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
  TRANSLATE

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

DSP-5336 granted orphan drug designation by the FDA

Aug 5, 2022
Share:
Learning objective: After reading this article, learners will be able to cite a new clinical development in AML

On August 3, 2022, DSP-5336, a small molecule inhibitor, 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

DSP-5336 is an investigational small-molecule inhibitor that blocks the binding of the scaffold nuclear protein menin to the mixed-lineage leukemia (MLL) protein, an interaction known to drive leukemic transformation. Menin is involved in biological pathways including regulation of cell growth, cell cycle control, and hematopoiesis. Preclinical studies have demonstrated that DSP-5336 has anticancer activity in human AML cell lines with MLL rearrangements or NPM1 mutations.1

A phase I/II dose-escalation and expansion study of DSP-5336 is currently underway in patients with relapsed/refractory AML with or without MLL rearrangements or NPM1 mutations (NCT04988555).

  1. Sumitomo Pharma Oncology, Inc. Sumitomo pharma oncology receives orphan drug designation for DSP-5336, an investigational menin and mixed-lineage leukemia binding protein for treatment of acute myeloid leukemia. https://oncology.sumitomo-pharma.com/news-media/20220803/. Published Aug 3, 2022. Accessed Aug 4, 2022

More about...

Newsletter

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