Myelofibrosis | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2019

Myelofibrosis (MF) is a hematological malignancy characterized by the disrupted production of blood cells and bone marrow scarring; symptoms include severe anemia, weakness, fatigue, and splenomegaly. Incyte/Novartis’s Jakafi/Jakavi is the first and only drug approved for myelofibrosis (MF) in the United States and EU5, but it . Although it offers a therapeutic option, the drug is associated with high discontinuation rates and a loss of response. Furthermore, not all patients benefit from Jakafi/Jakavi, nor does the agent address all MF symptoms. Therefore, significant market opportunity remains for safe and tolerable treatments that can effectively manage the full spectrum of symptoms—thereby minimizing the need for polypharmacy over long periods of time—as well as for treatment options for patients with baseline thrombocytopenia and anemia.


  • What are the key diagnostic challenges in MF? What are common misdiagnoses, and how frequently does misdiagnosis occur?
  • 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?
  • How will novel therapies impact the treatment algorithm for MF, and which MF patients will be served by these treatment options in 2028?
  • What are the key unmet needs and areas of commercial opportunity for MF? How well will the MF pipeline address these needs?

Geographies: United States, France, Germany, Italy, Spain, and the United Kingdom and EU5.

Primary Research: Six country-specific interviews with thought-leading hematologists with expertise in treating myelofibrosis. Supported by survey data collected for this study.

Epidemiology: Diagnosed incidence and prevalence of primary and secondary MF by country, diagnosed incidence and prevalence of primary MF by risk (DIPSS and IPSS, respectively), comorbidities and biomarkers (JAK2 mutation, peripheral blasts > 1%, splenomegaly), with population-specific drug-treatment rates.

Forecast: Drug-level sales and patient share of key MF therapies in 2028.

Emerging Therapies: Phase III / PR: 3 drugs. Phase II: > 10 drugs; coverage of select preclinical and Phase I products.


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.

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