FDA approves new hemophilia drug

FDA has approved Coagulation Factor IX (Recombinant) (Rixubis, Baxter International) for routine prophylactic treatment, control of bleeding episodes, and perioperative management in adults with hemophilia B.

Rixubis is the first new recombinant factor IX (rFIX) approved for hemophilia B in more than 15 years and is the only rFIX indicated for both routine prophylaxis and control of bleeding episodes in the United States for adult patients living with this chronic condition. Hemophilia B is the second most common type of hemophilia and is the result of insufficient amounts of clotting factor IX, a naturally occurring protein in blood that controls bleeding.

“The FDA approval of for Rixubis provides another therapeutic option for patients with hemophilia B after the lack of a new treatment on the market for several years,” said Abimbola Farinde, PharmD, MS, clinical staff pharmacist at Clear Lake Regional Medical Center, Webster, Texas.  “This drug has the potential to reduce bleeding episodes thus potentially improving the quality of life and increasing the ability to engage in daily functions for many individuals who live with this genetic disorder.”

Rixubis’ approval is based on a phase 1/3 study demonstrating that twice-weekly prophylactic treatment with Rixubis for 6 months achieved a median annualized bleed rate (ABR) of 2.0 with 43% of patients experiencing no bleeds. In this study, no patients developed an inhibitory antibody to FIX and no cases of anaphylaxis were reported. One patient developed a transient antibody to furin. Dysgeusia, pain in extremity, and positive test for furin antibody, were the most common adverse reactions observed in >1% of subjects in clinical studies. The study was presented at the 54th Annual Meeting and Exposition of the American Society of Hematology (ASH) in Atlanta.

Baxter was granted orphan-drug designation by FDA as part of the Rixubis application.