Research Reports | Market Assessment
December 2018

Myelofibrosis | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2018

Myelofibrosis 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. Although it offers a therapeutic option for patients with MF, the drug is associated with high discontinuation rates and a loss of response (i.e., patients become refractory to treatment). Furthermore, not all patients benefit from Jakafi/Jakavi, nor does the agent address all MF symptoms (e.g., anemia). 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.

QUESTIONS ANSWERED. 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 2027? What are the key unmet needs and areas of commercial opportunity for MF? How well will the MF pipeline address these needs?

PRODUCT DESCRIPTION Niche & Rare Disease Landscape & Forecast. Comprehensive market intelligence providing 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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