DRG expects the next ten years to be transitional for patients with hemophilia A, with and without inhibitors, as both the number of therapy options and size of the hemophilia market increase. With the emergence of Roche / Chugai’s bispecific antibody Hemlibra and a diverse pipeline, both in terms of technology type and administration route, we forecast a significant change in approach to disease management for many patients. The R&D emphasis has shifted to nonfactor treatment approaches to restore coagulation. Examples include Sanofi’s anti-ATIII siRNA fitusiran, Novo Nordisk’s anti-TFPI MAb concizumab, and BioMarin’s gene therapy BMN 270. Despite novel nonfactor hemophilia agents forecast to launch, we expect the demand for factor-based concentrates to persist.

QUESTIONS ANSWERED

  • How will physicians treating hemophilia A differentiate between the emerging therapies? Which therapies will gain the greatest market share in an increasingly stringent regulatory environment?
  • What clinical roles will Roche / Chugai’s Hemlibra, Sanofi’s fitusiran, Novo Nordisk’s concizumab, and gene therapy play in the evolving hemophilia A treatment landscape?
  • How will the management of patients with inhibitors change when new treatment options become available? How will Hemlibra’s head start on its novel competitors play out over the forecast period?
  • How will the factor VIII concentrate market evolve? How will nonfactor treatment options impact the multitude of brands vying for patient share in this space?
  • What do interviewed payers advise developers to do to drive market access of new hemophilia drugs? To what extent will the cost of FVIII gene therapy drive its rationing in terms of a patient’s capacity to derive benefit?

PRODUCT DESCRIPTION

Disease Landscape & Forecast provides comprehensive market intelligence with world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.

