Myelofibrosis (MF) is a hematological malignancy characterized by disrupted blood cell production and bone marrow scarring. Symptoms include severe anemia, weakness, fatigue, and splenomegaly. Ruxolitinib and fedratinib (JAK inhibitors) are the only FDA-approved drugs for MF in the United States, while ruxolitinib is the only approved drug in the EU5. Although JAK inhibitors offer a therapeutic option, these agents are associated with high discontinuation rates and are not able to address all MF symptoms. Therefore, significant market opportunity remains for safe and tolerable treatments that can effectively manage the full spectrum of symptoms (thereby reducing the need for polypharmacy), as well as for options to treat patients with thrombocytopenia and anemia.

QUESTIONS ANSWERED

  • What are the sizes of the U.S. and EU5 diagnosed prevalent and incident primary and secondary MF populations? What are the distributions of risk level among diagnosed incident and prevalent cases of primary MF?
  • What is the current treatment landscape for the management of MF? What are physicians’ experience and satisfaction levels with current key therapies?
  • How have ruxolitinib and fedratinib been integrated into the treatment landscape of MF? How will these drugs compete in the market for the treatment of MF?
  • What are the key unmet needs and areas of commercial opportunity for MF? How will novel therapies impact the treatment algorithm for MF, and which MF patients will be served by these treatment options in 2029?

