We develop rFIXFc for the treatment of hemophilia B, the goal being that patients will need less frequent prophylactic injections than with currently existing therapies. We have just completed a phase I/II clinical study and based upon the positive outcome a registrational trial will commence.

Biovitrum and Biogen Idec are jointly developing long-acting recombinant coagulation factors including the replacement of both factor VIII and factor IX in hemophilia; rFIXFc being our lead compound. The recently completed phase I/II study showed rFIXFc to be tolerable and the criteria for prolonged plasma half-life were met. Our decision to proceed to a registrational trial signifies something new and very important for Biovitrum. A key factor in reaching our goal to create a long-term profitable company is the launch of our own products and this decision is a large step in that direction.

“The development team has done a great job. The decision to initiate our first registrational program represents true progress in our efforts to offer hemophilia B patients treatment that makes a significant difference.” said Peter Edman, CSO at Biovitrum.
 

 “I am glad that we have reached this milestone for Biovitrum in the development of a longer acting FIX product. The next step in development will be the initiation of the pivotal study which has been named B-LONG. We hope to be able to start this study early next year and Biovitrum will be responsible for the clinical operations in Europe.” says Lena Jendeberg, Biovitrum, who is leading the program together with Samantha Truex, Biogen Idec.

Hemophilia B is a bleeding disorder caused by genetic defects resulting in lack of the blood clotting factor IX, which impacts coagulation. Patients suffering from this disease need injections of Factor IX in order to prevent and stop frequent bleedings that would otherwise lead to pain, irreversible joint damage and possibly life-threatening hemorrhages.

There is an increasing trend toward use of prophylaxis treatments of Hemophilia B, which often requires several infusions per week with the currently available factor IX products. The extended half-life of rFIXFc could enable effective treatment for both prophylaxis and on-demand therapy with less frequent intravenous injections and thereby provide a new, improved therapy for these patients.

For more information, see the press release.