According to a story from Globe Newswire, the gene therapy company uniQure recently announced that it has received conditional marketing authorization for its gene therapy etranacogene dezaparvovec (marketed as HEMGENIX®). This therapy is the first one-time gene therapy approved for the treatment of adults with severe to moderately severe hemophilia type B. The drug is cleared for use in patients that do not have a history of factor IX inhibitor use. As the first gene therapy approved for this disease in the EU, this treatment marks a historic moment for the hemophilia B community and has the potential to transform outcomes for patients.

About Hemophilia

Hemophilia is a genetic disorder which affects the ability of the blood to form clots, a process that is vital for stopping bleeding after a wound is sustained. The severity of symptoms can vary widely. The disorder is caused by a mutation found on the X chromosome. Symptoms include bleeding for a long time after an injury, risk of bleeding in the brain and joints, and easy bruising. Bleeding in the joints can cause permanent damage and brain bleeding can lead to headaches, decreased consciousness, and seizures. There are multiple types of hemophilia, with the most common types being type A and type B, which are distinguished by having deficiencies in different clotting factors. Treatment involves replacing the missing clotting factor. Drugs that thin the blood should be avoided. To learn more about hemophilia, click here.

A New Treatment

Hemophilia B is the result of a deficiency in clotting factor IX. Traditionally, treatment has involved a form of factor replacement therapy. This is done through regular infusions and while this approach is effective for many patients, they are forced to commit to a strict treatment schedule for their entire lives. There are also still patients that experience bleeds and other damaging symptoms, such as severe pain, joint damage, and mobility limitations.

In clinical trials, patients treated with HEMGENIX were able to successfully produce their own factor IX. In the trial, 96% of patients were able to halt their infusions of factor IX. This trial included a total of 67 adult patients.