Creating Hope Act 2010 Bill Would Extend Priority Review Voucher System To Rare Pediatric Diseases
August 9, 2010 by Chris Hempel
Filed under Featured Stories
Great news for the pediatric rare disease community came out late last week — rare disease advocates please get this out on your blogs! Senators Sam Brownback (R-KS), Sherrod Brown (D-OH), and Al Franken (D-MN) are supporting the bipartisan bill S. 3697, the “Creating Hope Act of 2010.” Nancy Goodman, Executive Director of Kids v […]
FDA Grants Orphan Drug Status For Cyclodextrin Compound To Treat Fatal Genetic Cholesterol Disease
May 16, 2010 by Chris Hempel
Filed under Featured Stories
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 […]
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 […]