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EP0042 granted orphan drug designation for the treatment of AML

Mar 14, 2023
Learning objective: After reading this article, learners will be able to cite a new development in 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

  1. Business Wire. Ellipses Pharma: EP0043 receives orphan drug designation from the US Food and Drug Administration. . Published Mar 14, 2023. Accessed Mar 14, 2023.
  2. Pharmaceutical Technology. US FDA approves Ellipses Pharma’s IND for AML therapy. Published Feb 01, 2023. Accessed Mar 14, 2023.
  3. D Taussig. A dual FLT3 and Aurora kinase inhibitor: Preliminary results of an ongoing phase I/IIa first in human study in patients with relapsed/ refractory acute myeloid leukemia. Poster #2768. Presented at: 64th American Society of Hematology Annual Meeting and Exposition; Dec 11, 2022; New Orleans, US.