The FDA has granted Orphan Drug Designation to eftilagimod alfa for the treatment of soft tissue sarcoma, supporting its development with incentives such as tax credits and market exclusivity if approved.
Eftilagimod alfa works by activating antigen-presenting cells and strengthening both innate and adaptive immune responses. It boosts CD8+ T cells that help the immune system recognize and attack tumor cells, especially when combined with radiotherapy and immunotherapy such as pembrolizumab.
In the phase 2 EFTISARC-NEO study, the combination of efti, pembrolizumab, and radiotherapy showed strong early results before surgery, with a high rate of tumor fibrosis compared with historical benchmarks and no treatment-related surgical delays. These findings suggest the regimen may improve tumor control before surgery, and it is now moving toward further clinical development.