The Food and Drug Administration (FDA) has approved Pemazyre® (pemigatinib) for the treatment of adults with relapsed or refractory myeloid/lymphoid neoplasms (MLNs) with fibroblast growth factor receptor 1 (FGFR1) rearrangement.

The approval was based on data from the open-label, multicenter phase 2 FIGHT-203 study ( Identifier: NCT03011372), which evaluated the efficacy and safety of pemigatinib, a selective FGFR inhibitor, in 28 patients with relapsed or refractory MLNs with FGFR1 rearrangement.

Patients received 13.5mg orally once daily in 21-day cycles, either on a continuous schedule or as an intermittent schedule. Pemigatinib was administered until disease progression or unacceptable toxicity or until patients were able to receive allogeneic hematopoietic stem cell transplantation.

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Among 18 patients with chronic phase in the marrow with or without extramedullary disease (EMD), findings showed a complete response (CR) rate of 78% (n=14; 95% CI, 52-94) with a median time to response of CR of 104 days (range, 44 to 435 days). The median duration of CR was not reached (range, 1+ to 988+ days).

Among 4 patients with blast phase in the marrow with or without EMD, 2 patients achieved a CR (duration: 1+ and 94 days). Of the 3 patients with EMD only, 1 patient achieved a CR (duration: 64+ days).

The complete cytogenetic response rate in all patients, including 3 patients without evidence of morphologic disease, was 79% (n=22/28; 95% CI, 59-92).

The most common adverse reactions reported were hyperphosphatemia (74%), nail toxicity (62%), alopecia (59%), stomatitis (53%), diarrhea (50%), dry eye (50%), fatigue (44%), rash (35%), abdominal pain (35%), anemia (35%), constipation (32%), dry mouth (32%), epistaxis (29%), retinal pigment epithelial detachment (26%), extremity pain (26%), decreased appetite (24%), dry skin (24%), dyspepsia (24%), back pain (24%), nausea (21%), blurred vision (21%), peripheral edema (21%) and dizziness (21%).

“In patients with relapsed or refractory MLNs with FGFR1 rearrangement treated with Pemazyre in FIGHT-203, the high rate of complete response and complete cytogenetic response in patients with chronic phase disease and the high rate of complete cytogenetic response in patients with blast phase disease is clinically meaningful, especially in light of the lack of these specific responses with existing first-line treatments,” said Dr Srdan Verstovsek, MD, PhD, Professor, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX, and principal investigator for the FIGHT-203 study.

Pemazyre is also indicated for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a FGFR2 fusion or other rearrangement as detected by an FDA-approved test. 


Incyte announces FDA approval of Pemazyre® (pemigatinib) as the first and only targeted treatment for myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangement. News release. Incyte. Accessed August 26, 2022.

This article originally appeared on MPR