Myelofibrosis (MF) is a hematological malignancy characterized by disrupted blood cell production and bone marrow scarring. Symptoms include severe anemia, weakness, fatigue, and splenomegaly. Ruxolitinib and fedratinib (JAK inhibitors) are the only approved drugs for MF in the United States and the EU5. Although JAK inhibitors offer a therapeutic option, these agents are associated with high discontinuation rates and are not able to address all MF symptoms. Therefore, significant market opportunity remains for safe and tolerable treatments that can effectively manage the full spectrum of symptoms (thereby reducing the need for polypharmacy), as well as for options to treat patients with thrombocytopenia and anemia.
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