Roche: Emizizumab

Main article: Hemophilia

Emicizumab is a bispecific antibody against factors IXa and X. It binds factors IXa and X - proteins involved in the natural coagulation cascade and restores the blood clotting process in hemophilia A. Emicizumab is a drug in the form of a finished solution that is administered subcutaneously once a week, every two weeks. or every four weeks (after a loading dose once a week for the first four weeks).

2023: Authorization 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.

As of July 2023, hemophilia A was reported to affect about 900,000 people worldwide, 4.5 approximately 14% of whom have moderate hemophilia. In Russia, about 7,000 patients were registered with a diagnosis of hemophilia A.

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We are sincerely pleased that emicizumab will become an affordable option for therapy, including for patients with moderate hemophilia A and severe phenotype in the Russian Federation, since such patients have bleeding that causes irreversible damage to the joints, which can significantly worsen the quality of life of patients. noted Ilya Nechaev, head of the center for unique expertise of the Russian division of Roche (Rosh-Moscow JSC)

Approaches to the therapy of severe hemophilia A have been carefully studied, recommendations have been developed that are widely used by doctors in clinical practice. Information and recommendations for the treatment of moderate hemophilia A are limited, which may lead to late diagnosis and untimely therapy of bleeding episodes. The severity of hemophilia A, traditionally measured by factor VIII activity, does not always reflect the clinical severity of the patient's condition: patients with a moderate form may have a severe bleeding phenotype (e.g., history of frequent bleeding, frequent hemarthrosis, severe history of bleeding). In addition, many patients with moderate hemophilia A do not receive preventive treatment. Within a year, approximately 85% of patients with moderate hemophilia A experience bleeding, and one in three patients results in long-term joint problems, many of which require surgery, ultimately affecting the patient's quality of life.

The approval of the indication is based on the results of the phase III study HAVEN 6, in which emicism demonstrated clinically significant bleeding control and a favorable safety profile in patients with moderate hemophilia A without factor VIII inhibitors who were shown to be prophylactic.

In patients receiving emicizumab prophylaxis, the median bleeding requiring treatment was 0 (0.00-0.98), with no additional safety signals. Expanding the indications for emicizumab enables patients with moderate hemophilia A and severe bleeding phenotype to receive prophylactic therapy with a favorable efficacy and safety profile. As of July 2023, emicizumab is approved for the treatment of patients with hemophilia A with factor VIII inhibitors in more than 110 countries and for patients without factor VIII inhibitors in more than 100 countries, including the Russian Federation. The drug is being studied in one of the largest clinical trial programs in hemophilia A patients with and without factor VIII inhibitors, including eight phase III studies.

2018: Registration in Russia

Emicizumab has been registered in Russia since October 2018 (marketing authorization No. LP-005110-151018).