The introduction of recombinant factor IX was an important step forward in the treatment of hemophilia B, but a single product is not sufficient to meet the needs of all people affected by this disease," said John P.
The IB1001 BLA filing includes a comprehensive set of pharmacokinetics safety, and efficacy data from a Phase 3 clinical trial in people affected by hemophilia B.
John Taylor, Co-Founder and Chairman of Inspiration, stated, "With sons affected by hemophilia B, Inspiration co-founder Scott Martin and I are acutely aware of the impact of limited treatment options on families.
IB1001 is an intravenous rFIX product being developed for the treatment and prevention of bleeding in individuals with hemophilia B.
Earlier-stage preclinical programs at Inspiration are focused on human recombinant factor VIIa (FVIIa), for individuals with either hemophilia A or hemophilia B
who have developed inhibitors, and for individuals with factor VII deficiency; and human recombinant FVIII, to treat individuals with hemophilia A.
Figure 14: Hemophilia B Therapeutics Market, The US, Revenue ($m), 2005-2010 30
Figure 17: Hemophilia B Therapeutics Market, The US, Forecasts ($m), 2010-2018 32
Figure 20: Hemophilia B Therapeutics Market, France, Revenue ($m), 2005-2010 35
Figure 23: Hemophilia B Therapeutics Market, France, Forecast ($m), 2010-2018 37
We are pleased to develop FIX:Fc with Syntonix because Syntonix' SynFusionTM technology has resulted in a very promising, long-acting recombinant Factor IX product that has the potential to reduce the frequency of infusions required for Hemophilia B patients to manage their disease.
One form, Hemophilia B results from mutations that impair the production of Factor IX.
Syntonix' lead product is a long-acting Factor IX product for the treatment of Hemophilia B, which is being developed in a strategic alliance with Biovitrum AB of Sweden.