Idec's Alprolix Is FDA Approved For Hemophilia B
By Cyndi Root
Biogen Idec announced in a press release that the Food and Drug Administration (FDA) has approved Alprolix. The recombinant DNA-derived therapy for Hemophilia B is the first approved for the condition in 17 years and is the first treatment requiring fewer injections. Alprolix, an orphan drug, controls and prevents bleeding in adults and children. George Scangos, CEO of Biogen Idec said, “Alprolix offers people with hemophilia B the ability to prevent or reduce bleeding episodes with prophylactic infusions starting at least a week apart.”
Hemophilia B
Hemophilia B, also known as factor IX deficiency or Christmas disease, is a common type of hemophilia. Hemophilia A is more common than Hemophilia B, which occurs in 25,000 males of all races and rarely in females. Hemophilia B is a factor IX deficiency and Hemophilia A is a factor VIII deficiency. Most hemophilia cases are hereditary, but in some cases, there is no history in the family and the disorder occurs due to gene mutation. People with hemophilia bleed longer than those without the disease and injuries can become serious. People have mild, moderate, or severe bleeding episodes, depending on factor IX, needed for blood clotting. Bleeding episodes cause hemorrhages, pain, and irreversible joint damage. Infusing factor IX on a regular basis prevents bleeding episodes.
Alprolix
Alprolix is an agent that fuses factor IX to the Fc portion of immunoglobulin G subclass 1 (IgG1). Investigators believe that this process keeps Alprolix in the body longer. Fc fusion is not new, but Biogen Idec has applied it to hemophilia. The new agent has proven effective, is well tolerated by patients, and safe. The approval for the drug includes the prevention of bleeding (prophylaxis) and treatment of bleeding (on-demand). People can use it on a regular schedule for prevention, for surgical procedures, or for bleeding episodes. Biogen intends to make Alprolix available in May.
Alprolix Approval
The FDA approved the drug based on the Phase 3 B-LONG study, safety data, and pharmacokinetic measurements. The B-LONG study included adolescents and adults who received infusions a week or more apart. One group took the drug once a week and another group took it once every 10 days. Investigators found that one dose of Alprolix controlled over 90 percent of bleeding episodes.