FDA
TALK PAPER

Food and Drug Administration
U.S. Department of Health and Human Services
Public Health Service 5600 Fishers Lane Rockville, MD 20857
FDA Talk Papers are prepared by the Press Office to guide FDA personnel in responding with consistency and accuracy to questions from the public on subjects of current interest. Talk Papers are subject to change as more information becomes available. 
T98-44                      Ivy Fleischer Kupec:   301-827-6242
July 16, 1998               
                            Consumer Hotline:      800-532-4440

FDA APPROVES THALIDOMIDE FOR HANSEN'S DISEASE SIDE EFFECT, IMPOSES UNPRECEDENTED RESTRICTIONS ON DISTRIBUTION

FDA today cleared thalidomide for marketing as a treatment for erythema nodosum leprosum (ENL), a serious inflammatory condition in patients with Hansen's disease (also known as leprosy) while at the same time it imposed unprecedented restrictions on the drug's distribution. Because of its well-known potential for causing birth defects, thalidomide will be among the most tightly restricted drugs ever to be marketed in the United States. The following may be used to answer questions:

In September 1997, FDA's Dermatologic and Dental Drugs Advisory Committee recommended thalidomide's approval based on an analysis of the totality of available data, including historical data based on the experience of the Public Health Service and published literature, that demonstrated thalidomide's effect in treating the skin lesions of ENL for more than 30 years.

At least 70-80 percent of patients with ENL responded to thalidomide therapy with improvements in skin lesions as assessed in double blind, controlled clinical trials, compared to approximately 25 percent of patients given placebo.

Thalidomide is a drug originally marketed as a sedative outside the United States from the 1950s until the early 1960s when it was linked with severe birth defects. In addition to causing serious fetal deformities, thalidomide has been associated with other adverse reactions, including peripheral neuropathy, a disorder that, in some patients, has resulted in permanent nerve damage. The product was never approved for use in the United States until now.

To prevent fetal exposure to thalidomide, the drug's manufacturer, Celgene, has developed the System for Thalidomide Education and Prescribing Safety (STEPS) program. Only physicians who are registered in the STEPS program may prescribe thalidomide to patients, and those patients -- both female and male -- must comply with mandatory contraceptive measures, patient registration and patient surveys.

Additionally, female patients' prescriptions will not be filled without a physician's written report of a negative pregnancy test that has been conducted within 24 hours of starting thalidomide therapy. Pregnancy testing will continue to be required weekly during the first month of use, then monthly thereafter in women with regular cycles, or every two weeks if cycles are irregular. Prescriptions are only for one month's supply. Women must use two reliable forms of contraception simultaneously while taking the drug.

Male patients will receive written and oral warnings of the risk of possible contraception failure and of the need to use condoms when having intercourse with women of childbearing age. It is unknown whether thalidomide is present in sperm or semen or whether its presence in sperm or semen would affect fetal development.

While birth defects are the most widely known side effect of thalidomide, this drug also can cause a potentially irreversible kind of nerve damage known as peripheral neuropathy, particularly when it is used chronically over a period of months. More information is needed to determine how dose and use of the drug affects onset of this side effect and irreversibility.

Additionally, thalidomide is known commonly to cause drowsiness and dizziness.

Celgene Corporation of Warren, N.J., will market thalidomide as Thalomid.

For more information on thalidomide, go to FDA/CDER Thalidomide Information on this Website.

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