Hemophilia

History

2023: "Emicizumab" is approved for use in the prevention of patients with moderate hemophilia

Emicizumab, previously approved for the treatment of inhibitory and severe non-inhibitory hemophilia A in the Russian Federation, can now be used for the prevention of patients with moderate hemophilia A. The middle form of hemophilia A, like the severe form, can have a significant impact on the lives of patients: only 15% of patients with moderate hemorrhage are not observed. This was announced on July 4, 2023 by representatives of Roche. Read more here.

2022: Treatment of hemophilia with Hemgenix for $3.5 million

In late November 2022, the United States approved gene therapy for hemophilia from CSL Behring, a drug called Hemgenix that exempts patients from regular treatment. The drug costs $3.5 million per dose, making it the most expensive drug in the world. Read more here.

Hemophilia A

Hemophilia A is a serious hereditary disease associated with impaired clotting function, blood resulting in uncontrolled and often spontaneous bleeding. As of December 2020, hemophilia A affects approximately 320 thousand people worldwide^[1]of which approximately 60% have a severe form of the disease.

In patients with hemophilia A, there is no or insufficient blood coagulation factor VIII - a protein involved in the blood coagulation process. When bleeding occurs in a healthy person, coagulation factor VIII combines coagulation factors IXa and X, an important step in blood clot formation and bleeding arrest. Depending on the severity of the disease, hemophilia A patients may often experience bleeding, especially in the joints or muscles. These bleeds can pose a significant health problem as they often cause pain and can lead to chronic swelling, deformity, reduced mobility and long-term joint damageFranchini ^[2]

A serious complication of treatment is the production of inhibitors to drugs that replace factor VIII. Inhibitors are antibodies produced by the body's immune system that bind to exogenous factor VIII and block its effectiveness, which makes it difficult or impossible to reach a level of factor VIII sufficient to control bleeding.

The number of patients in Russia is 9 thousand people

According to the data announced in September 2019 by the President of the All-Russian Society of Hemophilia, Co-Chairman of the All-Russian Union of Patients Yuri Zhulev, at that time more than 9 thousand Russians have a diagnosis of hemophilia, while over the past decade a serious breakthrough in drug provision has been made and cases of severe disability have significantly decreased, and the life expectancy of patients with hemophilia has almost equaled the indicators of healthy people. At the same time, Yuri Zhulev noted the need to organize modern treatment for patients with severe inhibitory hemophilia A.

"If we talk about children with hemophilia, about 10% of them suffer from a severe form of the disease, the treatment of which in some cases involves intravenous infusions every two to three hours. Poor venous access does not allow proper treatment and relief of bleeding, which leads to the disability of the child in the future. Innovative subcutaneous therapy, registered in Russia in 2018, involves a gentle regimen of administration once a week or once every four weeks, while minimizing episodes of bleeding, "said Yuri Zhulev.

Organizations

• All-Russian Society of Hemophilia (VOG)

Notes