Therapeutic could improve hemophilia B patients’ quality of life with reduced jabs

vaccineA drug developer has initiated enrollment for a phase 3 trial for a hemophilia therapeutic that could reduce the frequency that people with the rare blood disorder need to take medication. The move is part of a larger trend of biotechnology companies in this area that focus on reducing injections from daily to weekly

CSL Behring, a King of Prussia, Pennsylvania-based company in suburban Philadelphia, specializes in developing plasma protein biotherapeutics.  The pediatric phase 3 study, which starts in the Czech Republic, will evaluate the safety, efficacy and pharmacokinetics (or how the body absorbs and distributes the drug) for recombinant fusion protein linking coagulation factor IX with recombinant albumin (rIX-FP) in previously treated children to aid with blood clotting.

The drug is part of a program at CSL to develop therapeutics to control and prevent bleeding in surgery, and in patients with factor IX deficiency, a rare blood disorder caused by a genetic mutation that interferes with the ability of the patient’s blood to clot.

Last year, it secured orphan drug status for the therapeutic, which gave it a longer patent life and tax incentives to reduce the cost of clinical trials. Drugs given this status are for treating patient populations of 200,000 or under.


Biogen submitted an application for its hemophilia B drug that would pare down the number of intravenous injections to the U.S. Food and Dug Administration for approval earlier this month. The estimated value of the hemophilia B market is $1 billion.

A report by Datamonitor published earlier this month pointed to two emerging trends in the hemophilia market: developing long-acting recombinant factor IX therapeutics and making these drugs more affordable.


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