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 […]
FDAs Orphan Drug Workshop Featured in Wall Street Journal – Push To Cure Rare Diseases
March 10, 2010 by Chris Hempel
Filed under Featured Stories
Below is a story that ran in today’s Wall Street Journal (Page A 3) on the FDA workshop I attended a few week ago at the Keck Graduate Institute in Claremont, CA. We filed an orphan drug application for Cyclodextrin for the treatment of Niemann Pick Type C disease, an ultra rare cholesterol disease that […]
A True Healthcare Reform Idea – Priority Review Vouchers For Ultra Rare Diseases
February 16, 2010 by Chris Hempel
Filed under Featured Stories
This week I have been reading all about priority review vouchers. Henry Grabowski and other faculty at Duke University came up with the idea for providing an incentive for drugmakers to target certain tropical and infectious diseases (such as TB, malaria, cholera, dengue, leprosy) affecting poor nations. Senators Sam Brownback (R-KS) and Sherrod Brown (D-OH) […]
The Road to Building a New Orphan Drug – Am I Mad?
February 4, 2010 by Chris Hempel
Filed under Cyclodextrin
On February 25-26, 2010, I will be attending the inaugural FDA Build-an-Orphan-Drug-Workshop hosted by the Center for Rare Disease Therapies at Keck Graduate Institute (KGI) in Claremont, Calif. FDA agency experts will be on hand to provide guidance on applying for orphan drug designation. I will be attending the workshop with Dr. Ron Browne, hired […]