WASHINGTON — Saying that "the face of AIDS is changing," federal health officials Monday announced marketing approval for a drug to treat an AIDS-related condition that often results in blindness and announced expanded pre-market distribution of a second drug that is effective in preventing serious anemia associated with the deadly disease.
A cure for AIDS remains doubtful, and the disease is some years from being controlled like such chronic diseases as hypertension or diabetes. But these new drugs and others have moved diagnosis of the disease, or of AIDS infection, away from being "a death sentence," said Dr. Frank E. Young, commissioner of the Food and Drug Administration.
Because life can be prolonged and its quality improved, there is now more impetus for individuals who are at risk of developing AIDS to find out if they are infected and "treat the disease early," he said.
"This is really good news," Young said at a press conference. "It indicates that AIDS is moving to a new stage. Even without a cure, this gives hope to people suffering from this disease. . . . Since (AIDS) was first seen in 1981, we've come a long, long way."
Health and Human Services Secretary Louis W. Sullivan said that "this and other recent developments in AIDS treatment demonstrate a commitment . . . to speed the availability of AIDS-related drugs."
The FDA gave full marketing approval to ganciclovir, also known as DHPG, which helps stave off cytomegalovirus retinitis, an eye infection that frequently results in blindness. It already has been widely available under a special FDA classification known as a Treatment IND (for investigational new drug). A Treatment IND status permits extensive distribution of a drug while it is still being studied.
The agency said also that it will grant a Treatment IND to r-erythropoietin, a protein product that alleviates severe anemia, which otherwise requires frequent blood transfusions. It is expected to be especially valuable in treating the anemia that is a side effect of AZT and which has affected an estimated 10,000 AIDS patients now taking that drug.
Five Drugs Available
This brings to five the number of AIDS-related drugs that have been made available before marketing to AIDS patients or to those infected by the disease. These include the anti-viral drug AZT, which inhibits replication of the AIDS virus in the body and has been shown to extend life, and aerosol pentamidine, which, when used as a prophylaxis, significantly reduces the chances of developing pneumocystis carinii pneumonia, a life-threatening infection commonly associated with AIDS. Both AZT and aerosol pentamidine now have full market approval.
Young said that cytomegalovirus retinitis threatens the sight of nearly one in four AIDS patients. CMV is an extremely common virus that has infected about 60% of the adult population, but it is of little consequence to people with healthy immune systems.
Young admitted that the agency had "a lot of difficulty" evaluating the DHPG study data but eventually was convinced that the drug was effective as a short-term therapy in delaying the spread of CMV retinitis. FDA officials were persuaded, in part, by the "substantive statements" made by many physicians who had prescribed it for their patients and were convinced that it is effective, Young said.
"I would be less than candid if I did not note that the effects seen to date are mostly transitory," Young said. "Much remains to be known about the full impact of ganciclovir therapy, and particularly about its longer-term effects."
Also, he said, there are some additional concerns that as many as 40% of all AIDS patients with CMV retinitis "may be unable to tolerate ganciclovir" because of possible adverse effects on the production of red blood cells and that the drug may interact poorly with AZT and other drugs. Young said that the drug's manufacturer, Syntex Corp. of Palo Alto, Calif., has agreed to sponsor extensive post-marketing studies.
Ganciclovir, which will be sold under the trade name Cytovene, must be taken intravenously twice a day for two to three weeks, followed indefinitely by maintenance therapy of daily infusions.
The cost of administering the drug is expected to be prohibitively high. It can be administered in the home but requires a visiting nurse. Although most insurance probably will pay for the cost of the drug, it is not known whether the cost of its being administered also will be covered.
R-erythropoietin, taken six times a week by injection, is a form of a protein produced in the kidneys, erythropoietin, which stimulates the body's production of red blood cells. Scientists have made quantities of the protein for treatment through the use of gene-splicing techniques.
Another erythropoietin product was approved for marketing by the FDA on June 1 as a treatment for anemia associated with chronic kidney failure, but the FDA's action Monday approved use of the product for AIDS-related anemia only as an experimental regimen. Ortho Pharmaceutical Corp. of Raritan, N. J., said that it would provide the drug at no cost.