Introduction

Turoctocog alfa pegol (trade name: Esperoct) has been approved in Germany since June 2019 for the treatment of hemophilia A in people aged 12 and over.

Hemophilia is an inherited disorder that affects the blood's ability to clot. Blood contains several components that quickly cause blood to clot following injury, in order to prevent blood loss. These components include various proteins called clotting factors. If some of these clotting factors are missing, the blood will clot more slowly or not 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, there's something wrong with the gene responsible for the production of factor VIII (antihemophilic factor).

Depending on how severe the disease is, the person needs to be given clotting factor VIII in order to improve blood clotting. Clotting factor VIII can either be extracted from human blood or manufactured artificially using genetically-modified microorganisms or cell cultures.

Turoctocog alfa pegol is a substitute for factor VIII that is manufactured using biotechnology to help compensate for the missing clotting factor. It can be used in people with congenital hemophilia A for the treatment or prevention of bleeding, for instance before surgery.

Application

Turoctocog alfa pegol is given as an infusion (a "drip") straight into the bloodstream. The dose depends on several factors: age, body weight and severity of the disease. How extensive the surgery is and how much bleeding is expected also play a role.

Other treatments

For decades now, a number of medications with blood clotting factor VIII have been available for the treatment of hemophilia A. These medications can be made from donated blood or manufactured using biotechnology.

Assessment

In 2019, the Institute for Quality and Efficiency in Health Care (IQWiG, Germany) wanted to look into the advantages and disadvantages of turoctocog alfa pegol compared with the standard treatments for hemophilia A in people aged 12 and over.

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

More information

This information summarizes the main results of a review produced by the Institute for Quality and Efficiency in Health Care (IQWiG, Germany). The review was commissioned by the German Federal Joint Committee (G-BA) as part of the “early benefit assessment of medications.” On the basis of the review and the hearings received, the G-BA passed a resolution on the added benefit of turoctocog alfa pegol (Esperoct).