Myelofibrosis – Landscape & Forecast – Disease Landscape & Forecast
Myelofibrosis (MF) is a hematological malignancy characterized by disrupted blood cell production and bone marrow scarring. Symptoms include severe anemia, weakness, fatigue, and splenomegaly. The JAK inhibitors ruxolitinib, fedratinib, and pacritinib are the only drugs approved for MF in the United States and EU5 (pacritinib in the United States only). Although JAK inhibitors offer a therapeutic option, they are associated with high discontinuation rates and are unable to address all MF symptoms. Therefore, significant market opportunity remains for safe and tolerable treatments that can effectively manage the full spectrum of MF symptoms (thus reducing the need for polypharmacy) and that can 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 MF? What are physicians’ experience and satisfaction with current therapies?
- How have ruxolitinib, fedratinib, and pacritinib been integrated into the MF treatment algorithm? How will these drugs compete in 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 2031?
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.
SOLUTION ENHANCEMENT
Niche & Rare Disease Landscape & Forecast introduces a new Drug Pipeline chapter with real-time, global pipeline intelligence content directly from Cortellis. This chapter is updated daily and features interactive figures that can be easily downloaded for detailed analysis or presentations.
Table of contents
- Myelofibrosis - Landscape & Forecast - Disease Landscape & Forecast
- Key findings
- Myelofibrosis key findings - December 2022
- Commercial outlook
- Key findings
- Regional sales of key therapies to treat myelofibrosis: 2031
- Regional total treated patients on key therapies to treat myelofibrosis: 2031
- Regional patient shares of key therapies to treat myelofibrosis: 2031
- Myelofibrosis SWOT analysis
- Drivers and constraints
- What factors are driving sales in myelofibrosis?
- What factors are constraining sales in myelofibrosis?
- JAK inhibitor-specific trends
- Key findings
- Forecast
- Sales of key therapies in myelofibrosis: 2031
- 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
- Disease overview
- Epidemiology
- Key findings
- Key updates
- Epidemiology populations
- Disease definition
- Methods and sources used
- Number of diagnosed incident cases of myelofibrosis: 2021-2031
- Disease definition
- Methods and sources used
- Number of diagnosed prevalent cases of myelofibrosis: 2021-2031
- Disease definition
- Methods and sources used for diagnosed incident cases of primary myelofibrosis
- Number of diagnosed incident cases of primary myelofibrosis: 2021-2031
- Disease definition
- Methods and sources used for diagnosed prevalent cases of primary myelofibrosis
- Number of diagnosed prevalent cases of primary myelofibrosis: 2021-2031
- Disease definition
- Methods and sources used for diagnosed incident cases of secondary myelofibrosis
- Number of diagnosed incident cases of secondary myelofibrosis: 2021-2031
- Disease definition
- Methods and sources used for diagnosed prevalent cases of secondary myelofibrosis
- Number of diagnosed prevalent cases of secondary myelofibrosis: 2021-2031
- Disease definition
- Methods and sources used for diagnosed incident cases of myelofibrosis by IPSS risk group
- Diagnosed incident cases of myelofibrosis by IPSS risk group: 2021-2031
- Disease definition
- Methods and sources used for diagnosed prevalent cases of myelofibrosis by DIPSS risk group
- Diagnosed prevalent cases of myelofibrosis by DIPSS risk group: 2021-2031
- Disease definition
- Methods and sources used for diagnosed incident cases of primary myelofibrosis by comorbidity
- Diagnosed incident cases of primary myelofibrosis by comorbidity: 2021-2031
- Disease definition
- Methods and sources used for diagnosed prevalent cases of primary myelofibrosis by comorbidity
- Diagnosed prevalent cases of primary myelofibrosis by comorbidity: 2021-2031
- Disease definition
- Diagnosed incident cases of primary myelofibrosis by biomarker status: 2021-2031
- Disease definition
- Methods and sources used for diagnosed prevalent cases of primary myelofibrosis by biomarker status
- Diagnosed prevalent cases of primary myelofibrosis by biomarker status: 2021-2031
- Disease definition
- Methods and 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: 2021-2031
