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Hepcidin Mimetic PTG-300 Receives U.S. FDA Orphan Drug Designation for the Treatment of Polycythemia Vera
NEWARK, Calif., June 17, 2020 /PRNewswire/ -- Protagonist Therapeutics, Inc. (Nasdaq: PTGX) today announced that the U.S. Food and Drug Administration (FDA)
About this update from Protagonist Therapeutics, Inc.
[{"type":"text","content":"NEWARK, Calif., June 17, 2020 /PRNewswire/ -- Protagonist Therapeutics, Inc. (Nasdaq: PTGX) today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for PTG-300 for the treatment of polycythemia vera. PTG-300 is an injectable synthetic peptide mimetic of the natural hormone hepcidin currently in clinical development for the treatment of polycythemia vera and hereditary hemochromatosis.\n\"Receiving FDA orphan drug designation is another important milestone for Protagonist and underscores the importance of our work in polycythemia vera,\" commented Samuel Saks, M.D., Protagonist Chief Medical Officer. \"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.\"\nProtagonist recently announced initial Phase 2 results in patients with polycythemia vera that demonstrated robust clinical response and clinically meaningful dose related control of hematocrit levels on individual patient basis. \nAbout Polycythemia Vera\nPolycythemia vera is a myeloproliferative neoplasm characterized primarily by the increased production of red blood cells. Well-established treatment guidelines focus on maintaining hematocrit levels below 45 percent to reduce the risk of thrombotic events. Unfortunately, current treatment options are unable to maintain hematocrit to below the 45 percent target for many patients. In addition, current options are intolerable to some patients and may be associated with serious side effects, such as exacerbation of iron deficiency with phlebotomy. There are an estimated 100,000 patients with polycythemia vera in the U.S. and approximately 100,000 patients in major EU countries. Patients are classified as either low risk or high risk based on prior thrombotic events and age. A treatment option that could provide consistent control of hematocrit over time without fluctuations above 45 percent could be an important component of care for both low and high risk polycythemia vera patient populations.\nAbout Orphan Drug Designation\nThe FDA g...