Data Updates on Momelotinib for Myelofibrosis


An expert on myelofibrosis discusses recently updated data on momelotinib, which was recently submitted for FDA approval.

Case: A 68-Year-Old Woman with Myelofibrosis

  • A 68-year-old woman presented to her physician with symptoms of mild fatigue, moderate night sweats, and abdominal pain/fullness lasting 4 months; she also reported increased bruising and unexplained weight loss
  • Spleen was palpable 8 cm below the left costal margin
  • Genetic testing shows a JAK2 V617F mutation; CALR negative; Karyotype: 46XX
  • Bone marrow biopsy: megakaryocyte proliferation and atypia with evidence of reticulin fibrosis
  • Blood smear reveals leukoerythroblastosis
  • Diagnosis: Primary myelofibrosis
  • Risk: DIPSS: Intermediate-2; MIPSS70: Intermediate risk
  • Labs demonstrate:
    • RBC 3.40 x 1012/L
    • HGB 13.2 g/dL
    • HCT 36%
    • MCV 94 fL
    • WBC 23.0 x 109/L
    • PLT 450 x 109/L
    • PB Blasts 1%


Abdulraheem Yacoub, MD: In the field of myeloproliferative neoplasms, physicians and patients are very excited about this new advancement and hopefully upcoming approval for momelotinib. Momelotinib is a unique JAK inhibitor that seems to have a significant benefit in anemia in myelofibrosis. In prior studies of momelotinib, there was a signal of activity and spleen reduction and symptom improvement. But a consistent finding in every study for momelotinib was improvement in anemia—sometimes a dramatic improvement—in the majority of patients. This has a physiological pathway that explains this very convincingly through the activin receptor and the blocking of the hepcidin pathway in patients with myelofibrosis.

Based on those observations, a dedicated study, MOMENTUM, was designed to investigate the momelotinib against another active therapy, danazol, in patients with myelofibrosis with high symptom burden and anemia. In these patients, the study was positive: momelotinib was the superior therapy in achieving the primary end point of spleen and symptom improvement. But it also showed a superiority, or noninferiority, in terms of anemia improvement, transfusion independence, and reduction of transfusion needs in these patients. In fact, for patients with anemia response on that study, it showed improvement in survival.

This study has been presented in multiple formats, including at ASH [American Society of Hematology Annual Meeting], showing that this clinical benefit is sustained and has been observed over long-term follow-up with little attrition of benefit over time. Multiple clinical trials of momelotinib have been completed and many patients observed on the study, and there haven’t been any unexpected toxicities of momelotinib. Based on these convincing efficacy and safety data, which have been consistent and concordant across multiple studies, we’re very excited for this advancement. We’re looking forward to this approval for patients with myelofibrosis in the second-line setting who continue to manifest symptoms and myelofibrosis-related anemia. Hopefully we’ll hear about that, and that will become a new therapeutic option for our patients with anemia and myelofibrosis.

Transcript edited for clarity.

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