On June 26, the FDA approved Rixubis (coagulation factor IX [recombinant]; Baxter) for routine prophylaxis and control of bleeding episodes in adults with hemophilia B. The drug also was approved for the perioperative management in adults with the bleeding disorder.
According to a Baxter press release, Rixubis is the first new recombinant factor IX (rFIX) approved for hemophilia B in more than 15 years.
“As the first recombinant coagulation factor IX indicated specifically for routine prophylaxis to prevent bleeding, Rixubis becomes a new weapon in our arsenal to protect hemophilia B patients,” Karen Midthun, MD, director of the FDA’s Center for Biologics Evaluation and Research, said in a statement. “This approval provides patients and physicians with an alternative treatment option to prevent or reduce the frequency of bleeding episodes.”
A purified protein generated by recombinant DNA technology, Rixubis is administered by intravenous injection after reconstitution with sterile water, according to an FDA press release. When used for routine prophylaxis, it is administered twice weekly. Rixubis received orphan-drug designation from the FDA.
The approval of Rixubis is based on a Phase I/III multicenter, open-label, uncontrolled study in which 73 male patients between 12 and 65 years of age received the medication for routine prophylaxis or as needed for treatment of bleeding episodes (on-demand). Overall, the annual bleeding rate for patients in the prophylaxis study was 75% lower than for patients who have historically received on-demand treatment, the FDA press release stated.
Serious adverse events, including anaphylaxis, have been reported with factor IX-containing products, the press releases noted. The most common adverse events observed in >1% of patients in clinical studies were dysgeusia, pain in an extremity, and atypical blood test results.