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March 16, 1990

Investigational New Drug (US) 'Orphan' Trials Now Use Thalidomide From Two Sources

JAMA. 1990;263(11):1474. doi:10.1001/jama.1990.03440110028005

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FOOD AND DRUG Administration (FDA) officials classify thalidomide as an orphan drug, one of 133 such pharmaceuticals currently being studied in the treatment of 96 relatively rare diseases (JAMA. 1990;263:793).

Two such studies of thalidomide are under way. In lepromatous leprosy, it has been the drug of choice since 1966 in treating erythema nodosum leprosum and now is in phase III (extensive clinical trials in humans) developmental status. In graft-versus-host disease, the most common cause of death following bone marrow transplantation, it has been in clinical trials since 1987 and is in phase II (effectiveness testing in humans) developmental status. (Please see accompanying article.)

In the United States, thalidomide is obtained under investigational new drug status from two sources. The National Hansen's Disease Center, Carville, La, which is conducting the leprosy study, obtains reagent-grade thalidomide from Andrulis Research Corporation, Bethesda, Md.

Andrulis manufactures the drug under contract with the US