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Myelofibrosis is defined in the MONDO ontology as partial or complete replacement of the bone marrow stroma by fibrous tissue. The definition describes it as a primary bone marrow lesion within the chronic myeloproliferative disorders (chronic idiopathic myelofibrosis), as a manifestation of acute myeloid leukemia (acute panmyelosis with myelofibrosis), or as a secondary phenomenon of bone marrow involvement by a metastatic tumor such as metastatic breast carcinoma. Prevalence, onset category and inheritance are not supplied in this packet.
The packet contains no curated phenotype annotations, so symptoms, frequencies and age of onset are unknown in this packet.
No genes, inheritance patterns or molecular mechanism are supplied in this packet. The definition describes fibrous replacement of marrow stroma arising in the contexts of a myeloproliferative disorder, acute myeloid leukemia, or metastatic tumor involvement of the marrow.
No diagnostic methods, GeneReviews diagnosis content or newborn-screening information are supplied in this packet, so diagnostic approaches are unknown here.
The packet's FDA records list two active NDA approvals through CDER: Jakafi (ruxolitinib; approved 2011-11-16) and Vonjo (pacritinib; approved 2022-02-28). Orphan-drug records list Jakafi with the indication treatment of myelofibrosis, and also list momelotinib (Ojjaara) with an approved status, although momelotinib is not among the packet's approved-treatment entries. Numerous other agents, including bomedemstat, imetelstat, luspatercept, navitoclax and selinexor, carry orphan designation for myelofibrosis; designation does not constitute FDA approval, and several designation records are marked withdrawn. No foundational-therapy content is supplied.
143 trials found
Prognosis is not certified in this packet; no natural history information is supplied.
ClinicalTrials.gov lists numerous registered studies. Examples in the packet include NCT07357727, a phase 3 study of pelabresib and ruxolitinib (Novartis; recruiting; 2026-05-27 to 2030-12-27); NCT07447817, a phase 2 study of selinexor and pacritinib (John Mascarenhas; recruiting; 2026-10-23 to 2030-05-24); NCT06661915, a phase 2 study of ASTX727 with or without iadademstat in advanced myeloproliferative neoplasms (National Cancer Institute; recruiting; 2025-08-14 to 2027-12-31); and NCT03441113, extended access of momelotinib (GlaxoSmithKline; active, not recruiting; 2018-05-03 to 2028-12-29). Individuals interested in clinical trials can search ClinicalTrials.gov.
Data assembled from 4 of 12 sources · Last updated Nov 3, 2026, 11:11 AM UTC
AI-curated news mentioning myelofibrosis
Updated Sep 30, 2026
The Myelofibrosis drug development pipeline is expanding, with over 50 companies contributing to ongoing clinical trials. Notably, Ryvu Therapeutics is evaluating RVU120, an oral selective inhibitor of CDK8 and CDK19, in a Phase II trial targeting myelofibrosis.
A case report highlights myeloid sarcoma as an extramedullary blast crisis in a patient with BCR-ABL1 e6a2+ chronic myeloid leukemia (CML) complicated by myelofibrosis and tuberculosis. This finding may provide insights into the complex interactions between these conditions.
Lilly reports early positive data for its new JAK inhibitor targeting hard-to-treat myelofibrosis, potentially positioning it as a competitor to Incyte’s Jakafi. The drug was acquired through Lilly's purchase of Ajax, indicating strategic growth in their hematology portfolio.
A study highlights the diagnostic challenges posed by the calreticulin type 26 mutation in myelofibrosis, a rare variant that complicates accurate diagnosis. Understanding this mutation is crucial for improving patient outcomes in myelofibrosis.
Karyopharm Therapeutics faces challenges in securing conventional approval for Xpovio in myelofibrosis after missing a co-primary endpoint in clinical trials. Analysts from Jefferies indicate that this setback could cloud the regulatory path for the drug.