Moms Receive EU Orphan Drug Designation From European Medicines Agency For Novel Cholesterol Drug Called Cyclodextrin

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 […]

FDA Grants Orphan Drug Status For Cyclodextrin Compound To Treat Fatal Genetic Cholesterol Disease

Drum roll, please! Dr. Caroline Hastings at Children’s Hospital Oakland and Research Center received a call on Friday afternoon from the U.S. Food and Drug Administration regarding the orphan drug submission we made at the end of February. While we have not received the official letter by mail (probably next week), it appears our Orphan […]

FDA and EMA Forge Rare Disease Collaboration; Announcement Coincides with World Rare Disease Day 2010

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 […]