Bob Lojewski, CSL Behring’s senior vice president and general manager of North America, talks about the changes in the treatment landscape for hemophilia B, including the recent use of gene therapy.
Hemophilia B is a rare bleeding disorder caused by dysfunction or deficiency of coagulation Factor IX. People with this condition may bleed for longer periods of time after injury or surgery. They are also susceptible to spontaneous bleeding in muscles, joints, and organs. These symptoms can be extremely painful and, in some cases, life-threatening.
As noted by Mr. Lojewski, the current standard of care for hemophilia B includes life-long prophylactic infusions of Factor IX. However, that type of therapy, over the course of a lifetime, is very expensive, inconvenient, and prone to reduced efficacy.
The New Gene Therapy
Last year, the U.S. Food and Drug Administration (FDA) approved etranacogene dezaparvovec (Hemgenix) for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated serious spontaneous bleeding episodes. Recently, it was announced that the first patient had received this gene therapy. CSL Behring noted that they are in discussion with medical insurance companies to assess best practices moving forward. Their goal is to allow as many patients with hemophilia B as possible to receive this gene therapy. The cost of the one-time therapy is $3.5 million but that could be a bargain for this condition. CSL Behring has noted that the total adult lifetime cost of managing a person with hemophilia B is more than $20 million.
People with hemophilia B interested in receiving this gene therapy can \enroll in the HEMGENIX Connect program where they will be assigned a Patient Resource Navigator and CSL Case Manager, who can assist with questions about gene therapy, the treatment journey, and insurance coverage.
To learn more about hemophilia B and other rare hematological disorders, visit checkrare.com/diseases/hematologic-disorders/