Hemophila B is a genetic disorder that's caused by a missing or defective factor IX, otherwise known as a clotting protein, according to the National Hemophilia Foundation. While about a third of cases result from a spontaneous gene mutation, they're typically passed down from parents to children.
The U.S. Food and Drug Administration (FDA) just approved Idelvion, Coagulation Factor IX (Recombinant), Albumin Fusion Protein, for use in both children and adults with hemophilia B. It's the first coagulation factor-albumin fusion protein product approved and the second Factor IX fusion protein product approved in the U.S. that's modified to last longer in the blood.
"The approval of Idelvion provides another important therapeutic option for children and adults with hemophilia B to help prevent or control bleeding and reduce the frequency of bleeding episodes," said Peter Marks, director of the FDA's Center for Biologics Evaluation and Research.
Patients with the disorder are missing the naturally occurring blood clotting factor, Factor IX, or the clotting factor is defective. Idelvion works by using recombinant DNA technology for the product, which lasts longer when given intravenously.
The new drug is given as needed to prevent bleeding episodes in hemophilia B patients. It also helps with the management of bleeding following surgery and as a routine preventative measure to reduce the frequency of bleeding episodes. However, Idelvion may require less frequent injections than unmodified Factor IX when used for prevention.
Researchers used two multicenter studies to test the drug's success, which included 90 adult and pediatric patients with hemophilia B between the ages of 1 and 61. Findings showed that Idelvion helped to control bleeding episodes and manage perioperative bleeding. The drug was also used as prophylaxis, which led to a reduction in the rate of spontaneous bleeding episodes each year, regardless of frequent Idelvion infusions. Meanwhile, the most common observed side effect was headache, with no safety concerns identified at this time.
The U.S. Centers for Disease Control and Prevention (CDC) estimates that hemophilia occurs in about 1 in 5,000 male births. Hemophilia B is about four times less common than hemophilia A, otherwise known as factor VIII deficiency or classic hemophilia, which is caused by a missing or defective factor VIII clotting protein. It is also genetic.
