(Health Korea News / Lee Soon-ho) JW Pharmaceutical’s hemophilia treatment ‘Hemlibra (ingredient name: emicizumab)’ has attracted attention with the disclosure of an actual case showing its effectiveness in patients with von Willebrand disease. Although the mechanism of action is different from existing von Willebrand disease treatments, it is expected to expand its role as a treatment for bleeding disorders as the patient’s condition improved.
A joint research team from the Department of Pediatrics and Department of Internal Medicine, University of Illinois College of Medicine, and the Bleeding & Clotting Disorders Institute, recently published a case study paper (case report) in the international academic journal ‘Blood Coagulation and Fibrinolysis’ confirming the bleeding prevention effect of administering ‘Hemlibra’ to three patients with hemophilia A and von Willebrand disease.
Von Willebrand disease is a rare inherited blood clotting disorder that occurs when there is too little or no proper functioning of von Willebrand factor (vWF), a special protein in the blood that helps form blood clots. Bleeding is the main symptom, and it is estimated that it occurs in about 1 in 10,000 people (0.01%). If a patient with hemophilia A also has von Willebrand disease, the severity or frequency of symptoms may increase due to the accompanying bleeding disorder.
Factor VIII replacement therapy, which is primarily used to treat hemophilia A, may not adequately prevent bleeding in patients with both hemophilia A and von Willebrand disease. For this reason, plasma-derived concentrates containing both factor VIII and von Willebrand factor are used in these patients.
However, these complex plasma free concentrates do not have a high patient compliance. There are several reasons, the most notable of which is that the drug must be administered intravenously 2-3 times a week. In particular, children have thin blood vessels and are sensitive to pain, so their compliance is even lower.
For this reason, a joint research team from the University of Illinois School of Medicine in the United States replaced the treatment method for three patients with severe hemophilia A and von Willebrand disease who were deemed inadequately prevented by intravenous injection of complex plasma-derived concentrate containing factor VIII and von Willebrand factor with the single administration of the subcutaneous injection ‘Hemlibra’. As a result, all of these patients experienced a significant decrease in annual bleeding rates (ABR).
The research team explained, “Although the mechanism of action of ‘Hemlibra’ does not directly affect or replace the function of von Willebrand factor, (based on this case) ‘Hemlibra’ can be an effective way to treat patients with hemophilia A and von Willebrand disease by replacing existing complex concentrates.”
‘Hemlibra’ is an antibody-based treatment approved for patients with hemophilia A, a bleeding disorder caused by a deficiency or dysfunction of clotting factor VIII. This treatment, administered as a subcutaneous injection, simultaneously binds to clotting factors IX and X, mimicking the action of factor VIII, thereby preventing or reducing bleeding in patients with hemophilia A. It can also be used in patients who develop antibodies due to factor VIII replacement therapy.
This product was developed by Japan’s Chugai Pharmaceutical, a subsidiary of the global pharmaceutical company Roche. In Korea, JW Pharmaceutical acquired the domestic development and distribution rights in 2017 and is currently selling it as a preventive treatment for severe hemophilia A.
Meanwhile, the number of patients with von Willebrand disease in Korea is not accurately estimated due to the low disease registration rate. According to the Hemophilia White Paper published by the Hemophilia Foundation last year, there are 172 patients with von Willebrand disease registered with the foundation, which is far less than the 5,175 patients, which is 0.01% of the Korean population (prevalence rate of von Willebrand disease).
This is a very small number compared to countries with similar numbers of hemophilia patients to Korea, such as Australia and Poland, and the actual number of patients is estimated to be much higher.
The number of newly registered von Willebrand disease patients with the Hemophilia Foundation is also usually no more than 10 each year. However, according to the Rare Disease Statistical Yearbook published by the Korea Disease Control and Prevention Agency in 2023, the number of newly registered von Willebrand disease patients in Korea (based on the number of newly registered rare disease calculation exceptions) was 83 in 2021 alone, an increase of nearly 1.6 times from 52 the previous year.
Copyright © Health Korea News. Unauthorized reproduction and redistribution prohibited.
Source: www.hkn24.com
