I am getting emails and requests from both pharmaceutical companies and now HTCs to help recruit patients for clinical trials. It’s tough: we have great products out there. What’s the incentive to try something experimental?
Some patients do it for free factor. Others do it to try to help advance science.
There has probably not been such a clogged pipeline for new products coming to market (we hope) in maybe 20 years. Patents have expired, which protected products from competition, and now manufacturers are all competing to get new products into the biggest hemophilia market on earth: the United States of America.
Here’s one I just read about:
CSL Behring has announced that the first patient has been enrolled in the pivotal pediatric phase III study to evaluate the safety, efficacy and pharmacokinetics of recombinant fusion protein linking coagulation factor IX with recombinant albumin (rIX-FP) in previously treated children (up to age 11 years). The study site for this first enrollment is the Czech Republic.
CSL Behring, in collaboration with its parent company, CSL Limited, is developing rIX-FP through the PROLONG-9FP clinical trial program for the prophylaxis and treatment of bleeding episodes, including control and prevention of bleeding in surgical settings
in patients with factor IX deficiency.
Results of a Phase I study evaluating recombinant fusion protein linking coagulation Factor IX with albumin (rIX-FP) in patients with severe hemophilia B were publicly presented earlier this year and published in BLOOD 2012 showing that rIX-FP achieved a 91.57 hours terminal half- life, incremental recovery of 1.376 (IU/dL) / (IU/kg), and clearance of 0.75 mL/h/kg. This was an extension in half-life of 5.3 times that of the current recombinant FIX therapy.
We all know that an extended half-life could potentially reduce the number of injections needed in
patients receiving prophylaxis from two or three injections per week to once weekly or significantly less frequently. Several companies and HTCs involved in research, are now in need patient to volunteer to participate.
