EP0042 granted orphan drug designation for the treatment of AML

On March 14, 2023, EP0042, a dual FLT3 and Aurora kinase inhibitor, was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) as a treatment for patients with acute myeloid leukemia (AML).¹ This follows on from approval by the U.S. FDA of an investigational new drug application for EP0042 in February 2023.²

Patients with AML and mutations in FLT3 are more likely to have a higher risk of relapse and poorer clinical outcomes.¹ Preliminary data from the ongoing phase I/II first-in-human trial of EP0042 in patients with relapsed/refractory AML (NCT04581512) as monotherapy and in combination with standard treatments in patients with FLT3 wild-type and FLT3-mutated AML demonstrated a tolerable safety profile alongside disease stabilization.³