Introduction

Emicizumab (trade name: Hemlibra) has been approved in Germany since March 2018 for the treatment of hemophilia A in people who have antibodies known as factor VIII inhibitors. Since March 2019, it can also be used for the treatment of severe hemophilia A in people who don't have factor VIII inhibitors. Hemophilia is an inherited disorder that affects the blood's ability to clot.

Blood contains several components which quickly cause blood to clot in order to prevent blood loss following injury. These components include various proteins called clotting factors. If some of these clotting factors are missing, the blood will clot more slowly or won’t clot at all. As a result, even small cuts can lead to quite a lot of blood loss.

There are different types of hemophilia, each caused by the lack of a different clotting factor. In hemophilia A, the gene responsible for the production of factor VIII (antihemophilic factor) is defective.

Depending on how severe the disease is, the person needs to be given clotting factor VIII in order to improve blood clotting. This clotting factor can either be extracted from human blood or manufactured using biotechnology. During this replacement treatment, antibodies against factor VIII may develop. These antibodies are also known as inhibitors. They decrease the effectiveness of the factor VIII injections, so excessive bleeding may occur despite treatment.

The drug emicizumab imitates the function of factor VIII in order to enable blood clotting even though inhibitors are present.

Application

During the first four weeks, emicizumab is injected under the skin once a week. After that, it may be used once a week, every two weeks or even every four weeks. The dose is determined based on the person’s body weight. People with hemophilia A can also give themselves injections after a doctor has shown them how.

Other treatments

For decades now, a number of medications with blood clotting factor VIII have been available for the treatment and prevention of bleeding in people with hemophilia A. These medications can be made from donated blood or manufactured artificially using genetically-modified microorganisms or cell cultures. In patients with hemophilia A and factor VIII inhibitors, "bypassing" agents are commonly used too. These sidestep the normal coagulation cascade so they aren’t affected by inhibitors.

Assessment

In 2019, the Institute for Quality and Efficiency in Health Care (IQWiG, Germany) again wanted to look into the advantages and disadvantages of emicizumab compared with the standard treatments for people with hemophilia A.

But the manufacturer didn’t provide any suitable data with which to do the assessment.

More information

This information summarizes the main results of reviews produced by the Institute for Quality and Efficiency in Health Care (IQWiG, Germany). The reviews were commissioned by the German Federal Joint Committee (G-BA) as part of the “early benefit assessment of medications.” On the basis of the reviews and the hearings received, the G-BA passed a resolution on the added benefit of emicizumab (trade name: Hemlibra).