All content on this site is intended for healthcare professionals only. By acknowledging this message and accessing the information on this website you are confirming that you are a Healthcare Professional. If you are a patient or carer, please visit Know AML.

The AML Hub uses cookies on this website. They help us give you the best online experience. By continuing to use our website without changing your cookie settings, you agree to our use of cookies in accordance with our updated Cookie Policy

The AML Hub website uses a third-party service provided by Google that dynamically translates web content. Translations are machine generated, so may not be an exact or complete translation, and the AML Hub cannot guarantee the accuracy of translated content. The AML Hub and its employees will not be liable for any direct, indirect, or consequential damages (even if foreseeable) resulting from use of the Google Translate feature. For further support with Google Translate, visit Google Translate Help.

Steering CommitteeAbout UsNewsletterContact

FDA grants orphan drug designation to OM-301 for the treatment of patients with AML and MM

Apr 20, 2023
Learning objective: After reading this article, learners will be able to cite a new clinical development in AML.

On April 11, 2023, it was announced that OM-301, an investigational drug, was granted orphan drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of patients with multiple myeloma (MM).1,2 This follows its previous orphan drug designation received for the treatment of patients with acute myeloid leukemia (AML) on January 5, 2022.3

OM-301 is a fusion peptide that binds to HDM2 (human double minute-binding protein-2), a protein overexpressed on the cell surface of several types of hematologic malignancies and solid tumors. The attachment of OM-301 to the cancer cell surface leads to a membrane tension that creates pores in those cells and causes cell lysis.1,2,4

OM-301 has shown strong anti-MM activity in preclinical models. In vitro, it has demonstrated cytotoxic effects against multiple myeloma cells lines, including TP53-mutated/null cell lines (L363, RPMI-8226, U266, JJN3, and KMS11), with minimal cytotoxic effects against human peripheral blood mononuclear cells, and has also significantly reduced the tumor volume and prolonged survival within in vivo models.5 In a recent human AML transplanted mouse model, OM-301 demonstrated high efficacy.2 Based on these data, OM-301 presents a novel and effective treatment option for both AML and MM.

The proposed administration for OM-301 is a once daily intravenous infusion given on specified days, which will be determined in upcoming trials. The first phase I/II clinical trial of OM-301 is set to begin in 2023.2

  1.  Oncolyze announces FDA orphan drug designation for OM-301 for the treatment of multiple myeloma. Published Apr 11, 2023. Accessed Apr 18, 2023.
  2. Oncolyze. Pipeline. Accessed Apr 18, 2023. 
  3.  Oncolyze announces FDA orphan drug designation for OM-301 for the treatment of acute myeloid leukemia. Published Jan 5, 2022. Accessed Apr 18, 2023
  4.  Oncolyze. Science. Accessed Apr 18, 2023.
  5. Nigam L, Zhu Y, Troadec E, et al. OM301, a synthetic polypeptide containing the p53TA (transactivation) domain, impairs mitochondrial activity and survival of myeloma cells. Blood. 2021;138(suppl 1):2661. DOI: 1182/blood-2021-151506


Subscribe to get the best content related to AML delivered to your inbox