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Orphan drug designation granted to PCLX-001 by the FDA

Oct 18, 2022
Learning objective: After reading this article, learners will be able to cite a new development in the treatment of AML.

On October 14, 2022, PCLX-001, a first-in-class, oral, small-molecule N-myristoylation inhibitor currently in clinical development, was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with acute myeloid leukemia (AML).1

PCLX-001 has shown positive results in animal models of AML and in AML patient-derived xenografts, where PCLX-001 treatment reduced human peripheral blood and bone marrow CD45+ cells by up to 95%.1

PCLX-001 is currently being evaluated in a phase I trial in patients with non-Hodgkin lymphoma and solid tumors in Canada (NCT04836195). An investigational new drug application has been filed to study PCLX-001 in patients with AML in the United States.1

  1. Pacylex announces orphan drug designation granted to PCLX-001 for the treatment of acute myeloid leukemia Published Oct 14, 2022. Accessed Oct 17, 2022.

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