Myelofibrosis (MF), a rare bone marrow disorder, is one of four chronic myeloproliferative neoplasms (MPN) with no curative therapy except for allogeneic hematopoietic stem cell transplantation. MF is characterized by expansion of a clonal stem cell population leading to bone marrow fibrosis and impaired hematopoiesis that commonly results in anemia, thrombocytopenia, extramedullary hematopoiesis, and splenomegaly along with the presence of constitutional symptoms (e.g., weight loss, fevers, fatigue, malaise, chills, night sweats, and decreased appetite), frequent infections, excessive bruising and bleeding, and a shortened life span.

This report provides an overview of the MF market, featuring a comprehensive analysis of patient populations, current therapies and medical practice, and opportunities for emerging therapies. Current treatment options for MF are very limited and patients with multiple manifestations of the disease often require treatment with several agents. Interviewed experts welcomed the 2011 (US) and 2012 (EU) launch of Incyte/Novartis’ Jakafi/Jakavi (ruxolitinib), the first and only drug approved for MF. While Jakafi/Jakavi offers a therapeutic option for patients with MF, the drug is associated with high discontinuation rates, not all patients benefit from the agent nor does it address the full array of symptoms (e.g., anemia). Hence, significant market opportunity remains for safe and tolerable treatments that can effectively manage symptoms, particularly in patients with baseline thrombocytopenia and anemia. The findings described in this report are derived from secondary research, best-in-class epidemiological analysis, and detailed interviews with experienced U.S. and European hematologists with distinct expertise in diagnosing and treating MF.

Questions Answered in This Report:

  • MF is a rare disease that shares overlapping characteristics with other MPNs, has poorly understood etiology, and a yet unidentified cause of disease progression. What prognostic factors and criteria are used to diagnose MF in the U.S. and EU5 (France, Germany, Italy, Spain, and the United Kingdom)? What are common misdiagnoses and how frequently does misdiagnosis occur? What do interviewed MF experts say about the challenges of diagnosing the disease?

  • In 2014, intermediate- and high-risk primary MF (PMF) accounted for the majority of the diagnosed prevalent population in the United States and the five major European markets and most of these cases were in the aged population. 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 amongst diagnosed incident and prevalent cases of PMF? What are the key subpopulations of those with PMF (e.g., JAK-2(V617F) mutation status, patients with splenomegaly, hepatomegaly, or with peripheral blasts greater than 1%)? How will these population sizes change through 2024?

  • Currently, there are no formal treatment guidelines for MF. What is the current medical practice for the management of disease-related symptoms in patients with MF across markets under study? How does risk level impact treatment approaches? What are the key drivers for prescribing in the MF market and what are the goals of therapy?

  • We forecast the launch of two novel JAK inhibitors in the near-term: CTI BioPharma/Baxter International’s pacritinib will be the first agent in this class to address the needs of MF patients with severe thrombocytopenia while Gilead Sciences’ momelotinib will be the first to improve anemia in patients with MF. What are the key primary and secondary clinical end points upon which investigational therapies are being evaluated? How will head-to-head trials (e.g., momelotinib vs ruxolitinib) impact the uptake of novel JAK inhibitors? How do interviewed MF experts expect these therapies to be integrated into current treatment approaches? What percentage of MF populations will be served by these treatment options by 2024? Which other emerging therapies are poised to meet the unmet needs of subpopulations of patients with MF?

  • Therapies in clinical development for MF span a wide range of drug classes, some of which offer a novel mechanism of action. What are the most promising avenues of research and development? Which unmet needs do interviewed MF experts anticipate will remain unaddressed by the nine profiled emerging therapies for MF that are in Phase II development? What will the therapeutic landscape look like in 2024? How will patient reported outcomes regarding MF-related symptoms impact the approval and labeling of new therapies?

Scope:

Market covered: United States, France, Germany, Italy, Spain, and the United Kingdom.

Primary research: Eight country-specific interviews with thought-leading hematologists with expertise in treating MF.

Epidemiology: Prevalent diagnosed cases of primary MF.

Emerging Therapies: Phase III: 2 Phase II: 9 Phase I/II: 3.

Author(s): Tara M. Stewart, M.A., Ph.D.
Michael Hughes, Ph.D.

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