WASHINGTON, D.C. - Thalidomide, the drug whose use nearly four decades ago produced thousands of babies with birth defects in Europe and Canada, was approved by the Food and Drug Administration (FDA) for use in treating patients suffering from erythema nodosum leprosum (ENL), or leprosy.
The decision came after an FDA advisory panel recommended last fall that the controversial drug be approved for use in leprosy patients.
As part of the approval, patients will be limited to 30-day supplies of the drug to prevent it from finding its way onto the black market. The company receiving the FDA approval, Celgene Corp., said it is in the midst of concluding several clinical trials and expect to file two more applications with the FDA for thalidomide use in AIDS patients - to reduce AIDS wasting and for treating painful AIDS-related mouth ulcers. Thalidomide works by inhibiting tumor necrosis factor-alpha production and affecting key components of the immune response.
Data presented to panel members resulted from a study conducted several years ago by the company and showed that 80% of leprosy patients in one double-blind, controlled clinical trial reacted favorably to the drug.
To ensure the drug does not cause similar problems as those seen in the 1950s and 1960s, Celgene officials have created a the System for Thalidomide Education and Prevention Safety (STEPS) program where doctors can council patients on the proper use of the drug. Doctors or pharmacists expressing interest in dispensing the drug must register with the company, read the materials sent to them on the potential dangers inherent in prescribing the drug, and follow the program to the letter, said Jerome Zeldis, MD, PhD, vice president of medical affairs at Celgene.
Female patient prescriptions will not be filled without a doctor's confirmation that the woman tested negative for pregnancy. These women will also have to be tested for pregnancy, beginning on a weekly basis then monthly, while on the drug.
"No fetus should ever be exposed to thalidomide," he said. "Women who are of child-bearing age [and taking thalidomide], should be using two forms of birth control: a highly effective method (hormonal) and an effective means (barrier)."
Thalidomide once sold in 48 countries for insomnia and morning sickness. It was banned in 1962 after some 12,000 babies were born with no limbs and tiny, flipper-like arms and legs, serious facial deformities and defective organs. Though it was never sold in the United States, some Americans did get it abroad or in research trials.
The drug has several adverse effects. It has been known to cause peripheral neropathy, somnolence and constipation, allergies and mild neutropenia. When thalidomide is used to treat patients taking corticosteroids to manage ENL, the steroids can often be discontinued. The American Academy of Pediatrics came out against the drug when it was first being considered, calling it a "notorious teratogen."
About 2.4 million people have ENL worldwide. About 7,000 Americans, mostly recent immigrants, have leprosy and about 200 new cases are reported each year.
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