PTG-300 Hepcidin Mimetic Receives FDA Orphan Drug Designation for Treatment of Polycythemia Vera

Protagonist Therapeutics, Inc. logoNEWARK, 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.

“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.”

Protagonist 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.

About Orphan Drug Designation

The FDA grants Orphan Drug Designation to novel drugs or biologics that treat rare diseases or conditions affecting fewer than 200,000 patients in the U.S. The designation allows the sponsor of the drug to be eligible for a seven-year period of U.S. marketing exclusivity on approval of the drug, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical trial design assistance, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees.  Click here for more information.

Clinical Trial Information

Protagonist Therapeutics clinical trial PTG-300The clinical trial is for a new treatment approach for PV, based on the activity of a natural hormone in the body, hepcidin. The study is for patients receiving several phlebotomies per year, who may or may not be taking any type of medication for polycythemia vera (these may include interferon, ruxolitinib, or cytoreductive medications such as hydroxyurea).

PTG-300, is designed to help regulate the amount of iron available for the production of red blood cells, may decrease the number of phlebotomies that are required, and may provide symptom relief from anemia related to iron deficiency as can occur following phlebotomy. The ongoing study across the U.S. is evaluating the ability for PTG-300 to control the amount of red blood cells (hematocrit level), reduce, delay or eliminate phlebotomy and improve quality of life.

The trial design consists of a 4 to 16-week dose escalation stage, a 12 to 24-week efficacy evaluation and maintenance stage at doses that generate desired hematocrit levels, and a up to 12-week randomized and blinded withdrawal stage. The study has an open-label extension for up to one year. Dosing can be conducted by one weekly self-administered subcutaneous injection.

The protocol allows for accommodations during the COVID-19 pandemic, allowing for local delivery of study medication and alternative study visits (phone or virtual visits), if and as needed.

Additional information is available at


About David Wallace

Founder of PV Reporter, a resource for Myeloproliferative Neoplasm (MPN) patients and caregivers. After being diagnosed with Polycythemia Vera (PV) in 2009, I utilized social media to connect with "informed patients" and develop a better understanding of emerging treatment options. My philosophy on patient care is straight forward - "educating the patient is essential, so the patient can guide their physician to meet his or her needs." PV Reporter is a comprehensive resource hub giving visitors vital tools to become "empowered patients."

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