On August 26, 2022, the FDA accelerated the approval of Pemazyre (pemigatinib; from Incyte), a tyrosine kinase inhibitor, for the treatment of relapsed or refractory myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangements in adults.
Pemazyre was previously approved for patients with advanced or metastatic (spreading) cholangiocarcinoma and an FGFR2 fusion or other rearrangement.
“In patients with relapsed or refractory myeloid/lymphoid neoplasms with FGFR1 rearrangement treated with pemigatinib [Pemazyre] in FIGHT-203, the high rate of complete response…is clinically meaningful,” said Srdan Verstovsek, MD, PhD, of M.D. Anderson Cancer Center.
This approval was based on results of the multicenter FIGHT-203 clinical trial that included 28 patients with relapsed or refractory MLNs and an FGFR1 fusion or other rearrangement. Eligible patients were either not candidates for, or had disease relapse after undergoing, an allogeneic hematopoietic stem-cell transplant or after receiving disease-modifying therapy.
A total of 14 of the 18 (78%) patients with chronic-phase disease, regardless of extramedullary disease (EMD) status, achieved a complete response to treatment with Pemazyre. The average time to complete response was 104 days (range, 44-435 days), and the average duration of response was not reached (range, 1-988 days).
Of the 4 patients with blast-phase disease or without EMD, 2 had a complete response, and 1 of the 3 patients with EMD reached a complete response lasting 64 days.
Of all the total 28 patients, including 3 patients without morphologic disease, 22 (79%) patients had a complete cytogenetic response to Pemazyre.
The most common side effects reported in 20% or more of the patients receiving Pemazyre were hyperphosphatemia, nail problems, hair loss, 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.
