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.
The MPN 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
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.
PTG-300 receives orphan drug designation from EMA for the treatment of PV
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
- Cision PR Newswire. Protagonist Therapeutics receives orphan drug designation from the European Medicines Agency for PTG-300 in polycythemia vera. https://www.prnewswire.com/news-releases/protagonist-therapeutics-receives-orphan-drug-designation-from-the-european-medicines-agency-for-ptg-300-in-polycythemia-vera-301156341.html. Published Oct 21, 2020. Accessed Oct 22, 2020.
Newsletter
Subscribe to get the best content related to MPN delivered to your inbox