FDA approves pemigatinib for myeloid/lymphoid neoplasms with FGFR1 rearrangement
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The FDA approved pemigatinib for treatment of adults with relapsed or refractory myeloid/lymphoid neoplasms and rearrangement of fibroblast growth factor receptor 1, according to a press release from the agent’s manufacturer.
Pemigatinib (Pemazyre, Incyte), a selective fibroblast growth factor receptor (FGFR) inhibitor, received FDA accelerated approval in 2020 for patients with locally advanced or metastatic cholangiocarcinoma whose tumors have a fusion or other rearrangement of FGFR2. The second indication for pemigatinib applies to individuals with myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangement, which may affect fewer than 1 in 100,000 people in the United States, according to an Incyte press release.
“The approval of Pemazyre represents an important treatment advancement for [these] people ... who currently have limited treatment options,” Hervé Hoppenot, CEO of Incyte, said in the 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.”
The FDA based the approval on data from the multicenter, single-arm phase 2 FIGHT-203 study, which included 28 patients with relapsed or refractory MLNs with FGFR1 rearrangement. Patients could have either experienced relapse after or not been candidates for allogeneic hematopoietic stem cell transplantation or other disease-modifying therapies.
Treatment consisted of once-daily pemigatinib, dosed at 13.5 mg in 21-day cycles on a continuous or intermittent (14 days on, 7 days off) schedule, until disease progression, unacceptable toxicity or ability to undergo allogeneic HSCT.
Results showed a complete response rate of 78% (95% CI, 52-94) among 18 patients with chronic-phase disease in the marrow with or without extramedullary disease. Median time to complete response was 104 days (range, 44-435) and median duration of complete response was not reached (range, 1+ to 988+ days).
Researchers also reported complete responses in two of four patients with blast-phase disease in the marrow (duration, 1+ and 94 days) and one of three patients with extramedullary disease only (duration, 64+ days).
In addition, results showed a complete cytogenic response rate among all patients, including three who had no evidence of morphologic disease, of 79% (95% CI, 59-92).
“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,” Srdan Verstovsek, MD, PhD, professor in the department of leukemia in the division of cancer medicine at The University of Texas MD Anderson Cancer Center and principal investigator of the FIGHT-203 study, said in the press release.
The most common adverse events among at least 20% of patients 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%).
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