Table of contents

  • Hemophilia - Landscape & Forecast - Disease Landscape & Forecast
    • Key Updates
      • Q2 2021
        • April 2021
      • Q1 2021
        • March 2021
        • February 2021
        • January 2021
      • Q4 2020
        • December 2020
      • Q3 2020
        • August 2020
      • Q2 2020
        • June 2020
      • Q1 2020
        • March 2020
    • Key Findings
      • Hemophilia A +/- Inhibitors - Key Findings - December 2020
        • March 2020
    • Market Outlook
      • Key Findings
        • Market Share of Drug Classes for Hemophilia A: 2019
        • Market Share of Drug Classes for Hemophilia A: 2029
        • Hemophilia A SWOT Analysis
      • COVID-19: Areas of Potential Forecast Impact
        • Market Drivers and Constraints
          • What Factors Are Driving the Market for Hemophilia A?
          • What Factors Are Constraining the Market for Hemophilia A?
        • Segment-Specific Trends
          • Patient Shares for Prophylactic Treatments of Severe Hemophilia A in the Major Markets: 2019-2029
          • Sales for Prophylactic Treatments of Severe Hemophilia A in the Major Markets: 2019-2029
          • Patient Share of Prophylactic Treatments of Hemophilia A With High-Titer Inhibitors in the Major Markets: 2019-2029
          • Sales of Prophylactic Treatments of Hemophilia A With High-Titer Inhibitors in the Major Markets: 2019-2029
          • Patient Share of On-Demand Treatments of Hemophilia A With High-Titer Inhibitors in the Major Markets: 2019-2029
          • Sales of On-Demand Treatments of Hemophilia A With High-Titer Inhibitors in the Major Markets: 2019-2029
      • Forecast
        • Market Forecast Assumptions - Hemophilia A [Severe - Prophylaxis] (2019-2029) - April 2021
        • Market Forecast Assumptions - Hemophilia A [Moderate - Prophylaxis] (2019-2029) - April 2021
        • Market Forecast Assumptions - Hemophilia A [Mild - Prophylaxis] (2019-2029) - April 2021
        • Market Forecast Assumptions - Hemophilia A [Severe - On Demand] (2019-2029) - April 2021
        • Market Forecast Assumptions - Hemophilia A [Moderate - On Demand] (2019-2029) - April 2021
        • Market Forecast Assumptions - Hemophilia A [Mild - On Demand] (2019-2029) - April 2021
        • Market Forecast Assumptions - Hemophilia A [High-Titer Inhibitors - Prophylaxis] (2019-2029) - April 2021
        • Market Forecast Assumptions - Hemophilia A [Low-Titer Inhibitors - Prophylaxis] (2019-2029) - April 2021
        • Market Forecast Assumptions - Hemophilia A [High-Titer Inhibitors - On Demand] (2019-2029) - April 2021
        • Market Forecast Dashboard - Hemophilia A - Severe - Prophylaxis (2019-2029) - April 2021
        • Market Forecast Dashboard - Hemophilia A - Moderate - Prophylaxis (2019-2029) - April 2021
        • Market Forecast Dashboard - Hemophilia A - Mild - Prophylaxis (2019-2029) - April 2021
        • Market Forecast Dashboard - Hemophilia A - High-Titer Inhibitors - Prophylaxis (2019-2029) - April 2021
        • Market Forecast Dashboard - Hemophilia A - Low-Titer Inhibitors - Prophylaxis (2019-2029) - April 2021
        • Market Forecast Dashboard - Hemophilia A - Severe - On Demand (2019-2029) - April 2021
        • Market Forecast Dashboard - Hemophilia A - Moderate - On Demand (2019-2029) - April 2021
        • Market Forecast Dashboard - Hemophilia A - Mild - On Demand (2019-2029) - April 2021
        • Market Forecast Dashboard - Hemophilia A - High-Titer Inhibitors - On Demand (2019-2029) - April 2021
      • Etiology and Pathophysiology
        • Disease Overview
          • Etiology
            • Genetics of Hemophilia A
          • Pathophysiology
            • Key Pathways and Drug Targets
              • Select Drug Targets for Hemophilia A
              • Select Novel Treatment Approaches for Hemophilia A
          • Epidemiology
            • Key Findings
              • Epidemiology Populations
                • Disease Definition
                • Methods
                • Sources Used for Diagnosed Prevalence of Hemophilia A
                • Number of Diagnosed Prevalent Cases of Hemophilia A in the Major Pharmaceutical Markets: 2019-2029
                • Disease Definition
                • Methods
                • Sources Used for Inhibitor Status of Hemophilia A
                • Diagnosed Prevalent Cases of Hemophilia A with Inhibitors in the Major Pharmaceutical Markets: 2019-2029
                • Drug-Treated Prevalent Cases of Severe Hemophilia A: 2019-2029
                • Drug-Treated Prevalent Cases of Moderate Hemophilia A: 2019-2029
                • Drug-Treated Prevalent Cases of Mild Hemophilia A: 2019-2029
                • Drug-Treated Prevalent Cases of Hemophilia A With High-Titer FVIII Inhibitors: 2019-2029
                • Drug-Treated Prevalent Cases of Hemophilia A With Low-Titer FVIII Inhibitors: 2019-2029
            • Current Treatment
              • Key Findings
                • Treatment Goals
                  • Key Endpoints Used in Clinical Trials for Hemophilia
                • Key Current Therapies
                  • Overview
                  • Mechanism of Action of Key Current Drugs or Drug Classes Used for Hemophilia
                  • Current Treatments Used for Hemophilia A
                  • Ongoing Clinical Development of SHL Factor VIII
                  • Expert Insight: SHL Factor VIII
                  • Ongoing Clinical Development of EHL Factor VIII
                  • Expert Insight: Recombinant FVIII Products
                  • Advantages and Disadvantages of NovoSeven
                  • Ongoing Clinical Development of NovoSeven
                  • Expert Insight: NovoSeven
                  • Advantages and Disadvantages of FEIBA
                  • Ongoing Clinical Development of FEIBA
                  • Expert Insight: FEIBA
                  • Ongoing Clinical Development of Byclot
                  • Expert Insight: Byclot
                  • Advantages and Disadvantages of Hemlibra
                  • Ongoing Clinical Development of Hemlibra
                  • Expert Insight: Hemlibra
                  • Advantages and Disadvantages of Desmopressin
                • Medical Practice
                  • Overview
                  • Country-Specific Guidelines for Hemophilia A+/- Inhibitor Treatment
