FDA approves Mimrylo for treatment of rare blood cancer
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The FDA has officially approved Mimrylo (rusfertide) to treat polycythemia vera, providing a first-in-class weekly injection that regulates iron and limits red blood cell overproduction.
The U.S. Food and Drug Administration (FDA) has approved Mimrylo for the treatment of polycythemia vera, a rare, chronic and slowly progressing blood cancer. STAT News reported the decision made on Friday.
Developer and sale of the drug
The active ingredient in Mimrylo is rusfertide, developed by the biotechnology company Protagonist Therapeutics. Japanese pharmaceutical company Takeda will sell the drug under the brand name Mimrylo.
Mimrylo offers a new treatment approach for patients whose disease has not been adequately controlled with existing therapies. It is the first approved treatment for polycythemia vera that mimics hepcidin, a hormone that naturally regulates iron in the body. By limiting the iron available to make new red blood cells, Mimrylo helps reduce their overproduction and keep red blood cell levels under control. This novel approach offers a new option for patients who have not achieved adequate control with existing therapies.
“People living with polycythemia vera have long faced the challenge of managing a chronic blood disorder with frequent blood draws to help address the consequences of the red blood cell overproduction,” said Tanya Wroblewski, M.D., Director of the Division of Nonmalignant Hematology within the FDA’s Center for Drug Evaluation and Research. “Today's approval of Mimrylo offers a new, first-in-class option that has the potential to meaningfully reduce patient burden."
A key goal of treatment for polycythemia vera is keeping the proportion of red blood cells in the blood (hematocrit) below 45% to reduce cardiovascular risks. This often requires phlebotomy, a procedure that removes blood from a vein to lower the number of red blood cells in the body. However, some patients continue to need frequent phlebotomies despite treatment, creating an ongoing burden.
Weekly administration
Mimrylo is administered as an injection once a week. According to STAT News, the drug is a new type of therapy for people with polycythemia vera.
Polycythemia vera causes excessive production of red blood cells. As a result, patients’ blood becomes thicker, increasing the risk of life-threatening heart attacks, strokes and blood clots. The FDA approved Mimrylo specifically for the treatment of this disease.
