, 2020

Figure 25: 8MM, Types of Treatment Among the Diagnosed Prevalent Cases of Hemophilia A and Hemophilia B, Both Sexes, All Ages, N, 2020

Figure 26: 8MM, Diagnosed Prevalent Cases of Acquired Hemophilia, N, Both Sexes, All Ages, 2020

Figure 27: Treatment Algorithm for Hemophilia A and B

Figure 28: Unmet Needs and Opportunities in Hemophilia A and B

Figure 29: Overview of the Development Pipeline in Hemophilia A and B

Figure 30: Key Late-Stage Trials for the Promising Pipeline Agents that GlobalData Expects to Be Licensed for Hemophilia A and B in the 8MM During the Forecast Period

Figure 31: Competitive Assessment of the Marketed and Pipeline Drugs For Hemophilia A and B Benchmarked Against the SOC Class of Drugs, Recombinant Replacement Factor

Figure 32: Competitive Assessment of the Marketed and Pipeline Drugs For Hemophilia A and B Benchmarked Against the SOC Class of Drugs, Alternative Coagulation Promoter

Figure 33: Analysis of the Company Portfolio Gap in Hemophilia A and B During the Forecast Period

Figure 34: Global (8MM) Sales Forecast by Country for Hemophilia A and B in 2020 and 2030

Figure 35: Sales Forecast by Class for Hemophilia A and B in the US in 2020 and 2030

Figure 36: Sales Forecast by Class for Hemophilia A and B in the 5EU in 2020 and 2030

Figure 37: Sales Forecast by Class for Hemophilia A and B in Japan in 2020 and 2030

Figure 38: Sales Forecast by Class for Hemophilia A and B in China in 2020 and 2030

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