Baxter International Inc. (NYSE:BAX) today announced that the company has submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for approval of RIXUBIS, recombinant factor IX (nonacog gamma) for the treatment and prophylaxis of bleeding in patients of all ages with hemophilia B. Hemophilia B, also known as Christmas disease, is the second most common type of hemophilia and results from insufficient amounts of clotting factor IX, a naturally occurring protein in blood that helps to control bleeding.
RIXUBIS was approved for adult patients and launched in the United States and Puerto Rico earlier in 2013.
The filing is based on results of research with hemophilia B patients previously treated with other therapies. A global Phase I/III study of adult patients with severe or moderately severe hemophilia B demonstrated that twice-weekly prophylactic treatment with RIXUBIS for six months achieved a median annualized bleed rate (ABR) of 2.0, with 43 percent (24/56) of patients experiencing no bleeds. In the study, no patients developed an inhibitory antibody to FIX and no cases of anaphylaxis were reported. The most common adverse reactions observed in >1% of subjects of this study were dysgeusia, and pain in extremity. The application is further supported by data from a study of 23 hemophilia B patients less than twelve years old.
“Our focus at Baxter is to strengthen and expand the portfolio of treatments for patients with bleeding disorders, particularly when there are limited treatment options, such as for patients with hemophilia B,” said Anders Ullman, M.D., Ph.D., vice president of global research and development in Baxter’s BioScience business. “We are actively working to extend access to these treatments and look forward to introducing RIXUBIS in Europe and other countries around the world.”
Current Indications for RIXUBIS
In the United States, RIXUBIS [Coagulation Factor IX (Recombinant)] is an antihemophilic factor indicated for:
- Control and prevention of bleeding episodes in adults with hemophilia B.
- Perioperative management in adults with hemophilia B.
- Routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults with hemophilia B.
RIXUBIS is not indicated for induction of immune tolerance in patients with hemophilia B.