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.
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
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
Access & reimbursement overview
Region-specific reimbursement practices
Expert insight: access and reimbursement
Expert insight: access and reimbursement
Appendix
Myelofibrosis bibliography
Abbreviation table
Vidhi Khosla
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
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
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.
Shreaa LNU
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