FDA Approves 'Alprolix' to Treat Rare Clotting Disorder Hemophilia B

The U.S. Food and Drugs Administration gave clearance to Biogen's Alprolix to treat patients with Hemophilia B. This is the first long acting treatment and requires lesser injections.

The newly approved drug Alprolix, Coagulation Factor IX, is recommended for both adults and children who have hemophilia, an inherited blood clotting disorder.

The drug helps control and prevent bleeding episodes. The drug has Factor IX molecule and this molecule makes the product last longer in circulation.

 "The approval of this product provides another therapeutic option for the treatment and prevention of bleeding in patients with Hemophilia B," Karen Midthun, M.D., director of the FDA's Center for Biologics Evaluation and Research, said in a statement.

Hemophilia B is a rare genetic disorder and is also called Christmas disease and affects those with low levels of blood protein i.e. Factor IX. It is far less common than Hemophilia A and affects 3,300 people in the U.S.. It primarily affects males and they experience repeated episodes of serious bleeding especially in joints, which get damaged due to the excessive bleeding.

The federal agency evaluated the safety and efficacy of the drug in  a clinical trial that compared each of the two prophylactic treatment regimens to on demand treatment.

The study included 123 subjects who had severe Hemophilia B of ages 12-71. They were followed for upto a year and a half.  The trials proved that the drug was effective in preventing and lowering the episodes of bleeding. Also it was effective during the perioperative management of those undergoing surgical procedures. During the trial, no safety concerns were identified.

Since the drug treats a rare disease or condition it received the orphan-drug designation by the federal agency.