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

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The hemophilia A & B drug market in 8MM was valued at $9.36 billion in 2020 and is expected to grow with a CAGR of more than 4% by 2030. The key driver of growth in the hemophilia A and B markets includes rising life expectancy for hemophilia patients due to reduced morbidity and mortality associated with severe complications and reduced risk of pathogen transmission from plasma-derived sources, increasing the treated population.

The US represents the largest market in hemophilia, accounting for more than 50% of total product sales in 2020, followed by the five major European markets (5EU: France, Germany, Italy, Spain, and the UK). Recombinant factor concentrates were the leading class of drug in the eight major market (8MM) countries (the US, China, Japan, and 5EU (France, Germany, Italy, Spain, and the UK)) in 2020. Gene therapies are expected to have the highest sales in 8MM by 2030. Some of the key companies in the hemophilia A & B drug market include Roche, Novo Nordisk, Pfizer, and Sanofi.

Hemophilia A & B drug market overview

Hemophilia A & B drug market overview

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What are the market dynamics of the hemophilia A & B drug market?

Major drivers of growth in the hemophilia A and B markets include the following:

Rising life expectancy for hemophilia patients, for example, due to reduced morbidity and mortality associated with severe complications and reduced risk of pathogen transmission from plasma-derived sources, increasing the treated population. The continued preference for prophylactic regimens due to proven benefits in terms of bleed rates and joint damage, supported by the availability of and switching to long-acting products and ACPs with lower dosing frequency and improved convenience.

Major barriers to growth in the hemophilia A and B markets include:

Declining prevalent cases of hemophilia, cases in Germany and Spain as well as delayed diagnosis of the disease in China. Possible market access issues for gene therapies due to their high upfront costs, lack of long-term data, and robust evidence of efficacy and safety. Limited scope for new products such as long-acting replacement factors to command a higher price point than current gold standards due to the already high cost of recombinant prophylaxis

What are the key categories of the hemophilia A & B drug market?

The hemophilia A & B drug market is categorized based on the class of drugs into DDAVP (desmopressin), plasma-derived factor concentrates, recombinant factor concentrates, monoclonal antibodies, antisense oligonucleotides, and gene therapies. Recombinant factor concentrates were the leading class of drug in 8MM, in 2020. Gene therapies is expected to have the highest sales in 8MM by 2030 with a market share of at least more than 35% in all of the major markets.

DDAVP, antisense oligonucleotide and gene therapies had the lowest sales in 2020 in 8MM, however, antisense oligonucleotide is expected to have moderate growth during the forecast period, while gene therapies are set to become the leading class of drugs by 2030.

Hemophilia A & B drug market, by key categories

Hemophilia A & B drug market, by key categories

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Which are the major companies in the hemophilia A & B drug market?

The leading companies in the hemophilia A & B drug market, include Aptevo, Bayer, BioMarin, Catalyst Biosciences, CSL Behring, GC Pharma, Kaifeng Pharmaceutical, KM Biologics, LFB, Novo Nordisk, Pfizer, Octapharma, Roche, Sanofi, SinoCelltech, Spark Therapeutics, Takeda, and UniQure. Historically, Takeda has been the dominant player in the recombinant hemophilia market with a wide range of products owned by the company including the rFVIII products Recombinate and Advate. However, the approval of Hemlibra to treat hemophilia A patients with and without inhibitors in 2018 saw Roche leapfrog Takeda to become the market leader. BioMarin is expected to become the market leader by 2030 if its gene therapy product, Roctavian, is approved for use in hemophilia A without inhibitors.

Hemophilia A & B drug market, by key players

Hemophilia A & B drug market, by key players

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Market report scope

Market size (year – 2020) $9.36 billion
Growth rate (CAGR)  >4%
Base year for estimation 2020
Forecast period 2021-2030
High potential countries 8MM (the US, China, Japan, and 5EU (France, Germany, Italy, Spain, and the UK))
Key categories DDAVP (desmopressin), plasma-derived factor concentrates, recombinant factor concentrates, monoclonal antibodies, antisense oligonucleotides, and gene therapies.
Key companies Aptevo, Bayer, BioMarin, Catalyst Biosciences, CSL Behring, GC Pharma, Kaifeng Pharmaceutical, KM Biologics, LFB, Novo Nordisk, Pfizer, Octapharma, Roche, Sanofi, SinoCelltech, Spark Therapeutics, Takeda, and UniQure.

This report provides a comprehensive overview of the hemophilia A & B drug market. It also includes:

  • Overview of Hemophilia A and B including epidemiology, pathophysiology, symptoms, diagnosis, and treatment guidelines.
  • Topline Hemophilia A and B market revenue, the annual cost of therapy, and major pipeline product sales in the forecast period.
  • Key topics: 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 a qualitative analysis of its implications.

Reasons to Buy

  • 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 counterstrategies 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.

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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Table

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

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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