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 (Incyte), fedratinib (Bristol Myers Squibb), pacritinib (CTI BioPharma), and momelotinib (GSK) are the only drugs approved to treat MF in the United States and EU5 (pacritinib and momelotinib only in the United States). 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 MF patients with thrombocytopenia and anemia.
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