News
02/06/1989

<TITLE>Pentamidine </TITLE>
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Pentamidine 
P89-3 
FOR IMMEDIATE RELEASE               Brad Stone (FDA)/ Elaine Baldwin (NIAID)
Feb. 6, 1989                        (301) 443-3285    (301) 496-5717

    The Food and Drug Administration today granted permission for the 
expanded distribution of an experimental, aerosolized version of the drug 
pentamidine to help prevent Pneumocystis carinii pneumonia, a potentially 
life-threatening infection that often afflicts AIDS patients. 
    Under the conditions of the FDA-approved protocol, the drug will be 
recommended for use in AIDS virus-infected individuals who have had at least
one episode of the pneumonia or who have T4 helper cell counts of 200 per 
cubic millimeter or less.  T4 helper cells are white blood cells that are 
critical components of the body's immune system which are destroyed by the
AIDS virus.  Healthy individuals normally have T4 helper cell counts of 
1,000 or more.
    The drug should be administered to patients through the Respirgard II 
nebulizer and at a recommended effective dosage of 300 milligrams every four
weeks, based on recently available clinical data from the comparative dose
trial conducted by the San Francisco Community Consortium, a group of 
physicians with experience treating people with AIDS.  In this study, headed
by Bruce Montgomery, M.D., David W. Feigel, M.D., M.P.H., and Gifford 
Leoung, M.D., individuals infected with the AIDS virus who were at high risk
of developing the pneumonia had a lower incidence of infection when treated 
                                   


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with this dose than similar patients treated with lower doses.  This regimen
is different from that used by many physicians who have tried this therapy
on individual patients.  This is the first treatment IND emanating from a 
clinical trial sponsored jointly by a community research initiative and a 
pharmaceutical company. 
    In addition, an epidemiologic study supported by the National Institute 
of Allergy and Infectious Diseases helped to determine the patient
population that might benefit the most from the use of aerosolized
pentamidine.
    Injectable pentamidine, as distinct from aerosolized pentamidine, was 
approved in 1984 for the treatment of those already suffering from
Pneumocystis carinii pneumonia. 
    FDA's announcement will make the aerosolized product available under the
agency's "treatment IND" regulations -- a plan for the use of an
investigational new drug (IND) in selected patients facing serious or 
life-threatening conditions.
    FDA Commissioner Frank E. Young, M.D., Ph.D., estimated that perhaps
50,000 AIDS patients would benefit from this treatment IND. 
    Dr Young added, "Thanks to the work of the San Francisco Community
Consortium and the NIAID, we now have a much better understanding of how
aerosolized pentamidine might be used.  The work also showed that in many 
cases the cost of prophylaxis represents only a fraction of the cost of 
treating patients with Pneumocystis carinii pneumonia." 
    The consortium initiated the clinical study of AIDS-infected individuals
at high risk for developing Pneumocystis carinii pneumonia in 1987. 
    The clinical study compared the safety and efficacy of aerosolized
pentamidine at 30 mg every two weeks, 150 mg every two weeks, or 300 mg 
every four weeks, and showed effective results with the last regimen.  This 
                                   



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did not provide complete protection, but did demonstrate the ability to 
significantly reduce the incidence of this pneumonia. 
    A large, long-term epidemiologic study undertaken by NIAID showed that a
significant percentage of persons who have been infected with the AIDS virus
and who have T4 helper cell counts of 200 or less are at risk of developing 
Pneumocystis carinii pneumonia even if they have no symptoms of HIV 
infection.
    Anthony S. Fauci, M.D., NIAID director, said, "NIAID is sharing these 
important study results before scientific publication to speed patients'
access to this 'state of the art' therapy.  We believe that aerosolized 
pentamidine will help to prevent recurrent episodes of Pneumocystis carinii 
pneumonia and also help prevent initial occurrences of the infection."
    In addition to sponsoring this epidemiologic study, NIAID through its 
AIDS Program provided valuable technical assistance in formulating and
developing of the treatment IND protocol. 
    Aerosolized pentamidine can provoke severe wheezing and coughing.  The
long term risks associated with aerosolized pentamidine are unknown.
    LyphoMed Inc. of Rosemont, Ill., which markets pentamidine, partially 
underwrote the San Francisco Community Consortium study and will sponsor the
treatment IND protocol.  The company has also filed a new drug application
with FDA seeking marketing approval for this version of the drug. 
Aerosolized pentamidine is the third AIDS-related therapy to have received
"treatment IND" status under new FDA regulations. 
    Physicians interested in learning about the details of this protocol can
contact a hotline set up by the company at 1-800-PCP-7003.
    The Respirgard II nebulizer, a filtered system used in the studies, is
manufactured by the Marquest Corp. of Engelwood, Colo.
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