NEWS
12/10/1992

<TITLE>The licensing of the first recombinant DNA-derived clotting factor</TITLE>
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P92-39                                          Food and Drug Administration
FOR IMMEDIATE RELEASE                           Monica Revelle (301) 443-4177

    The Food and Drug Administration today announced the licensing of the     
first recombinant DNA-derived clotting factor for use in persons with         
hemophilia A.  The product, antihemophilic factor (recombinant), is intended  
for the prevention and control of excessive bleeding and for people with      
hemophilia A who require surgery.
    Hemophilia A is an inherited disorder, sometimes called classic           
hemophilia, in which the blood clotting protein factor VIII is deficient or   
missing.  Affected persons are unable to form blood clots adequately and,     
therefore, risk serious and life-threatening bleeding.  Replacement therapy   
with factor VIII concentrate corrects the defect temporarily but must be      
given by intravenous infusion, in many cases daily or more often.
    Prior to the licensure of antihemophilic factor (recombinant), factor     
VIII concentrates made from human plasma have been relied upon.  To obtain    
sufficient quantities of factor VIII, plasma pools of thousands of donations  
were used.  
    In the past, some factor VIII made from such pools transmitted hepatitis  
and AIDS viruses to patients, but all anti-hemophilic factors currently       
licensed in the United States are now manufactured in ways that are believed  
to eliminate the risk of transmission of these viruses.
    "The production of factor VIII by recombinant DNA technology eliminates   
even the theoretical possibility of the transmission of viruses from          
plasma," said FDA Commissioner David A. Kessler, M.D.  "Its approval is a     
milestone in the history of treatment of hemophilia."
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                                                  Page 2, P92-39, Factor VIII
    Antihemophilic factor (recombinant) is produced by Chinese hamster ovary  
cells that have been modified by recombinant DNA techniques to introduce and  
express the gene for human factor VIII.  The antihemophilic factor            
(recombinant) is highly purified by several steps involving biotechnology     
processes.
    Studies of antihemophilic factor (recombinant) were performed both in     
persons who had been exposed previously to clotting factor concentrates or    
other blood products and those who had not.  The trials demonstrated          
pharmacokinetic equivalence to plasma-derived factor VIII and showed that     
antihemophilic factor (recombinant) is safe and effective when used           
prophylactically or therapeutically in persons with hemophilia A.
    In December 1991, FDA's Blood Products Advisory Committee recommended     
approval of antihemophilic factor (recombinant).  However, the committee      
requested that additional clinical trials be conducted in previously          
untreated patients because antibodies that inhibited the activity of factor   
VIII were observed in some persons.  At this time, it does not appear that    
the antibodies are more of a problem with antihemophilic factor               
(recombinant) than with plasma-derived factor VIII, but more data are         
needed.  Such data are being collected from persons who have not previously   
received factor VIII.
    Antihemophilic factor (recombinant) is manufactured under a shared        
manufacturing agreement between Genetics Institute, Inc., Cambridge, Mass.,   
and Baxter Healthcare Corporation, Glendale, Calif.  Genetics Institute will  
make the bulk product, and Baxter Healthcare Corporation will manufacture     
the final dosage form.  It will be sold under the brand name Recombinate.
                                   -MORE-


                                            Page 3. P92-39, Factor VIII
Antihemophilic factor (recombinant) was approved on the same day in both the  
United States and Canada, through collaborative efforts of FDA, the Canadian  
Bureau of Biologics and Baxter.
    FDA is a Public Health Service agency within HHS.
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