Polycythemia vera (PV) is a rare blood disorder characterised by the overproduction of red blood cells in the bone marrow. At present, limited therapeutic choices are available for individuals diagnosed with PV, which can effectively regulate haematocrit levels, minimise phlebotomy requirements, and decrease the occurrence of thrombosis. The current standard of care for PV patients is hydroxyurea, whereas agents such as ruxolitinib are used for relapsed/refractory (R/R) patients. Protagonist Therapeutics’s rusfertide is a synthetic compound that mimics hepcidin, acting by binding to ferroportin; this interaction leads to a decrease in the accessibility of iron within the bone marrow, effectively mitigating abnormal erythrocytosis.

At the European Hematology Association (EHA) conference during 8-11 June 2023, results were presented in the late-breaking oral session on the Phase III REVIVE study evaluating the efficacy of rusfertide in patients with PV. The response rate, serving as the primary endpoint of the study, was significantly higher in the rusfertide group compared to the placebo group, with 69.2% of rusfertide-treated patients showing a response, while only 18.5% of placebo-treated patients responded (p=0.0003). Subgroup efficacy analysis revealed that rusfertide was superior to placebo in both concurrent therapy groups: phlebotomy alone at 66.7% in the rusfertide arm and 18.2% for the placebo arm (p=0.02), whereas with phlebotomy with cytotherapy, the rusfertide and placebo arm were reported to be 75% and 18.8%, respectively (p=0.02). During part one of the trial, the phlebotomy-free rate was 76.9% from week one to 17 and increased to 87.3% from week 17 to 29. In part two, the phlebotomy-free rate reached 92.3% in the rusfertide cohort. Rusfertide was generally well tolerated by the participants, with 83% of treatment-emergent adverse events (TEAEs) classified as grade 1–2 and 17% as grade 3 while no grade 4 or 5 TEAEs were reported.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

Protagonist Therapeutics’s rusfertide, estimated to launch in late 2024, is expected to be in fierce competition with JAK inhibitors such as momelotinib currently dominating the PV pipeline. However, considering the potential lethality of the side effects associated with JAK inhibitors, rusfertide appears to hold an advantage. Nonetheless, the recent approval of Besremi in the US and its expected approval in Japan provide the interferon with an enormous opportunity to gain a large PV market share, thus being a direct competitor for rusfertide. GlobalData’s Consensus Forecast database projects global peak sales for rusfertide to reach $388m by 2029.