Moms Receive EU Orphan Drug Designation From European Medicines Agency For Novel Cholesterol Drug Called Cyclodextrin
September 14, 2011 by Chris Hempel
Filed under Featured Stories
Last year, after Dr. Caroline Hastings and I received the U.S. Food and Drug Administration orphan drug designation for Hydroxy-propyl-beta-cyclodextrin (HPBCD) for the treatment of Niemann Pick Type C disease, I was contacted by Sue French. Sue lives in the United Kingdom and her son William is also afflicted with the rare and fatal genetic […]
Guidelines To Write and Submit an Orphan Drug Application For A Rare Disease
March 17, 2010 by Chris Hempel
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 […]
FDA and EMA Forge Rare Disease Collaboration; Announcement Coincides with World Rare Disease Day 2010
February 28, 2010 by Chris Hempel
Filed under Featured Stories, Health Care Policy
In recognition of World Rare Disease Day 2010, the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) announced that they will collaborate together and now accept a single annual report from sponsors with an orphan drug designation for rare medical conditions. The joint announcement was made by Dr. Timothy Cote, director […]