Introduction

Rurioctocog alfa pegol (trade name: Adynovi) has been approved in Germany since January 2018 for the treatment of hemophilia A in adults and children from 12 years of age. Hemophilia is an inherited disorder that affects the blood's ability to clot.

Blood contains several components which cause blood to clot following an injury in order to prevent blood loss. These components include a number of 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, a substitute for clotting factor VIII is needed in order to improve blood clotting. This clotting factor can either be extracted from human blood or manufactured using biotechnology.

Rurioctocog alfa pegol is a substitute for factor VIII that is manufactured using biotechnology. It can be used for the treatment or prevention of bleeding, for instance before a tooth extraction or surgery.

Application

Rurioctocog alfa pegol is injected into a vein. The dose of the drug and duration of treatment will depend on several factors, including the patient's weight and the severity of the condition. The degree of the bleeding and the kind of surgery that is planned will play a role too.

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.

Assessment

In 2018, the Institute for Quality and Efficiency in Health Care (IQWiG, Germany) wanted to look into the advantages and disadvantages of rurioctocog alfa pegol compared with other medications containing clotting factor VIII for people with hemophilia A.

But the manufacturer did not 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 rurioctocog alfa pegol (Adynovi).