Pemazyre Now Approved for Relapsed or Refractory Myeloid/Lymphoid Neoplasms with FGFR1 Rearrangement

October 2022, Vol 13, No 5


On August 26, 2022, the FDA accelerated the approval of pemigatinib (Pemazyre; Incyte Corporation), a tyrosine kinase inhibitor, for the treatment of relapsed or refractory myeloid/lymphoid neoplasms with fibroblast growth factor receptor (FGFR)1 rearrangement in adults. The FDA granted pemigatinib a breakthrough therapy designation for this indication.

Pemigatinib was previously approved for the treatment of previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma with FGFR2 fusion or other rearrangement in adults, as detected by an FDA-approved test.

The approval of this new indication was based on the results of the multicenter, open-label, single-arm FIGHT-203 clinical trial that included 28 adults with relapsed or refractory myeloid/lymphoid neoplasms and FGFR1 rearrangement. To be eligible, patients could not be a candidate for or relapsed after having an allogeneic hematopoietic stem-cell transplant (HSCT) or after receiving a disease-modifying therapy, such as chemotherapy. Patients received pemigatinib until disease progression, unacceptable adverse events, or until they were not able to receive an allogeneic HSCT.

The primary efficacy measure was complete response (CR) per the response criteria that was relevant to the morphologic disease type. A total of 14 of the 18 (78%) patients with chronic phase in the marrow with or without extramedullary disease (EMD) achieved a CR (95% confidence interval [CI], 52-94). The median time to CR was 104 days (range, 44-435 days), and the median duration of response was not reached (range, 1-988 days). A total of 2 of the 4 patients with blast phase in the marrow or without EMD had a CR (duration, 1-94 days), and 1 of the 3 patients with EMD only reached a CR (duration, 64 days).

Of the 28 total patients, including 3 patients without morphologic disease, 22 (79%) patients had a complete cytogenetic response (95% CI, 59-92).

The most common (≥20%) adverse events were hyperphosphatemia, nail toxicity, alopecia, stomatitis, diarrhea, dry eye, fatigue, rash, abdominal pain, anemia, constipation, dry mouth, epistaxis, serous retinal detachment, extremity pain, decreased appetite, dry skin, dyspepsia, back pain, nausea, blurred vision, peripheral edema, and dizziness. The most common (≥10%) grade 3 or 4 laboratory abnormalities were decreases in phosphate, lymphocytes, leukocytes, platelets, and neutrophils and increased alanine aminotransferase.

The recommended dose of pemigatinib is 13.5 mg orally once daily until disease progression or unacceptable toxicity.

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