Myelofibrosis: meeting the needs of the ruxolinitib-resistant/intolerant?

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  • In a phase 2 study, the Janus kinase-2 (JAK2)-selective inhibitor fedratinib delivered meaningful clinical benefit to ruxolitinib (Jakafi)-resistant/intolerant patients with myelofibrosis.
  • Further study is required to elucidate toxicities.

Why this matters

  • 50% of patients discontinue ruxolitinib within 3-5 y.
  • An unmet need remains for patients with myelofibrosis who are resistant to or unable to tolerate ruxolitinib.

Study design

  • Phase 2 study to investigate the safety and efficacy of fedratinib in ruxolitinib-resistant/intolerant patients with intermediate/high-risk myelofibrosis.
  • All patients had palpable splenomegaly.
  • 97 patients were enrolled; 83 were assessable for efficacy.
  • Funding: Sanofi.

Key results

  • 55% of patients achieved ≥35% reduction from baseline spleen volume at the end of Cycle 6, including 53% of...