Important note: Information in this article was accurate in 1991. The state of the art may have changed since the publication date.
Food & Drug Administration, U.S. Department of Health and Human Services - January 3, 1991
Pat Dorsey--(301) 443-3285
Hemophilia B, also known as Christmas Disease, is incurable and affects approximately 2,800 Americans. It can be managed through administration of the deficient clotting factor during bleeding episodes to prevent the loss of large amounts of blood. The name Christmas Disease comes from the surname Christmas -- the family in which the disease was first studied in detail and identified as a hereditary disorder in 1952.
Currently, patients with factor IX deficiencies are treated with a less purified type of clotting preparation known as factor IX complex. But it contains various impurities and can cause very serious complications, such as pulmonary embolism, due to excessive blood clotting.
FDA Commissioner David Kessler, M.D., said, "Because hemophilia B is a rare disease, this product has so far been studied only in a small number of people. It is hoped that the new, more purified, product will not cause these complications. The manufacturer will perform a post approval study to further examine the safety of the product."
Coagulation factor IX will be marketed as a dry concentrate, to be reconstituted with sterile water and administered intravenously. It can be used in the treatment and prevention of bleeding episodes including those caused by accidental cuts, abrasions and bruising. It can also be administered prophylactically to prevent uncontrolled hemorrhage in hemophilia patients undergoing surgery.
Donors of plasma used in the production of coagulation factor IX are tested for viral infections, and the product is heat treated at 60 degrees centigrade for 20 hours during manufacture to further reduce the possibility of disease transmission.
In clinical trials of the new product, a total of l1 patients received ll5 infusions of the new factor IX, and no adverse reactions were reported. In one trial to demonstrate efficacy in providing protection against bleeding, six hemophilia patients undergoing surgery received between 8,ll0 and 35,350 units of the product in an average of l7 infusions. Results of this study showed no post-surgery bleeding.
Coagulation Factor IX (Human) was developed and will be marketed by Alpha Therapeutics Corp. of Los Angeles, Calif.
910103
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SOURCE: Food and Drug Administration (FDA).
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