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 the MPN Advocates Network.
Introducing
Now you can personalise
your MPN Hub experience!
Bookmark content to read later
Select your specific areas of interest
View content recommended for you
Find out moreThe MPN 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 MPN Hub cannot guarantee the accuracy of translated content. The MPN 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.
The MPN Hub is an independent medical education platform, sponsored by AOP Health and GSK, and supported through an educational grant from Bristol Myers Squibb. 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.
Bookmark this article
The European Medicines Agency (EMA) has granted orphan drug designation to PTG-300 for the treatment of polycythemia vera (PV). PTG-300 is an injectable synthetic hepcidin mimetic peptide that is currently being developed for the treatment of PV and hereditary hemochromatosis.1
In June 2020, PTG-300 was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of PV.
PV is a type of myeloproliferative neoplasm causing elevated levels of red blood cells, which may lead to thrombotic events. Maintaining hematocrit levels below 45% may help reduce the risk of thrombotic events, and PTG-300 provides a non-cytoreductive mechanism targeting iron regulation to control hematocrit levels. Targeting hepcidin may also improve symptoms and systemic iron deficiency in patients with PV.
A phase II study is currently enrolling, and there are ongoing discussions for a pivotal study with PTG-300 in patients with PV, expected to begin in mid-2021.1
Your opinion matters
Subscribe to get the best content related to MPN delivered to your inbox