                  • Factors Influencing Drug Selection in Hemophilia A+/- Inhibitors
                  • Generalized Treatment Decision Tree for Hemophilia A
              • Unmet Need Overview
                • Current and Future Attainment of Unmet Needs in Hemophilia A
                • Top Unmet Needs in Hemophilia A: Current and Future Attainment
                • Expert Insight: Unmet Need in Hemophilia A
              • Emerging Therapies
                • Key Findings
                  • Pipeline Trends in Hemophilia A +/- Inhibitors
                • Key Emerging Therapies
                  • Key Therapies in Development for Hemophilia A+/- Inhibitors
                  • Estimated Launch Dates of Key Emerging Therapies for the Treatment of Hemophilia A
                  • Esperoct Profile
                  • Analysis of the Clinical Development Program for Esperoct
                  • Expert Insight: Esperoct
                  • Expectations for Launch and Sales Opportunity of Esperoct in Hemophilia A
                  • Efanesoctocog alfa (BIVV001) Profile
                  • Analysis of the Clinical Development Program for Efanesoctocog alfa (BIVV001)
                  • Key Development Milestones for Efanesoctocog alfa (BIVV001) in the Treatment of Hemophilia A
                  • Expert Insight: Efanesoctocog alfa (BIVV001)
                  • Expectations for Launch and Sales Opportunity of Efanesoctocog alfa (BIVV001) in Hemophilia A
                  • SEVENFACT Profile
                  • Analysis of the Clinical Development Program for SEVENFACT
                  • Key Development Milestones for SEVENFACT in the Treatment of Hemophilia A
                  • Expectations for Launch and Sales Opportunity of SEVENFACT in Hemophilia A with FVIII Inhibitors
                  • Marzeptacog Alfa (Activated) Profile
                  • Analysis of the Clinical Development Program for Marzeptacog Alfa (Activated)
                  • Key Development Milestones for Marzeptacog Alfa (Activated) in the Treatment of Hemophilia A
                  • Expert Insight: Marzeptacog Alfa (Activated)
                  • Expectations for Launch and Sales Opportunity of Marzeptacog Alfa in Hemophilia A with FVIII Inhibitors
                  • Concizumab Profile
                  • Key Ongoing Clinical Trials of Concizumab in the Treatment of Hemophilia A
                  • Analysis of the Clinical Development Program for Concizumab
                  • Key Development Milestones for Concizumab in the Treatment of Hemophilia A
                  • Expert Insight: Concizumab
                  • Expectations for Launch and Sales Opportunity of Concizumab in Hemophilia A +/- Inhibitors
                  • Fitusiran Profile
                  • Analysis of the Clinical Development Program for Fitusiran
                  • Key Development Milestones for Fitusiran in the Treatment of Hemophilia A
                  • Expert Insight: Fitusiran
                  • Expectations for Launch and Sales Opportunity of Fitusiran in Hemophilia A +/- inhibitors
                  • Marstacimab (PF-06741086) Profile
                  • Key Ongoing Clinical Trials of Marstacimab (PF-06741086) in the Treatment of Hemophilia A
                  • Analysis of the Clinical Development Program for Marstacimab (PF-06741086)
                  • Key Development Milestones for Marstacimab (PF-06741086) in the Treatment of Hemophilia A
                  • Expectations for Launch and Sales Opportunity of Marstacimab (PF-06741086) in Hemophilia A
                  • Roctavian (BMN 270; Valoctocogene Roxaparvovec) Profile
                  • Analysis of the Clinical Development Program for Roctavian (BMN 270; Valoctocogene Roxaparvovec)
                  • Key Development Milestones for Roctavian (BMN 270; Valoctogene Roxaparvovec) in the Treatment of Hemophilia A
                  • Expert Insight: Roctavian (BMN 270; Valoctocogene Roxaparvovec)
                  • Expectations for Launch and Sales Opportunity of Roctavian (BMN 270; Valoctocogene Roxaparvovec) in Hemophilia A
                  • Giroctocogene Fitelparvovec Profile
                  • Key Ongoing Clinical Trials of Giroctocogene Fitelparvovec in the Treatment of Hemophilia A
                  • Analysis of the Clinical Development Program for Giroctocogene Fitelparvovec
                  • Key Development Milestones for Giroctogene Fitelparvovec in the Treatment of Hemophilia A
                  • Expectations for Launch and Sales Opportunity of Giroctocogene Fitelparvovec in Hemophilia A
                  • SPK-8011 Profile
                  • Key Ongoing Clinical Trials of SPK-8011 in the Treatment of Hemophilia A
                  • Analysis of the Clinical Development Program for SPK-8011
                  • Key Development Milestones for SPK-8011 in the Treatment of Hemophilia A
                  • Expectations for Launch and Sales Opportunity of SPK-8011 in Hemophilia A
                  • BAY-2599023 (DTX-201) Profile
                  • Key Ongoing Clinical Trials of BAY-2599023 (DTX-201) in the Treatment of Hemophilia A
                  • Analysis of the Clinical Development Program for BAY-2599023 (DTX-201)
                  • Key Development Milestones for BAY-2599023 (DTX-201) in the Treatment of Hemophilia A
                  • Expectations for Launch and Sales Opportunity of BAY-2599023 (DTX-201) in Hemophilia A
                • Early-Phase Pipeline Analysis
                  • Select Compounds in Early-Phase Development for Hemophilia A
              • Access & Reimbursement Overview
                • Region-Specific Reimbursement Practices
                  • Key Market Access Considerations in Hemophilia A+/- Inhibitors: United States
                  • General Reimbursement Environment: United States
                  • Key Market Access Considerations in Hemophilia A+/- Inhibitors: EU5
                  • General Reimbursement Environment: EU5
                  • Key Market Access Considerations in Hemophilia A+/- Inhibitors: Japan
                  • General Reimbursement Environment: Japan
              • Appendix
                • Key Abbreviations Related to Hemophilia A
                • Brands, Marketers, and Generic Availability of Key Therapies for Hemophilia A +/- Inhibitors: by Market
                • Hemophilia Bibliography

