A few weeks ago Vector brought you the backstory of how a clotting factor for hemophilia was made to last longer in the blood, allowing injections to be pared to once every week or two, rather than two to three per week.
Developed by Biogen Idec under the trade name Alprolix™, rFIXFc—a modified version of clotting factor IX—is the fruition of a technology first envisioned by three researchers—gastroenterologists Wayne Lencer, MD, of Boston Children’s Hospital, and Richard Blumberg, MD, of Brigham and Women’s Hospital, and immunologist Neil Simister, DPhil, of Brandeis University—for large protein drugs. Their idea: to extend the drugs’ half-lives by protecting them from being ground up by cells.
In the Phase III clinical trial, published in the New England Journal of Medicine, Alprolix stayed in patients’ blood streams nearly five times longer than un-fused factor IX, and dramatically reduced both injection frequency and the rate of bleeding events in patients who received the drug prophylactically.
With the drug on the cusp of clinical availability, patients and their doctors are waiting to learn one more thing: what it will cost. As Ellis Neufeld, MD, PhD, of the Boston Hemophilia Center and Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, told Vector:
“If [the price is] not too high, [Alprolix] could revolutionize the treatment of hemophilia B, because patients might never have to worry about low factor IX levels again.”