Introduction

Lonoctocog alfa (trade name: Afstyla) has been approved in Germany since January 2017 for the treatment of hemophilia A.

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 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, 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: It can either be extracted from human blood or manufactured using biotechnology.

Lonoctocog alfa is a substitute for factor VIII that is manufactured using biotechnology. It can be used in patients with congenital hemophilia A for the treatment or prevention of bleeding, for instance before a tooth extraction or surgery.

Application

Lonoctocog alfa 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

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

Assessment

In 2017, the Institute for Quality and Efficiency in Health Care (IQWiG, Germany) wanted to look into the advantages and disadvantages of lonoctocog alfa compared with the standard treatments 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 lonoctocog alfa (Afstyla).