            Author(s): Tim Blackstock, M.B., Ch.B., M.Phil.; Shilpa Thakur

            Tim Blackstock, M.B. Ch.B., M.Phil., is a senior director on the Cardiovascular, Metabolic, Renal, and Hematologic (CMRH) Disorders team at DRG, part of Clarivate. He has authored numerous reports focusing on metabolic indications, chiefly dyslipidemia, obesity, and nonalcoholic steatohepatitis (NASH), and has expertise in renal disorders and hemophilia. He has collaborated extensively with DRG’s Real World Data and Analytics team to provide expert analysis and advice, particularly related to NASH projects. Prior to joining DRG, Dr. Blackstock worked in the medical communications industry, providing scientific writing and editorial assistance to leading pharmaceutical companies in a variety of therapeutic areas. He holds bachelor degrees in medicine and surgery from the University of Otago in New Zealand and practiced medicine for five years, rotating through various specialties, including internal medicine, general surgery, and psychiatry.

            Shilpa Thakur, M.P.H., is an associate epidemiologist at DRG, part of Clarivate. Before joining DRG, she monitored HIV sentinel surveillance in Himachal Pradesh. She has also studied the patterns of antimicrobial resistance in India. She received her M.P.H. from the Postgraduate Institute of Medical Education and Research with a specialization in epidemiology and biostatistics.


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