Wednesday, April 1, 2020

Guidelines To Write and Submit an Orphan Drug Application For A Rare Disease

March 17, 2010 by  
Filed under Featured Stories

While working on our orphan drug application for Cyclodextrin for the treatment of Niemann Pick Type C disease, I searched the Internet looking for examples of an actual orphan drug application that was filed with the U.S. Food and Drug Administration. Not surprisingly, I could not find any ‘real applications’ that could be used as […]