A Comprehensive Monograph on Palifermin (Kepivance®): From Molecular Biology to Clinical Application

Executive Summary

Palifermin, marketed under the brand name Kepivance®, is a truncated recombinant human keratinocyte growth factor (KGF) representing a significant milestone in supportive oncology care. It is a biotech therapeutic produced in Escherichia coli and specifically engineered for enhanced stability. Its primary, narrowly defined indication is to decrease the incidence and duration of severe oral mucositis (OM) in patients with hematologic malignancies receiving myelotoxic therapy in the setting of autologous hematopoietic stem cell support, particularly with preparative regimens predicted to cause a high incidence of severe mucositis.

The mechanism of action of palifermin is rooted in its function as an agonist of the KGF receptor (FGFR2b), which is selectively expressed on epithelial cells. This interaction stimulates a cascade of cellular activities, including proliferation, differentiation, and the upregulation of cytoprotective mechanisms, effectively thickening and strengthening the mucosal barrier against the cytotoxic insults of chemotherapy and radiation.

The efficacy of palifermin was unequivocally established in a pivotal Phase 3 clinical trial, which demonstrated statistically significant and clinically meaningful reductions in the incidence, duration, and severity of severe OM, leading to decreased patient-reported soreness, reduced opioid use, and less need for parenteral nutrition. However, its efficacy is highly context-specific, with a lack of benefit observed in patients undergoing allogeneic stem cell transplantation or those receiving high-dose melphalan conditioning regimens.