FDA Grants Approval to Pemigatinib for FGFR1 Rearranged Relapsed/Refractory Myeloid/Lymphoid Neoplasms


Pemigatinib had been approved by the FDA as a treatment for patients with FGFR1 rearranged relapsed/refractory myeloid/lymphoid neoplasms.

Pemigatinib (Pemazyre) has been approved by the FDA for the treatment of patients with FGFR1 rearranged relapsed/refractory myeloid/lymphoid neoplasms (MLN), according to a press release from Incyte.

The selective FGFR1 inhibitor was approved based on findings from the phase 2 FIGHT-203 study (NCT03011372), which assessed the agent in a population of 28 patients diagnosed with FGFR1 rearranged relapsed/refractory MLNs. Patients received 13.5 mg once daily of pemigatinib for 21-day cycles on a continuous or an intermittent schedule. Patients received treatment until disease progression, unacceptable toxicity, or they were able to receive an allogeneic hematopoietic stem cell transplant.

Treatment with pemigatinib resulted in a complete response (CR) rate of 78% (95% CI, 52%-94%) among patients with chronic phase in marrow or without extramedullary disease (EMD). Investigators reported a median time to CR of 104 days and the median duration of CR was not reached. Among those with blast phase in the marrow with or without EMD, the CRs were achieved in 2 patients, while 1 patient in the EDM only cohort achieved a CR. The complete cytogenic response rate was 79% (95% CI, 59%-92%) in the overall patient population

“The approval of Pemazyre represents an important treatment advancement for people living with MLNs with FGFR1 rearrangement who currently have limited treatment options,” Hervé Hoppenot, chief executive officer at Incyte, said in a press release. “These are complex hematologic malignancies with a range of presentations, and this approval highlights Incyte’s continued leadership and commitment to advancing care for patients with rare blood cancers.”

Common adverse effects included 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%).


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. August 26, 2022. Accessed August 26, 2022. https://bwnews.pr/3QRq8eE

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