Today Biogen Idec (NASDAQ: BIIB) announced that Health Canada has approved ALPROLIX™ [Coagulation Factor IX (Recombinant), Fc Fusion Protein], for the control and prevention of bleeding episodes and routine prophylaxis in adults, and children aged 12 and older, with hemophilia B. ALPROLIX is the first approved long-acting hemophilia B therapy and is indicated to prevent or reduce the frequency of bleeding episodes with prophylactic (protective) infusions starting at once weekly or once every 10-14 days.
This is the first regulatory approval worldwide for ALPROLIX, which is currently under review by regulatory authorities in several other countries, including the United States, Australia and Japan.
"Health Canada's approval of ALPROLIX marks the first significant treatment advance in hemophilia B in 17 years, and reinforces our commitment to developing innovative therapies that help address the critical needs of the hemophilia community," said George A. Scangos, Ph.D., chief executive officer of Biogen Idec. "We believe that the safety, efficacy and prophylactic dosing schedule demonstrated with ALPROLIX will provide people with hemophilia B a meaningful new way to manage their condition."
The World Federation of Hemophilia recommends a prophylactic (prevention of bleeding) regimen as the goal of treatment for people with severe hemophilia. Guidelines established by the Medical and Scientific Advisory Council of the National Hemophilia Foundation recommend prophylactic infusions two or more times a week with traditional hemophilia treatments. Frequent prophylactic infusions can be a burden to people with hemophilia and may reduce adoption to this type of treatment regimen.
The Health Canada approval of ALPROLIX is based on results from the global, Phase 3 B-LONG study, the largest registrational study in hemophilia B ever completed. It demonstrated that ALPROLIX safely and effectively prevented, or reduced, bleeding episodes with prophylactic infusions given once weekly or once every 10-14 days in adults and adolescents with severe hemophilia B. In addition, more than 90 percent of all bleeding episodes were controlled by a single ALPROLIX infusion.
"Health Canada's approval of ALPROLIX provides people with hemophilia B an important new option in maintaining a prophylactic regimen," said Manuel Carcao, M.D., Pediatric Hematologist and co-director of the Comprehensive Care Hemophilia Program at the Hospital for Sick Children in Toronto. "ALPROLIX, the first approved long-acting factor concentrate, has been shown to help individuals with hemophilia B achieve effective bleed prevention with prophylactic dosing once a week or once every 10 to 14 days."
Hemophilia B is a rare, chronic, inherited disorder in which the ability of a person's blood to clot is impaired, which can lead to recurrent and extended bleeding episodes. It is due to a substantial reduction of, or no factor IX activity, which is needed for normal blood clotting. People with hemophilia B experience bleeding episodes that can cause pain, irreversible joint damage and hemorrhage. Hemophilia B affects approximately one in 25,000 male births, or about 700 people in Canada. The World Federation of Hemophilia global survey conducted in 2012 estimates that about 28,000 people are currently diagnosed with hemophilia B worldwide.
"The Canadian Hemophilia Society (CHS) is pleased that Health Canada has approved ALPROLIX, a second recombinant factor IX product to treat hemophilia B," said Craig Upshaw, CHS President. "Moreover, it is the first in a promising new class of factor products with extended half-life. We hope it will be available to Canadian patients in the very near future."
Biogen Idec is committed to helping people with hemophilia B and is working with Canadian Blood Services, the Service de Biovigilance du Ministère de la Santé et des Services sociaux and other provincial and national authorities to make ALPROLIX commercially available to all Canadians with hemophilia B.