The Food and Drug Administration counts as one of its greatest regulatory triumphs its refusal to allow the sale of thalidomide, the antinausea drug that went on to cause birth defects in more than 100 children in countries where its sale was allowed.
That was in 1960. Now the FDA is considering a new proposal to sell the drug in the U.S.
An FDA panel has scheduled an unusual meeting next week to discuss an application by Celgene Corp. seeking approval of thalidomide for treating leprosy. Celgene is also conducting studies of thalidomide for treating AIDS wasting, a loss of weight and muscle that is a common cause of death in AIDS patients.
Another company, Andrulis Corp., is planning to seek approval to use thalidomide in treating canker sores in AIDS patients.
The question is whether the companies and the FDA can prevent the deformities seen decades ago. The consensus seems to be no, that some accidents will happen.
Still, says Dr. Debra Birnkrant of the FDA, "Clearly if a woman has AIDS and aphthous ulceration [canker sores] then ethically we cannot deny her this product."
"The question is how do you distribute a drug that is a known teratogen — meaning it causes birth defects — while assuring access to people who need it," says Sol Barer, president and chief operating officer at Celgene. "I don't think the answer is to prohibit the use of the drug
to women of childbearing years."
The issue is causing some distress, however, among victims of thalidomide.
"The last thing we wanted was to deal with a drug like thalidomide," says Randolph Warren, chief executive and cofounder of the Thalidomide Victims Association of Canada. Along with other victims, the 36-year-old Mr. Warren, whose mother was given the drug while in Germany, has fought hard to keep the public informed about the drug's dangers. The association is concerned that as thalidomide is approved for more purposes, the chances grow that it will be taken for uses for which it is not approved.
"We are forced to see regulated thalidomide as opposed to unlicensed availability," he says. "We are not advocating its use [but] we can't deny this to people who can have their lives extended."
In Europe, 125 cases of birth deformity were reported in connection with the drug. Some infants had no legs at all, just toes sprouting from their hips, along with foreshortened, flipper-like arms. And, although it was never approved in the U.S., 17 cases of birth defects were reported in this country, where some people received it as a sample.
The victims group wants the FDA to ensure that patients will not have automatic refills of the drug. And Mr. Warren says the group has Celgene's word that anytime the drug is sold under a brand name, the word thalidomide will also appear.
Dr. Birnkrant, who heads the FDA's Thalidomide Working Group, says, "We do know a lot about thalidomide and know when children are exposed to structural damage." One reason for the large number of birth-defect cases is that thalidomide was prescribed to pregnant women specifically to combat the nausea of morning sickness in the first trimester.
"We have a better understanding about how and when to avoid use," says Dr. Birnkrant, "but that is not to say we won't see other side effects if pregnancies are exposed later in gestation. We don't know."
Dr. Barer believes the answer is to make sure women know the risks and use birth control, and that doctors don't issue automatic refills. The FDA panel next week will attempt to answer the question of how to help women taking the drug prevent pregnancies.
Meanwhile, the National Institutes of Health has scheduled a meeting for Sept. 9 and 10 to publicly address the potential benefits and risks of thalidomide.
The goal of the thalidomide working group is to develop and implement uniform safety-monitoring procedures for all patients, Dr. Birnkrant says. Officials will also set up
an accounting system of all patients receiving the drug so they can be followed in the future.
Thalidomide is currently available to a variety of patients on an investigational basis. The patients in those studies, male and female, must use at least two forms of birth
control, FDA officials say, and routine pregnancy tests are given to women.
Celgene wants to market thalidomide to treat erythema nodosum leprosum, or ENL, in leprosy patients. Though rare in the U.S., leprosy afflicts 2.4 million people world-wide. About 30% of them suffer from ENL, which is characterized by acute inflammation, fever and loss of appetite. The AIDS wasting population is estimated at 80,000, and Celgene is hoping to capture 20,000 of that population, analysts say. Some analysts say Celgene will sell the drug at $3,000 per person per year at the lowest dosage rate of 100 milligrams per day, but other analysts are unsure of the pricing.
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