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| Hemophilia | |
| FIXFc and FVIIIFc Syntonix and Biovitrum AB of Sweden have a strategic alliance to jointly develop and commercialize FIXFc as a treatment for Hemophilia B and FVIIIFc for Hemophilia A. Upon approval, Syntonix is responsible for marketing in North America and Biovitrum is responsible for marketing in Europe, Russia and the Middle East. The companies share the costs and profits for development and commercialization of FIXFc. FIXFc has the potential to reduce the frequency of intravenous injections required for Hemophilia B patients to manage their disease through prophylaxis treatment or on demand therapy. Syntonix is evaluating the potential of FVIIIFc constructs to treat Hemophilia A and has other early stage programs with potential application to improve therapy for hemophilia patients. About Hemophilia Hemophilia is a rare hereditary disorder in which the ability of patients’ blood to clot is impaired. As a result, the patient suffers from excessive bleeding and uncontrolled internal bleeding, leading to pain and eventual permanent damage to joints and muscles. There are two different forms of hemophilia, both generated by mutations to distinct genes. Hemophilia A results from mutations that impair the production of Factor VIII. Hemophilia B results from mutations that impair the production of Factor IX. It has been reported that even with “proper treatment” the life expectancy of hemophilia patients is about 10 years less than for individuals without hemophilia. Increasingly, the normal mode of treatment for younger patients is a prophylaxis regimen where patients are infused two or three times per week to maintain a better circulating level of coagulation factor. Long term studies in Sweden and elsewhere demonstrate that such regimens greatly reduce if not eliminate progressive joint deterioration. Market Opportunity Both disorders occur predominantly in males with Hemophilia A affecting about 14,500 people and Hemophilia B affecting about 3,600 people in the U.S. Today, most patients in the U.S., Western Europe, and Japan are treated with recombinantly produced Factor VIII and Factor IX drugs. The worldwide market for hemophilia drugs exceeded $3.8 billion in 2004. More background information on hemophilia can be found on www.hemophilia.org and www.whf.org |
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