The FDA has approved Mimrylo (rusfertide) to treat adults with polycythemia vera whose disease has not been adequately controlled with existing therapies.
Mimrylo is the first approved treatment for polycythemia vera that mimics hepcidin, a hormone that regulates iron in the body, according to an Aug. 28 news release. The drug limits the iron available for red blood cell production, helping prevent the body from making too many red blood cells.
The approval was based on a phase 3 trial of 293 adults who required frequent phlebotomies despite ongoing standard-of-care therapy. Patients were randomized 1:1 to receive Mimrylo or a placebo for 32 weeks. Overall, 76.9% of patients receiving Mimrylo did not require a phlebotomy during the 32-week period, compared with 32.9% of those receiving a placebo. The most common adverse reactions were injection site reactions and anemia.
