FDA grants orphan drug designation to GDX012 for R/R AML

On May 25, 2021, the first-in-class GDX012, a blood-based, allogeneic, nongene-edited variable delta 1 gamma-delta T-cell therapy, was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of relapsed/refractory (R/R) acute myeloid leukemia (AML).¹

Although progress has been made in the treatment of AML, there are limited curative therapy options due to the lack of specific targets. Following the investigational new drug application clearance, a phase I multicenter trial of GDX012 will start enrolling patients with measurable residual disease-positive AML to evaluate the safety, tolerability, and antitumor activity of the novel agent.