Thalidomide, the drug whose use in the 1960s produced thousands of babies with flipperlike arms and other gross deformities, is on the verge of receiving approval from the Food and Drug Administration, nearly four decades after the agency's legendary decision to reject it for use in the United States.
FDA officials announced Monday that they intended to approve thalidomide for use in leprosy patients, as long as the New Jersey-based company seeking to market it could adhere to several conditions, including elaborate restrictions designed to keep the drug away from women who are or might become pregnant.But the potential benefits of thalidomide are vast, and experts expect doctors to quickly begin prescribing it for "off-label" uses. In recent years, thalidomide has been experiencing a revival of sorts, and scientists are experimenting with it for maladies as diverse as AIDS, brain cancer, lupus and other autoimmune diseases. Advocates for people with these conditions have been urging the agency to make it available.
"The company has met its scientific obligation in showing that the benefits outweigh the risks" in treating leprosy, said Dr. Murray Lumpkin, deputy director of the FDA's Center for Drug Evaluation and Research. "The issue is how can you manage the down-sides of this drug? That is the challenge."
Lumpkin could not say how soon the drug might be approved for use. But an official with Celgene Corp., the company seeking the approval, said he expected that Celgene would be able to satisfy the FDA's conditions, which were outlined in a letter sent to the company on Friday, in a matter of weeks. The official, Bruce Williams, said the company had already produced enough of the drug to market it immediately after approval. "When we agree on the final labeling, then we'll be ready to roll," he said.
News of the letter to Celgene left surviving thalidomide victims saddened, but not surprised. "This is not a happy day," said Randy Warren, who was born without hips after his mother took the drug and who now leads the Thalidomide Victims Association of Canada. "But it is certainly not a day we didn't expect. It's another stop on the journey of tears."
Health experts said approval of the drug, especially if it was used for a variety of conditions, would almost certainly lead to the births of babies with defects seen in countries where thalidomide was marketed as a sedative in the 1960s.
"Thalidomide is becoming a wonder drug," said Dr. Norman Fost, an ethicist and pediatrician at the University of Wisconsin. "Once it is approved by the FDA, there is nothing to stop doctors from prescribing it for other things."