Takeda has announced US FDA approval of the New Drug Application (NDA) for Mimrylo (rusfertide) for the treatment of erythrocytosis in adults with polycythaemia vera (PV), a blood cancer.
Mimrylo is a first-in-class hepcidin mimetic designed to regulate iron distribution in the body and red blood cell overproduction to control haematocrit levels, which is the ratio of red blood cells to the total amount of blood in the body. Maintaining controlled haematocrit levels below 45% is the primary treatment goal in PV.
Affecting approximately 90,000 people in the U.S., PV is characterised by the overproduction of red blood cells (erythrocytosis), leading to elevated haematocrit which can increase blood viscosity, or thickness. This has the potential to result in life-threatening thrombotic events, including stroke, deep vein thrombosis and pulmonary embolism. M
aintaining haematocrit levels consistently below 45% can prevent thrombotic events and alleviate burdensome symptoms, including severe fatigue, pruritus (itching), difficulty concentrating and night sweats. An estimated 78% of patients still experience uncontrolled haematocrit with current standard of care, including phlebotomy and cytoreductive therapies. Patients with PV experiencing uncontrolled haematocrit have a four times higher risk of cardiovascular death or major cardiovascular events.
The approval was supported by data from the global randomised phase 3 study that included 293 patients with PV, showing that Mimrylo met all efficacy endpoints and demonstrated a favourable safety profile.