PRODUCT DESCRIPTION

Niche & Rare 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

  • Myelofibrosis - Landscape & Forecast - Disease Landscape & Forecast
    • COVID-19
      • Key Findings
        • Myelofibrosis Key Findings - October 2020
      • Commercial Outlook
        • Key Findings
          • Regional Sales of Key Therapies to Treat Myelofibrosis: 2029
          • Regional Total Treated Patients on Key Therapies to Treat Myelofibrosis: 2029
          • Regional Patient Shares of Key Therapies to Treat Myelofibrosis: 2029
          • Myelofibrosis SWOT Analysis
        • Drivers and Constraints
          • What Factors Are Driving Sales in Myelofibrosis?
          • What Factors Are Constraining Sales in Myelofibrosis?
        • JAK Inhibitor-Specific Trends
          • Alternative Market Scenarios
            • Alternative Scenarios for the Myelofibrosis Market Through 2029
        • Forecast
          • Sales of Key Therapies in Myelofibrosis
        • Etiology and Pathophysiology
          • Disease Overview
            • Etiology
              • Molecular Genetic Loci Implicated in Myelofibrosis
            • Pathophysiology
              • Constitutive JAK2(V617F) Activation of JAK/STAT Signaling
              • Pathophysiology of Myelofibrosis and Myeloproliferative Neoplasms
            • Clinical Presentation of Myelofibrosis
              • Myelofibrosis Disease Progression
            • Key Pathways and Drug Targets
            • Epidemiology
              • Key Findings
                • Key Updates
              • Epidemiology Populations
                • Disease Definition
                • Methods
                • Sources Used for Diagnosed Incident Cases of Myelofibrosis
                • Number of Diagnosed Incident Cases of Myelofibrosis: 2019-2029
                • Disease Definition
                • Methods
                • Sources Used for Diagnosed Prevalent Cases of Myelofibrosis
                • Number of Diagnosed Prevalent Cases of Myelofibrosis: 2019-2029
                • Disease Definition
                • Methods
                • Sources Used for Diagnosed Incident Cases of Primary Myelofibrosis
                • Number of Diagnosed Incident Cases of Primary Myelofibrosis: 2019-2029
                • Disease Definition
                • Methods
                • Sources Used for Diagnosed Prevalent Cases of Primary Myelofibrosis
                • Number of Diagnosed Prevalent Cases of Primary Myelofibrosis: 2019-2029
                • Disease Definition
                • Methods
                • Sources Used for Diagnosed Incident Cases of Secondary Myelofibrosis
                • Number of Diagnosed Incident Cases of Secondary Myelofibrosis: 2019-2029
                • Disease Definition
                • Methods
                • Sources Used for Diagnosed Prevalent Cases of Secondary Myelofibrosis
                • Number of Diagnosed Prevalent Cases of Secondary Myelofibrosis: 2019-2029
                • Disease Definition
                • Methods
                • Sources Used for Diagnosed Incident Cases of Myelofibrosis by IPSS Risk Group
                • Diagnosed Incident Cases of Myelofibrosis by IPSS Risk Group: 2019-2029
                • Disease Definition
                • Methods
                • Sources Used for Diagnosed Prevalent Cases of Myelofibrosis by DIPSS Risk Group
                • Diagnosed Prevalent Cases of Myelofibrosis by DIPSS Risk Group: 2019-2029
                • Disease Definition
                • Methods
                • Sources Used for Diagnosed Incident Cases of Primary Myelofibrosis by Comorbidity
                • Diagnosed Incident Cases of Primary Myelofibrosis by Comorbidity: 2019-2029
                • Disease Definition
                • Methods
                • Sources Used for Diagnosed Prevalent Cases of Primary Myelofibrosis by Comorbidity
                • Diagnosed Prevalent Cases of Primary Myelofibrosis by Comorbidity: 2019-2029
                • Disease Definition
                • Methods
                • Diagnosed Incident Cases of Primary Myelofibrosis by Biomarker Status: 2019-2029
                • Disease Definition
                • Methods
                • Sources Used for Diagnosed Prevalent Cases of Primary Myelofibrosis by Biomarker Status
                • Diagnosed Prevalent Cases of Primary Myelofibrosis by Biomarker Status: 2019-2029
                • Disease Definition
                • Methods
                • Sources Used for Diagnosed Incident Cases of Secondary Myelofibrosis by MYSEC-PM Risk Group
                • Diagnosed Incident Cases of Secondary Myelofibrosis by MYSEC-PM Risk Group: 2019-2029
                • Number of Diagnosed and Drug-Treated Prevalent Cases of Myelofibrosis in the Major Pharmaceutical Markets: 2019-2029
            • Current Treatment
              • Key Findings
                • Diagnosis
                  • WHO and BSH Diagnostic Criteria for Myelofibrosis, Primary Myelofibrosis, and Prefibrotic Myelofibrosis
                  • IWG‐MRT and BSH Diagnostic Criteria for Post-PV/Post-ET Myelofibrosis
                  • Treatment Providers and Referral Patterns
                • Disease Staging
                  • Prognostic Scoring Systems for Myelofibrosis
                  • Prognostic Risk Scoring and Median Survival of Myelofibrosis Risk Groups
                • Treatment Goals
                  • Key Endpoints Used in Clinical Trials for Myelofibrosis
                • Key Current Therapies
                  • Overview
                  • Mechanism of Action of Key Current Drug Classes Used for Myelofibrosis
                  • Current Treatments Used for Myelofibrosis
                  • Clinical Trial Outcomes for Ruxolitinib
                  • Advantages and Disadvantages of Ruxolitinib
