Introduction

Nonacog beta pegol (trade name: Refixia) has been approved in Germany since June 2017 for the treatment of hemophilia B in people over the age of 12.

Hemophilia is an inherited disorder that affects the blood's ability to clot. Blood contains several components which cause blood to clot in order to prevent blood loss following injury. 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 B, the gene responsible for the production of factor IX (antihemophilic globulin B or Christmas factor) is defective. Depending on how severe the disease is, a substitute for clotting factor IX is needed in order to improve blood clotting. This clotting factor can be extracted from human blood or manufactured using biotechnology.

Nonacog beta pegol is a substitute for factor IX that is manufactured using biotechnology. It can be used in patients with congenital hemophilia B for the treatment or prevention of bleeding, for instance before a tooth extraction or surgery.

Application

Nonacog beta 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 play a role too.

Other treatments

A number of medications containing factor IX are already available for the treatment and prevention of bleeding in people with hemophilia B.

Assessment

In 2018, the Institute for Quality and Efficiency in Health Care (IQWiG, Germany) wanted to look into the advantages and disadvantages of nonacog beta pegol when compared with other medications containing factor IX for the treatment of hemophilia B in people over the age of 12.

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 nonacog beta pegol (Refixia).