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.
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.
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.