Abstract
ABSTRACT EIGHTY-FIVE companies have 133 drugs in various stages of development for 96 rare diseases.This is the first comprehensive listing of orphan drugs since the Orphan Drug Act became law in 1983. The list was compiled by the Pharmaceutical Manufacturers Association in response to a request by the National Commission on Orphan Diseases (JAMA. 1989;261: 2938).More Than One Disease Some drugs are being studied for more than one rare disease. Of the 299 orphan drug designations granted by the Food and Drug Administration, 172 are in development and 39 designations involving 36 drugs have been approved.The 88 remaining designations are in early preclinical trial, are diagnostic agents, or are no longer under active investigation, says Gerald J. Mossinghoff, Pharmaceutical Manufacturers Association president. There are 64 research projects in the final stages of development and 40 of these are currently being reviewed for approval by the Food and Drug
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