Thalidomide, once the world’s most notorious drug, was recommended by a federal panel Friday for sales at American pharmacies with tight restrictions aimed at avoiding the birth-defect horrors of the 1960s.
The scientific panel urged approval by the Food and Drug Administration just for a small number of leprosy patients. But doctors are expected to prescribe it for a wider range of conditions, and all sides agreed that accidents are inevitable.
Even one of the world’s top leprosy physicians, Dr. Thomas Rea of Los Angeles County Medical Center, told the FDA’s advisory panel that “one day a thalidomide baby will be born” because of the approval.
“I don’t know what I will do when I look that child in the eye,” Rea said.
If the FDA follows the panel’s advice, thalidomide would become the most severely restricted drug ever sold in this country.
Although banned worldwide 35 years ago, the drug was never sold in the United States because an FDA scientist uncovered early signs of toxicity and blocked approval. Still, some Americans got it overseas or in clinical trials.
Today, said the agency’s Dr. Murray Lumpkin, experts know the risks and the drug is being studied as a medicine to treat only devastating diseases that leave their victims little hope.
Americans with leprosy are very rare - less than 50 a year are diagnosed with the agonizing inflammation the drug targets - and most of them are in special research programs that already allow them to get experimental doses.
But the drug also is being studied for a host of diseases from AIDS-related wasting and ulcers to cancer, lupus and rheumatoid arthritis.
That means doctors probably will prescribe it to many more people than leprosy patients, the FDA panel acknowledged. But the scientists said thalidomide would still pose a danger without approval because it is sold underground, sometimes via the Internet, and smuggled from abroad with no government oversight.
Instead, the FDA panel said the government should set unprecedented curbs on which pharmacies can distribute thalidomide and force any company that makes the drug to educate physicians about the dangers.
A photo of a child thalidomide victim - ideally a videotape of such a child - should be shown to every doctor who prescribes thalidomide and every patient who gets it, the panel said.
In addition, the panel endorsed plans by New Jersey-based Celgene Corp., which hopes to become the first of three competitors to sell thalidomide, that would forbid pharmacies from distributing thalidomide until they receive a form signed by patient and doctor that both know the risks, and that a female patient is adequately protected against pregnancy.
The FDA is not bound by advisory committee decisions but typically follows them.
xxxx Notorious drug Thalidomide was banned worldwide 35 years ago after causing horrific birth defects in 12,000 babies. At that time, it was sold as a sleeping pill and morning sickness cure in 48 countries. No one had known that just one pill in early pregnancy could result in a baby with no limbs or with flipper like arms and legs.