Mononine, or human coagulation factor IX, is a factor IX concentrate indicated for the prevention and control of bleeding in hemophilia B. The U.S. Food and Drug Administration (FDA) approved its use in 1992, making it the first highly purified factor IX on the U.S. market.
CSL Behring, the therapy’s manufacturer, announced that it was discontinuing this product in 2020 due to falling demand among patients moving to newer, next-generation factor IX treatments.
How Mononine works
Hemophilia B, also known as Christmas disease, is an X-linked inherited blood-clotting disorder characterized by insufficient or abnormal synthesis of factor IX, a blood-clotting protein synthesized in the liver. Due to deficiency of this protein, the blood does not clot properly, causing excessive bleeding. Mononine consists of human plasma, or the liquid part of the blood, which contains human coagulation factor IX. It provides this lacking factor IX and temporarily supports the blood-clotting mechanism.
Studies involving Mononine
Mononine has been investigated in various studies for the treatment of hemophilia B. In one study, its safety and effectiveness were evaluated in 10 patients with hemophilia B. The results, published in the scientific journal Blood, revealed that patients who used Mononine showed an excellent response in hemostasis (the process that causes bleeding to stop) during their bleeding episodes. The results of the study also indicated that the therapy was a hemostatically effective factor IX replacement as it avoided several extraneous thrombogenic substances.
In another study, Mononine’s safety and effectiveness were investigated in people who required factor IX replacement for surgery, trauma, or spontaneous bleeding. The results of this study, published in the American Journal of Hematology, revealed that the rate of hemostasis was excellent. Patients who were administered Mononine showed no thrombotic complications.
Researchers also evaluated the safety and effectiveness of Mononine in two clinical trials with 32 patients who had mild, moderate, or severe hemophilia B and who were previously untreated. The results of the study, published in the journal Thrombosis and Haemostasis, revealed that the rate of hemostasis was excellent in patients who took Mononine.
Other details
Mononine may cause side effects ranging from vomiting and nausea to severe symptoms such as hypersensitivity reactions.
In its 2020 statement on discontinuing this medication, CLS Bering stated that it expected its supply of Mononine to last through mid-2021, at the least.
Updated: Sept. 21, 2020
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