The FDA has just approved the drug Rixubis [Coagulation Factor IX (Recombinant)] for people over the age of 16 suffering from hemophilia B.

Hemophilia B is an inherited chronic blood disorder where the blood does not clot properly, it is caused by mutations in the Factor IX gene. The symptoms of hemophilia are characterized by serious bleeding (particularly in the joints) and easy bruising.

New insights into the root causes of uncontrollable bleeding in hemophilia patients were revealed in a study published in Biophysical Journal. The research indicated that blood clots spread in traveling waves through vessels, a finding which may lead to the development of new and more effective treatments.

Rixubis, a purified protein made with recombinant DNA technology, is intended to prevent and control the excessive bleeding associated with hemophilia. The drug is the first new form of therapy for people suffering from hemophilia B, which affects over 3,300 people in the U.S and approximately 25,000 around the world.

Karen Midthun, M.D., director of the FDA’s Center for Biologics Evaluation and Research, said:

“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. This approval provides patients and physicians with an alternative treatment option to prevent or reduce the frequency of bleeding episodes.”

Rixubis is administered by intravenous injection twice a week for the routine prevention of bleeding episodes.

The efficacy of the drug was assessed in a a late-stage trial which included a total of 73 male patients between the ages of 12 to 65 years.

The participants received Rixubis as needed in response to symptoms of bleeding and for routine prophylaxis.

Patients who received Rixubis for routine prophylaxis experienced a significant reduction in annual bleeding – 75 percent lower compared to those who received other treatment options.

Serious and uncommon side effects of the drug include life-threatening allergic reactions.

However, the more common and less severe side effects of the drug include:

  • dysgeusia (distorted taste)
  • atypical blood results
  • pain in an extremity

Researchers at St. Jude Children’s Research Hospital in Memphis found that adults with the hemophilia B experienced significant improvements in their symptoms after a single treatment with gene therapy.

A clinical trial conducted at the University College London (UCL) in the U.K, demonstrated that the treatment is safe. The study appeared in the online edition of the New England Journal of Medicine.

Written by Joseph Nordqvist