In the 19th century, Queen Victoria passed the Hemophilia B to diverse royal houses of Europe. Today, its characteristic heavy bleedings can be treated quite well, as Swedish Orphan Biovitrum (Sobi) and its partner Biogen showed in the results of an ongoing long-term study.
Sobi was formed in 2001 through a fusion of several units of today’s Pfizer. Dedicated to treating rare diseases, it mostly lives from sales revenues and the manufacturing of Pfizer’s hemophilia treatment. Sobi can actually handle the disease quite well with its proper treatment Alprolix.
Hemophilia B, or Christmas disease, is a genetic disease caused by a mutation in the factor IX gene. Since it’s located on the X chromosome, the disease affects mostly males (males have just one X chromosome, so one mutation means they get the disease) as we know it from the history of European royalty. The corresponding protein, factor IX, is needed for normal blood clotting. In approximately 28,500 patients worldwide, this protein has reduced or no activity, which can result in heavy bleeding. These potentially life-threatening episodes can cause pain and irreversible joint damage.
Sobi developed a treatment that protects the patients prophylactically. Every one or two weeks, the therapy is injected to replace the lacking clotting factors and control potential bleedings. The treatment is the first on the market that links the factor IX to a portion of an immunoglobulin to prolong its blood circulation in the adults and children. It is already approved in the United States, Canada, Australia, and Japan. As for Europe, Alprolix has requested approval from the European Medicines Agency.
Yesterday, the company reported novel results of their ongoing long-term study with children and adults suffering from hemophilia B. The treatment Alprolix could effectively lower the annual bleeding rates to a fifth compared to patients that receive a treatment just at the moment when a bleeding episode occurred.
Another asset: none of the patients in the study developed a so-called inhibitor. These are sometimes produced from the immune system when it recognizes the clotting factor treatment as foreign to the body and starts to fight it. Good for Sobi and Biogen, the non-existence of these inhibitors is the primary endpoint of the study.
All in all, these results show that treating hemophilia is a reality today and there mustn’t be a resistance to the therapy. This is excellent news for all the patients worldwide, including the European ones, which may soon be able to receive it too. It could even have saved the royal family from severe bleedings back in the 19th century.