                  • Key Results from Select Clinical Trials Investigating Ruxolitinib for the Treatment of Myelofibrosis
                  • Key Ongoing Clinical Trials of Ruxolitinib in the Treatment of Myelofibrosis
                  • Expert Insight: Ruxolitinib
                  • Advantages and Disadvantages of Cytoreductive Therapies
                  • Expert Insight: Cytoreductive Therapies
                  • Advantages and Disadvantages of Erythropoiesis-Stimulating Agents
                  • Expert Insight: Erythropoiesis-Stimulating Agents
                  • Advantages and Disadvantages of Immunomodulatory Drugs
                  • Expert Insight: Immunomodulatory Drugs
                  • Advantages and Disadvantages of Interferons
                  • Expert Insight: Interferons
                  • Advantages and Disadvantages of Androgens
                  • Expert Insight: Androgens
                  • Advantages and Disadvantages of Red Blood Cell Transfusions
                  • Expert Insight: Red Blood Cell Transfusions
                  • Advantages and Disadvantages of Hypomethylating Agents
                  • Expert Insight: Hypomethylating Agents
                  • Advantages and Disadvantages of Allogeneic Hematopoietic Stem Cell Transplantation
                  • Expert Insight: Allogeneic Hematopoietic Stem Cell Transplantation
                • Medical Practice
                  • Overview
                  • Treatment Guidelines
                  • Country and Regional Myelofibrosis Treatment Guidelines
                  • Generalized Treatment Decision Tree for Myelofibrosis
              • Unmet Need Overview
                • Current and Future Attainment of Unmet Needs in Myelofibrosis
                • Top Unmet Needs in Myelofibrosis: Current and Future Attainment
                • Expert Insight: Unmet Need in Myelofibrosis
              • Emerging Therapies
                • Key Findings
                  • Pipeline Trends for Myelofibrosis
                • Key Emerging Therapies
                  • Key Therapies in Development for Myelofibrosis
                  • Estimated Market Authorization Dates of Key Emerging Therapies for the Treatment of Myelofibrosis
                  • Pacritinib Profile
                  • Key Ongoing Clinical Trials of Pacritinib in the Treatment of Myelofibrosis
                  • Analysis of the Clinical Development Program for Pacritinib
                  • Expert Insight: Pacritinib
                  • Expectations for Launch and Sales Opportunity of Pacritinib in Myelofibrosis
                  • Fedratinib Profile
                  • Key Ongoing Clinical Trials of Fedratinib in the Treatment of Myelofibrosis
                  • Analysis of the Clinical Development Program for Fedratinib
                  • Expert Insight: Fedratinib
                  • Expectations for Launch and Sales Opportunity of Fedratinib in Myelofibrosis
                  • Momelotinib Profile
                  • Key Ongoing Clinical Trials of Momelotinib in the Treatment of Myelofibrosis
                  • Analysis of the Clinical Development Program for Momelotinib
                  • Expert Insight: Momelotinib
                  • Expectations for Launch and Sales Opportunity of Momelotinib in Myelofibrosis
                  • Navitoclax Profile
                  • Key Ongoing Clinical Trials of Navitoclax in the Treatment of Myelofibrosis
                  • Analysis of the Clinical Development Program for Navitoclax
                  • Expert Insight: Navitoclax
                  • Expectations for Launch and Sales Opportunity of Navitoclax
                  • Luspatercept Profile
                  • Key Ongoing Clinical Trials of Luspatercept in the Treatment of Myelofibrosis
                  • Analysis of the Clinical Development Program for Luspatercept
                  • Expert Insight: Luspatercept
                  • Expectations for Launch and Sales Opportunity of Luspatercept
                  • Parsaclisib Profile
                  • Key Ongoing Clinical Trials of Parsaclisib in the Treatment of Myelofibrosis
                  • Analysis of the Clinical Development Program for Parsaclisib
                  • Expectations for Launch and Sales Opportunity of Parsaclisib
                  • Imetelstat Profile
                  • Key Ongoing Clinical Trials of Imetelstat in the Treatment of Myelofibrosis
                  • Analysis of the Clinical Development Program for Imetelstat
                  • Expert Insight: Imetelstat
                  • Expectations for Launch and Sales Opportunity of Imetelstat
                • Early-Phase Pipeline Analysis
                  • Select Compounds in Phase II Development for Myelofibrosis
                • Patient Registries
                  • Myelofibrosis Patient Registries
                  • Prominent Myelofibrosis and Myeloproliferative Neoplasm Patient Organizations
              • Access & Reimbursement Overview
                • Region-Specific Reimbursement Practices
                • Appendix
                  • Key Abbreviations Related to Myelofibrosis
                  • Brands, Marketers, and Generic Availability of Key Therapies for Myelofibrosis by Market
                  • Myelofibrosis Bibliography

              Author(s): Kanika Ujla; Narendra Parihar

              Narendra is an associate epidemiologist within the epidemiology team at Decision Resources Group. Narendra specializes in developing epidemiological forecasts for multiple indications within the DRG syndicated portfolio.

              His qualifications include an MPH with specialization in Health Policy, Economics and Finance from the Tata Institute of Social Sciences, Mumbai, and a Bachelor’s degree in Dentistry from the Rajasthan University of Health Sciences.


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