Protagonist Therapeutics, Inc. announced that the European Medicines Agency (EMA) has granted orphan drug designation for PTG-300 in the treatment of polycythemia vera. PTG-300 is an injectable synthetic peptide mimetic of the natural hormone hepcidin and has previously received orphan drug designation for the treatment of polycythemia vera from the U.S. Food and Drug Administration (US FDA).
"This designation reflects the potential of PTG-300 as a treatment candidate and the global need for novel treatments for individuals living with polycythemia vera," commented Samuel Saks, M.D., Protagonist Chief Medical Officer. "Early clinical results that were reported in May, as well as the novel non-cytoreductive therapeutic mechanism of PTG-300 in regulating iron, suggest that PTG-300 may help a broad population of polycythemia vera patients."
He has further added, "We are currently engaged in discussions with leaders in treatment of polycythemia vera to design a pivotal study. Completion of enrollment for the ongoing study of 50 patients is expected in mid-2021. A pivotal study is expected to begin in the second half of 2021, pending our planned discussions with regulatory agencies."
Polycythemia vera is a myeloproliferative neoplasm characterized primarily by the increased production of red blood cells. Well-established treatment guidelines focus on maintaining hematocrit levels continuously below 45 per cent to reduce the risk of thrombotic events. Unfortunately, current treatment options are unable to maintain hematocrit to below the 45 per cent target for many patients and may be associated with serious side effects. There are an estimated 100,000 patients with polycythemia vera in the U.S. and approximately 100,000 patients in major EU countries.