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FDA approves Alprolix for hemophilia B
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The FDA today approved the first long-acting recombinant coagulation factor IX concentrate for children and adults with hemophilia B.
Alprolix (Biogen Idec) — which received an orphan-drug designation — is indicated for the control of bleeding episodes, the management of bleeding episodes during surgery and routine prophylaxis. Alprolix demonstrates durable circulation time and thus requires fewer injections to prevent or reduce the frequency of bleeding.
The FDA based its approval in part on results of a multicenter trial that included 123 patients aged 12 to 71 years with severe hemophilia B. Patients were assigned one of two prophylactic treatment regimens or on-demand treatment. During up to 18 months of follow-up, Alprolix demonstrated safety and efficacy in the control and prevention of bleeding episodes, as well as in perioperative management.
“The approval of this product provides another therapeutic option for the treatment and prevention of bleeding in patients with hemophilia B,” Karen Midthun, MD, director of the FDA’s Center for Biologics Evaluation and Research, said in a press release.
Perspective
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Patrick F. Fogarty, MD
Hemophilia has a significant impact on people whom it affects, throughout their lives. Alprolix addresses a critical need by allowing people with hemophilia B to maintain factor levels with prophylactic infusions once weekly or once every 10 days. We hope this will facilitate use of prophylactic therapy.
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Katherine A. High, MD
This is an exciting step forward in the effort to improve life for people with severe hemophilia B. The long-acting factors — of which this is the first to be approved — will allow less frequent dosing, a benefit in and of itself. In addition, based on the dosing regimen selected, it may be possible to reduce the rate of breakthrough bleeding that occurs even in people on prophylactic regimens. As is the case for any new product in hemophilia, there will be a period of adjustment as patients learn to manage their disease in a different way, based on differences in circulating factor levels compared to their prior treatment regimens.
After a period of relatively few new products, therapeutic products in the field of hemophilia are now proliferating, with a number of new treatments now in clinical testing. These include approaches like RNAi knockdown of ATIII, and gene therapy approaches using AAV vectors. Whether any of these, or the new long-acting factors, will improve access for the world’s hemophilia population — most of whom are inadequately treated — remains to be seen.
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