Protagonist Therapeutics Receives FDA Breakthrough Therapy Designation for Rusfertide in Polycythemia Vera

[Manouche]

"We are thrilled to receive the Breakthrough Therapy Designation for rusfertide in PV, a serious disease where the need for different and better treatment options is clear and pressing," said Suneel Gupta, PhD, Chief Development Officer at Protagonist. "

« Designation further validates registrational path for rusfertide in polycythemia vera and facilitates potentially expedited development and review »

prnewswire.com/news-release...

[MPNBlog]

Hi Manouche. Thanks for the link. I have been watching this drug since the report from ASH in December 2020. Some other quotes that I found interesting: "Rusfertide...is for the reduction of erythrocytosis in those patients who do not require further treatment for thrombocytosis and/or leukocytosis." "Rusfertide is a natural hormone mimetic and may stand out as the first non-cytoreductive therapeutic drug for PV. " "The data showed that when treated with rusfertide, a majority of patients were able to eliminate therapeutic phlebotomies, maintain a target hematocrit level of less than 45 percent, reverse iron deficiency, and experience symptom improvements." Sounds good if it delivers. I find Pegasys doesn't keep my red cells in check so I need occasional phlebotomies. I wonder if rusfertide will be more effective for some people with PV but not others, in the way that interferons are effective for some people but not all. Best wishes.

[Manouche in reply to MPNBlog]

Sounds like you could be a good candidate for rusfertide. Pegasys added to rusfertide coud indeed be more effective than Pegasys alone for all those who still need phlebotomies. In my opinion, rusfertide will be available in less than 3 years.

[hunter5582]

Good news! this will be a good option for people with PV when it becomes commonly available.