About 16,000 individuals in the United States suffer from hemophilia A, a genetic bleeding disorder in which the sufferer's blood does not clot properly because of low levels of the factor VIII gene, an essential blood-clotting protein.
The rare disease, which more often affects males, can cause spontaneous bleeding which can lead to seizures, paralysis, joint disease and even death. Individuals who suffer from bleeding disorders need to replace the missing blood clotting factor regularly so their blood can clot properly preventing serious problems that can be caused by extended bleeding.
Traditional hemophilia A prophylactic therapy, according to the guidelines provided by the National Hemophilia Foundation (NHF), involves infusion every other day or three times a week but a new therapy that has just gotten the approval of the FDA, would allow hemophilia patients to get infusions less frequently.
On Friday, the Food and Drug Administration (FDA) green-lighted Eloctate [Antihemophilic Factor (Recombinant), Fc Fusion Protein] as new treatment for adults and children suffering from hemophilia. The new therapy, which is manufactured by Massachusetts-based biotechnology company Biogen Idec, Inc., is the first hemophilia A therapy that requires less frequent injections to prevent and reduce bleeding episodes.
"Eloctate is the first Hemophilia A treatment designed to require less frequent injections when used to prevent or reduce the frequency of bleeding," the FDA said in a statement. "Eloctate consists of the Coagulation Factor VIII molecule (historically known as Antihemophilic Factor) linked to a protein fragment, Fc, which is found in antibodies."
FDA's approval of Elocatate, which was co-developed by the Swedish Orphan Biovitrum (SOBI), was based on results of the phase 3 A-LONG clinical study which identified no safety concerns and showed the therapy's effectiveness. The drug was approved for controlling and preventing bleeding, management of bleeding in surgical procedures and reducing and preventing the frequency of bleeding episodes.
Unlike with traditional treatments, Biogen's Eloctate is the only therapy with prophylactic infusions, or the infusion of clotting factor concentrate into the patient's vein, that can be done every three to five days. In its statement announcing FDA's approval of Eloctate, Biogen said that the therapy was developed using a process known as Fc fusion, which prolongs its circulation in the patient's blood.
"Prophylactic treatment is recommended for people with severe hemophilia, and following a protective regimen can be burdensome given the frequency of infusions required," said Penn Comprehensive Hemophilia and Thrombosis Program director Patrick Fogarty. "Infusion frequency is a major challenge for people with hemophilia, and I believe ELOCTATE begins to address this burden while protecting against bleeding episodes."
