AIDS drug approval recommended.

AIDS drug approval recommended

A U.S. Food and Drug Administrationadvisory committee recommended last week that the FDA approve azidothymidine (AZT), a drug that, though not a cure, has been shown to prolong the lives of people with AIDS. If FDA Commissioner Frank E. Young accepts the committee's decision, as he is expected to do, AZT will become the first drug for the treatment of AIDS generally available in the United States.

The committee recommnded thatAZT be approved for AIDS patients with certain opportunistic infections and for people with AIDS-related complex (ARC), a set of AIDS-like symptoms that usually leas to AIDS itself. Once the drug is on the market, however, physicians can prescribe it for any patients they wish.

In addition to toxicologic data suppliedby the drug's maker, Burroughs Wellcome Co. of Research Triangle Park, N.C., the committee based its approval of two studies on AIDS victims with Pneumocystis carinii pneumonia and of people with ARC. In the first study, a double-blind trial that ended in September 1986, 1 of 145 people given the drug died within a six-month period, compared with 19 of 137 people who received a placebo (SN: 9/27/86, p.196). In the second study, which includes more people but is noncomparative, AZT has been made available to 3,247 people since September. Although the death rate has been lower than what would be expected in patients not getting the drug -- and, some clinicians say, opportunistic infections have been fewer and milder -- 97 people receiving AZT have died, including 21 who died more than three weeks after beginning treatment.

While recommending the approval ofAZT, committee members express concern about making it generally available. Among their worries:

* The drug is toxic, so patients must bemanaged carefully. In the initial trials, AZT has caused anemia and white blood cell depletion; transfusions and dosage interruptions or reductions were often necessary.

* People in whom the drug has not yetbettn proven effective, such as people who test positive for AIDS antibodies but have no signs of the syndrome and people who have AIDS-related Kaposi's sarcoma, will demand the drug. For the near future, however, Burroughs Wellcome expects to be able to make only enough AZT for the recommended group.

* Data are available only on a limitednumber of patients, who have been on the drug for a short period of time. AIDS patients are expected to need the drug on a long-term basis.

FDA's Ellen Cooper, who is in charge ofthe AZT application, told members of the committee at its open hearings that, compared with people in the control group, people receiving the drug showed a "highly significant difference in mortality and time until first infection." But, she added, more toxicologic testing on animals is needed, the optimal dose for humans still is not clear, and more human data are needed.

Several committe members suggestedsetting up a special category for the drug so that its use after approval could be controlled. But the FDA has no mechanism for instituting and enforcing such a system. Burroughs officials said they are working on a plan to control the distribution, so that doctors and patients would have to be registered to receive it. The drug, which the company is now calling Retrovir, is expected to cost several thousand dollars a year per patient.