Myelofibrosis | Niche and Rare Pharmacor | G7 | 2015

Myelofibrosis (MF), a rare bone marrow disorder, is one of four chronic myeloproliferative neoplasms (MPN) with no curative therapy except for allogeneic hematopoietic stem cell transplantation. MF is characterized by expansion of a clonal stem cell population leading to bone marrow fibrosis and impaired hematopoiesis that commonly results in anemia, thrombocytopenia, extramedullary hematopoiesis, and splenomegaly along with the presence of constitutional symptoms (e.g., weight loss, fevers, fatigue, malaise, chills, night sweats, and decreased appetite), frequent infections, excessive bruising and bleeding, and a shortened life span.

This report provides an overview of the MF market, featuring a comprehensive analysis of patient populations, current therapies and medical practice, and opportunities for emerging therapies. Current treatment options for MF are very limited and patients with multiple manifestations of the disease often require treatment with several agents. Interviewed experts welcomed the 2011 (US) and 2012 (EU) launch of Incyte/Novartis’ Jakafi/Jakavi (ruxolitinib), the first and only drug approved for MF. While Jakafi/Jakavi offers a therapeutic option for patients with MF, the drug is associated with high discontinuation rates, not all patients benefit from the agent nor does it address the full array of symptoms (e.g., anemia). Hence, significant market opportunity remains for safe and tolerable treatments that can effectively manage symptoms, particularly in patients with baseline thrombocytopenia and anemia. The findings described in this report are derived from secondary research, best-in-class epidemiological analysis, and detailed interviews with experienced U.S. and European hematologists with distinct expertise in diagnosing and treating MF.

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