U.S. FDA grants Orphan Drug Designation to the NK cell therapy IGNK001

On April 30, 2024, the U.S. Food and Drug Administration granted Orphan Drug Designation to IGNK001, an investigational allogeneic natural killer cell therapy derived from healthy donors, for the treatment of patients with acute myeloid leukemia.¹ IGNK001 is already approved by the Korean Ministry of Food and Drug Safety, and several U.S. clinical trials are due to be initiated shortly.¹

IGNK001 has recently been investigated in this phase IIb trial (NCT02477787) in 76 patients.² Patients treated with IGNK001 (n = 40) had a lower 30-month cumulative incidence of disease progression vs the control arm (n = 36) (35% vs 61%, respectively).² At 3 months posttransplant, patients treated with IGNK001 demonstrated a 1.8- and 2.6-fold higher median absolute blood count of natural killer and T cells, respectively, compared to patients in the control arm.²