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Hemophilia A and B - Global Drug Forecast and Market Analysis to 2030

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    Report

  • 134 Pages
  • December 2021
  • Region: Global
  • GlobalData
  • ID: 5530770
Description Jump to:
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 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

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.

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.

Table of Contents

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
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 Outlook13 Appendix
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

Companies Mentioned (Partial List)

A selection of companies mentioned in this report includes, but is not limited to:

  • 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

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