FDA Approves Pemigatinib for Relapsed or Refractory Myeloid/Lymphoid Neoplasms With FGFR1 Rearrangement

Pemigatinib is the first targeted therapy to gain FDA approval for the treatment of adult patients with relapsed or refractory myeloid/lymphoid neoplasms with FGFR1 rearrangement.

The FDA has approved pemigatinib (Pemazyre; Incyte) for the treatment of adult patients with relapsed or refractory (R/R) myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangement. Pemigatinib, a selective fibroblast growth factor receptor (FGFR) inhibitor, was evaluated under FDA Priority Review.

Pemigatinib is the first targeted therapy to gain FDA approval for the treatment of adult patients with R/R MLNs with FGFR1 rearrangement.

“The approval of Pemazyre represents an important treatment advancement for people living with MLNs with FGFR1 rearrangement who currently have limited treatment options,” said Hervé Hoppenot, Incyte chief executive officer, in a press release.

The approval was based on data from the phase 2 FIGHT-203 study, a multicenter open-label, single-arm trial analyzing the safety and efficacy of pemigatinib in 28 patients, 18 years of age and older, with R/R MLNs with FGFR1 rearrangement. Patients were enrolled based on having relapsed following allogeneic hematopoietic stem cell transplantation (allo-HSCT), after a disease-modifying treatment, or those were not candidates for allo-HSCT or other disease-modifying treatments.

Patients in the trial were administered pemigatinib 13.5 mg once daily in 21-day cycles, either on a continuous schedule—which is the approved recommended starting dosage for use in patients with MLNs with FGFR1 rearrangement—or on an intermittent schedule of 14 days on, 7 days off—which is an unapproved dosage regimen in MLN with FGFR1 rearrangement. Patients received pemigatinib until disease progression, unacceptable toxicity, or until they were able to receive allo-HSCT.

Enrolled patients included those with documented MLNs with an 8p11 translocation on conventional cytogenetics and/or an FGFR1 rearrangement on break-apart FISH testing. In those with chronic phase in the marrow with or without EMD (N = 18), the complete response (CR) rate was 78% (14/18; 95% CI 52, 94); median time to response of CR was 104 days (range, 44 to 435 days); and median duration of CR was not reached (range, 1+ to 988+ days).

In those with blast phase in the marrow with or without EMD (N = 4), 2 patients achieved a CR (duration: 1+ and 94 days); in those with EMD only (N = 3), 1 patient achieved a CR (duration: 64+ days); and for all patients enrolled (N = 28 including 3 patients without evidence of morphologic disease), the complete cytogenetic response rate was 79% (22/28; 95% CI: 59, 92).

The most common (≥ 20%) adverse events observed in the trial 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%).

MLNs with FGFR1 rearrangement are an extremely rare and aggressive type of blood cancer affecting fewer than 1 in 100,000 people in the United States. Patients with MLN with FGFR1 rearrangement may present with bone marrow involvement with a chronic myeloid malignancy, including myeloproliferative neoplasm (MPN), myelodysplastic syndrome/MPN, or a blast phase malignancy, including B- or T-cell acute lymphoblastic leukemia/lymphoma, acute myeloid leukemia, or mixed phenotype acute leukemia.

“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,” FIGHT-203 study principal investigator Srdan Verstovsek, MD, PhD, professor, Department of Leukemia, Division of Cancer Medicine, University of Texas MD Anderson Cancer Center, said in a press release.


Incyte Announces FDA Approval Of Pemazyre® (Pemigatinib) As The First And Only Targeted Treatment For Myeloid/Lymphoid Neoplasms (MLNs) With FGFR1 Rearrangement. Incyte. News release. August 26, 2022. https://investor.incyte.com/news-releases/news-release-details/incyte-announces-fda-approval-pemazyrer-pemigatinib-first-and

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