Protagonist Therapeutics has bagged the orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for PTG-300 for polycythemia vera treatment.
Polycythemia vera is a type of blood cancer, which is mainly characterized by the increased production of red blood cells.
According to the California-based biopharma company, PTG-300 is an injectable synthetic peptide mimetic of the natural hormone hepcidin. The drug candidate is presently in the clinical stage for the treatment of polycythemia vera and also hereditary hemochromatosis, which is a disorder in which the intestine absorbs excessive dietary iron.
Recently, Protagonist Therapeutics disclosed initial phase 2 results in patients with polycythemia vera that showed a strong clinical response and clinically meaningful dose-related control of hematocrit levels on an individual patient basis.
Well-established treatment guidelines for polycythemia vera treatment concentrate on keeping the hematocrit levels under 45% to cut down the risk of thrombotic events. However, the presently available treatment options are not being able to sustain the hematocrit levels under the 45% mark for many patients.
Additionally, current polycythemia vera treatment options are not being tolerated by some patients and could be associated with serious side effects like exacerbation of iron deficiency with phlebotomy.
Commenting on PTG-300 FDA ODD for polycythemia vera treatment, Samuel Saks – Protagonist Therapeutics Chief Medical Officer said: “Receiving FDA orphan drug designation is another important milestone for Protagonist and underscores the importance of our work in polycythemia vera.
“Individuals living with polycythemia vera face a high disease burden. PTG-300 has a non-cytoreductive therapeutic mechanism in the treatment of polycythemia vera and has shown a well-tolerated safety profile to date. Because of its properties, PTG-300 may help provide sustained control of hematocrit and potentially help address symptoms of polycythemia vera and systemic iron deficiency in these patients.”