Approval of AZT

Date: March 20, 1987
Source: Department of Health and Human Services (DHHS)
Author: Public Health Service (PHS)

Robert E. Windom, M.D., assistant secretary for health, today announced that the Food and Drug Administration has approved the drug zidovudine, commonly known as azidothymidine, or AZT, to help certain patients with Acquired Immunodeficiency Syndrome (AIDS) and advanced AIDS-Related Complex (ARC).

The drug will be marketed by Burroughs Wellcome Co. of Research Triangle Park, N.C., under the trade name Retrovir.

Retrovir is the first approved treatment of AIDS. Because of limited supply of the drug, distribution will be restricted initially by the company to those patients for whom it is indicated under the approved labeling, including AIDS and ARC patients with specific laboratory evidence of severely depressed immunity or a history of Pneumocystis carinii pneumonia (PCP).

Today's approval marks an important step, but by no means a final victory, in our ongoing war against AIDS," Dr. Windom said. "Retrovir is not a cure for AIDS, but it has a demonstrated ability to improve the short-term survival of AIDS patients with recently diagnosed PCP and certain patients with advanced ARC."

He noted that available clinical data were sufficient for approving the use of Retrovir only for certain indications, and not for all AIDS-associated conditions. "Nevertheless," Dr. Windom said, "today's action means that significant medical relief will be available to thousands of those afflicted with this dreaded disease."

Current data," Dr. Windom said, "indicate that about 32,000 persons have been afflicted with AIDS in the United States, with about 14,000 of them still living. Most of them, particularly those who have had a serious opportunistic infection associated with AIDS, are expected to qualify for Retrovir treatment under the approved indications. Certain patients with advanced ARC, a condition that frequently precedes and develops into AIDS within a short period of time, have also been shown to benefit from therapy with Retrovir."

Advanced ARC patients have symptoms that include weight loss, persistent fever and diarrhea, and less severe opportunistic infections such as oral candida and herpes infections. They also have a markedly reduced number of T-helper lymphocytes, critical elements of the immune system which are destroyed by the AIDS virus. It is estimated that about two to three time as many Americans may suffer from advanced ARC as suffer from AIDS.

Retrovir was originally developed in 1964 by Dr. Jerome Horowitz of the Michigan Cancer Foundation as a possible treatment for cancer. In February 1985, the National Cancer Institute, under the direction of Dr. Samuel Broder, tested AZT and found that it was a potent inhibitor of AIDS.

Dr. Windom said, "the work of Dr. Horowitz, Dr. Broder, NCI and Burroughs Wellcome should be commended. In addition, we must salute Dr. Frank Young, Commissioner of food and drugs and his staff for their speedy and thorough action in approving this drug."

Dr. Windom noted that under FDA's 1-AA priority review designation for AIDS drugs, the agency's review and approval of the new drug application for Retrovir was accomplished within less than four months -- one of the shortest approval actions on record. The 1-AA designation, which gives AIDS drugs top priority for review within the agency, is an extension of the official 1-A, 1-B, 1-C classification system FDA uses to prioritize the review of drugs within the review process and exemplifies FDA's effort to produce more timely and efficient reviews for all breakthrough drugs.

"Today's action is an important demonstration of FDA's ability to more swiftly, with impressive scientific precision, to review and approve promising treatments for AIDS, when such action is justified by sound and convincing clinical evidence," Dr. Windom concluded.