...

By 2000, there was both the Division of Blood Application and the Office of Blood Research and Review at the FDA’s Center for Biologics Evaluation and Research (CBER).

Blood and Blood Products:  Hemophilia

An early blood research project in the FDA Bureau of Biologics concerned provisioning increased supplies of antihemophilic factor through two sources, antihemophilic factor concentrate (AHF) and cryoprecipitated antihemophilic factor (CAF).  For example, staff evaluated the possibility of producing the latter from fresh frozen single donor plasma that was beyond its date by assaying samples of donor frozen plasma at different time periods.  The Bureau also contracted with the American Red Cross to investigate the harvestability of AHF from blood stored up to about a week.  These studies were ongoing.

Interest in developing improved assays for potency and safety for products used in blood clotting diseases led the Bureau in the mid-1970s to launch a study into purification and other elements associated with blood components that play a role in clotting, particularly Factor IX (associated with Christmas disease), the clinical use of which had been problematical. An animal model was identified to help in the observation of in vivo clotting characteristics, and in vitro observations were facilitated through electron microscopy.  

Data from the early 1980s indicated that nearly a third of those with hemophilia who die have severe liver disease, either as the cause of death or a contributing factor. So, the approval of Travenol Laboratories’ heat-treated Factor VIII (the antihemophilic factor) in 1983 was an especially important move for those with this disease.  Animal models used by the firm indicated that heat decreased the infectivity of Hepatitis B and eliminated non-A, non-B Hepatitis. 

The Center for Biologics Evaluation and Research (CBER) licensed Monoclate late in 1987, the first blood product purified by biotechnological techniques, an antihemophilia factor. This was produced from normal human plasma with monoclonal antibodies to extract Factor VIII:C from plasma. This treated hemophilia A, which affects more than 20,000 patients at that time.
The development and licensing of recombinant Factor VIII in 1992 and 1993 eliminated the possibility that the nation’s 10,000 suffering from hemophilia A would have to face the possibility of the transmission of hepatitis or AIDS. Recombinate was the first, licensed in Dec. 1992, and Kogenate received its license in the following February.