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 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 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.
The AML Hub is an independent medical education platform, sponsored by Astellas, Daiichi Sankyo, Johnson & Johnson, Kura Oncology and Syndax, and has been supported through educational grants from Bristol Myers Squibb and the Hippocrate Conference Institute, an association of the Servier Group. The funders are allowed no direct influence on our content. The levels of sponsorship listed are reflective of the amount of funding given. View funders.
Now you can support HCPs in making informed decisions for their patients
Your contribution helps us continuously deliver expertly curated content to HCPs worldwide. You will also have the opportunity to make a content suggestion for consideration and receive updates on the impact contributions are making to our content.
Find out moreCreate an account and access these new features:
Bookmark content to read later
Select your specific areas of interest
View AML content recommended for you
On 14th August 2017, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to H3B-8800 for the treatment of patients with Acute Myeloid Leukemia (AML).1
High frequencies of somatic mutations in core spliceosome genes, including Splicing Factor 3b Subunit 1 (SF3B1) U2 Small Nuclear Ribonucleoprotein Auxiliary Factor 1 (U2AF1) and Serine/Arginine-Rich Splicing Factor 2 (SRSF2), have been reported in AML patients. Mutations in splicesome genes result in aberrant mRNA splicing and have been shown to contribute to leukemogenesis in AML patients. H3B-8800 is an oral, potent and selective inhibitor of SF3B1. H3B-8800 binds and blocks the activity of SF3B1 thereby modulating RNA splicing, which in turn leads to an induction of apoptosis and prevention of cell proliferation. 2,3
H3B-800 is currently being investigated in a phase I study (NCT02841540) which aims to evaluate the safety, pharmacokinetics and pharmacodynamics of H3B-800 in patients with AML.
References