- Number of diagnosed and 1L drug-treated prevalent cases of myelofibrosis in the major pharmaceutical markets: 2021-2031
- Key findings
- Current treatment
- Key findings
- Diagnosis
- WHO and BSH diagnostic criteria for myelofibrosis, primary myelofibrosis, and prefibrotic myelofibrosis
- IWGu2010MRT 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
- Expert insight: treatment goals
- Key current therapies
- Overview
- Mechanism of action of key current drug classes used for myelofibrosis
- Current treatments used for myelofibrosis
- Clinical trial outcomes for Jakafi / Jakavi
- Advantages and disadvantages of Jakafi / Jakavi
- Key results from select clinical trials investigating Jakafi / Jakavi for the treatment of myelofibrosis
- Key ongoing clinical trials of ruxolitinib in the treatment of myelofibrosis
- Expert insight: Jakafi / Jakavi
- Clinical trial outcomes for Inrebic
- Advantages and disadvantages of Inrebic
- Key results from select clinical trials investigating Inrebic for the treatment of myelofibrosis
- Key ongoing clinical trials of Inrebic in the treatment of myelofibrosis
- Expert insight: Inrebic
- Vonjo
- Clinical trial outcomes for Vonjo
- Advantages and disadvantages of Vonjo
- Key results from select clinical trials investigating Vonjo for the treatment of myelofibrosis
- Key ongoing clinical trials of Vonjo in the treatment of myelofibrosis
- Expert insight: Vonjo
- 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
- Key findings
- 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
- Drug Pipeline
- Pipeline
- Regulatory Milestones
- Indication Comparison
- 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
- 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
- Expert insight: 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
- Pelabresib (CPI-0610) profile
- Key ongoing clinical trials of pelabresib (CPI-0610) in the treatment of myelofibrosis
- Analysis of the clinical development program for pelabresib (CPI-0610)
- Expert insight: pelabresib (CPI-0610)
- Expectations for launch and sales opportunity of pelabresib (CPI-0610)
- Early-phase pipeline analysis
- Select compounds in Phase II development for myelofibrosis
- Patient registries
- Myelofibrosis patient registries
- Prominent myelofibrosis and myeloproliferative neoplasm patient organizations
- Key findings
- Access & reimbursement overview
- Region-specific reimbursement practices
- Expert insight: access and reimbursement
- Expert insight: access and reimbursement
- Region-specific reimbursement practices
- Appendix
- Myelofibrosis bibliography
- Abbreviation table
- Key findings
Vidhi Khosla, M.Sc., is an analyst on the Infectious, Niche, and Rare Diseases team at Clarivate. She has several years of experience providing consulting, research, and analytic support in the pharmaceutical and healthcare domain and has worked in numerous therapy areas, including neurology, women’s health, metabolic disorders, and rare diseases. Prior to joining Clarivate, Ms. Khosla worked at WNS Global Services and DelveInsight Business Research LLP. She earned her bachelor’s and master’s (gold medalist) degrees in biochemistry from Delhi University and Kurukshetra University, respectively.
Muthana Sweis, M.P.H., Healthcare Research & Data Analyst, Infectious, Niche, and Rare Diseases. Muthana conducts primary and secondary research for a range of indications and authors reports related to his findings. He earned his master’s degree in public health from DePaul University.
Rafael Widjajahakim, M.S., is an analyst on the Infectious, Niche, and Rare Diseases team at Clarivate. Prior to joining the company, he was a clinical research coordinator at University of Massachusetts Medical School. He received his master of science degree in clinical investigation from Boston University and his bachelor of science degree in biology from Suffolk University.
Ullas Ulahannan, MPH., Epidemiologist, Epidemiology. Before joining Clarivate, he worked at Jhpiego (a John Hopkins University affiliate) as a senior program officer evaluating, implementing, and monitoring various health programs in collaboration with the state and central governments of India. His qualifications include a master’s degree in public health from Manipal University, Manipal, and a bachelor’s degree in nursing from Rajiv Gandhi University of Health Sciences, Bangalore
