According to a story from Scoop Independent News, citizens of New Zealand that have the rare bleeding disorder hemophilia A will soon have access to the drug emicizumab (marketed as Hemlibra). This drug will be useful for patients who are no longer seeing benefits from standard clotting factor replacement therapies.
Hemophilia is a genetic disorder which affects the ability of the blood to form clots, a process that is vital for stopping bleeding after a wound is sustained. The severity of symptoms can vary widely. The disorder is caused by a mutation found on the X chromosome. Symptoms include bleeding for a long time after an injury, risk of bleeding in the brain and joints, and easy bruising. Bleeding in the joints can cause permanent damage and brain bleeding can lead to headaches, decreased consciousness, and seizures. There are multiple types of hemophilia, with the most common types being type A and type B, which are distinguished by having deficiencies in different clotting factors. Treatment involves replacing the missing clotting factor. Drugs that thin the blood should be avoided. To learn more about hemophilia, click here.
Why New Treatment is Needed
Hemophilia A is caused by a deficiency in clotting factor VIII. Of the 800 people in New Zealand with hemophilia, 365 of them have this type. For most people standard clotting factor replacement is sufficient to maintain normal blood clotting activity and prevent unexpected bleeding. However, about a quarter of patients eventually develop immune system antibodies to the introduced clotting factor. These antibodies can eventually prevent the replacement factor from working at all.
How Hemlibra Works
Hemlibra will offer a solution to this group of patients that develop antibodies, because it works by mimicking the normal clotting activity that factor VIII is responsible for. The drug can be delivered once per week with an injection under the skin, making administration relatively simple. This is another advantage over earlier treatments, which often require lengthy and frequent infusions.
Dr. Mark Smith of Otago University says that advances in treatment have greatly improved life expectancy for patients, but there is still a serious need for access to testing and consultation with specialists in New Zealand. Hopefully, continued research will allow for Hemlibra to be used with all hemophilia A patients.