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The Food and Drug Administration approved a new recombinant DNA-derived clotting factor for people with hemophilia B, a rare congenital blood disorder. The product, Coagulation Factor IX (Recombinant) is intended for the prevention and control of excessive, potentially life-threatening bleeding in hemophilia B patients, including those who require surgery.
Hemophilia B, also known as Christmas Disease, affects approximately 3,000 Americans who are unable to form blood clots adequately because of a deficiency or defect in clotting factor IX. Treatment with factor IX products, given by intravenous infusion, corrects the defect temporarily. Highly purified factor IX products obtained from pooled human plasma are also available for people with hemopilia B.
This new purified recombinant product is free from the risk of transmitting plasma-derived human viruses because it is produced in Chinese hamster ovary cells that have been modified to express the gene for human factor IX.
Clinical trials assessing the safety and efficacy of this recombinant factor IX product include a trial with 36 patients who had previously received plasma-derived products. The trials indicated that higher doses of the recombinant product may be needed in some patients, as compared to plasma-derived products. The product was also studied in 12 surgical patients.
Few data are available regarding the rare, serious side- effects that can occur with any factor IX product. These side- effects include antibody responses that neutralize the effects of factor IX, and excessive blood clotting.
In clinical trials of this recombinant factor IX product, one patient developed low levels of neutralizing antibodies that later disappeared. Another patient developed a renal infarct, or damage to the kidney, that may have been related to a blood clot.
Information regarding the use of this product in previously untreated patients is limited. Therefore, a clinical trial involving up to 50 such patients for up to 5 years is ongoing. The sponsor will also follow previously treated patients for two years.
Coagulation factor IX (recombinant) is manufactured by Genetics Institute, Cambridge Mass, and will be marketed as BeneFix.
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