RARE Daily

FDA Grants Protagonist Breakthrough Therapy Designation for Rusfertide in PV

June 3, 2021

The U.S. Food and Drug Administration granted Protagonist Therapeutics Breakthrough Therapy designation for its lead experimental therapy rusfertide for the treatment of the rare blood cancer polycythemia vera.

Photo: Suneel Gupta, chief development officer at Protagonist

Rusfertide is being developed to reduce erythrocytosis in those polycythemia vera (PV) patients who do not require further treatment for thrombocytosis and/or leukocytosis.

Breakthrough Therapy Designation requires that the drug candidate treat a serious or life-threatening disease or condition. It also requires preliminary clinical evidence that indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. The designation has the potential to expedite the development and regulatory review process.

Suneel Gupta, chief development officer at Protagonist, said that PV is a serious disease where the need for different and better treatment options is “clear and pressing.” “Rusfertide is a natural hormone mimetic and may stand out as the first non-cytoreductive therapeutic drug for PV,” he said.

The designation for rusfertide was supported in part by promising data from the ongoing phase 2 clinical trial in patients with 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.

The FDA previously granted orphan drug status and Fast Track designation to rusfertide in PV. Breakthrough Therapy designation offers additional advantages over Fast Track designation, including FDA actions to expedite both planned clinical trials and plans for expediting the manufacturing development strategy.

Author: Rare Daily Staff

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