FDA Approves Pemazyre (Pemigatinib) for Myeloid/Lymphoid Neoplasms with FGFR1 Rearrangement
Key claim: The FDA approved pemigatinib (Pemazyre) as the first targeted treatment for relapsed/refractory myeloid/lymphoid neoplasms with FGFR1 rearrangement, achieving a 78% complete response rate in chronic phase disease in the FIGHT-203 trial.
Abstract
FDA approved pemigatinib (Pemazyre) for relapsed/refractory myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangement. This marks the first targeted treatment for this patient population, based on the phase 2 FIGHT-203 trial showing 78% complete response rate in chronic phase disease. This regulatory approval has relevance to certain MPN presentations.
Why this matters
This is the first FDA-approved targeted therapy for a molecularly defined myeloid/lymphoid neoplasm subtype outside the classical Ph-negative MPNs, establishing FGFR1-rearranged disease as a distinct, actionable subtype with dramatically different management than PMF or MPN-U. The 78% CR rate in chronic-phase FIGHT-203 patients reframes natural history for a historically aggressive entity previously dependent on intensive chemo and transplant. It also raises the diagnostic bar for the MPN subtype workflow: FGFR1 rearrangement must be excluded before settling on MPN-U or fibrosis-predominant PMF diagnoses.