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Patient Advocacy: Foundations Receiving Orphan Drug Designations

May 20, 2014


This is the second Blog Post in a series that examines the influence and power of patient advocacy groups, foundations, and alliances in helping and influencing the different stages of drug development in the rare disease and orphan drug space.

This Blog Post presents a patient advocacy organization as an example of patient families establishing a not-for-profit, leading the way for funding research, and receiving a FDA Orphan Drug Designation (ODD) for treatment of a rare disease. The chart below identifies a drug that receives a FDA ODD for a sponsor that is an advocacy group and not the usual pharmaceutical company :

Generic Name/Trade Name Sponsor ODD Date Indication
Bezafibrate/ Bezalip Barth Syndrome Foundation 07.24.13 Barth Syndrome


The Barth Syndrome Foundation (BSF) is created in 2000 by families from around the world with the purpose of advocating for treatments and a cure for the rare disease, Barth Syndrome (BTHS). BTHS is a rare, X-linked genetic disorder of lipid metabolism that primarily affects males across different ethnicities. There is currently no specific treatment for BTHS.

BSF files for FDA ODD in May 2013 for the drug Bezalip (Bezafibrate) for BTHS. Two months later in July 2013, BSF receives FDA ODD for the drug for the therapeutic treatment of BTHS. Bezalip is a drug that has been used for over 25 years to treat high triglyceride levels. This is an example of drug repositioning or drug repurposing – application of existing drugs to new indications or diseases. Per the most recent BSF Newsletter:

“ … This achievement (FDA ODD) allows BSF and researchers to focus on studying this drug to determine if it is safe and effective for Barth syndrome individuals. The next step in this process is to file an Investigational New Drug (IND) application with the FDA … This lengthy and ongoing process has only been possible through the outstanding help of: the SMARTT group at the National Heart, Lung, and Blood Institute (NHLBI), the license holder of bezafibrate (Tribute Pharmaceuticals), and the healthcare professionals who are planning and will perform the clinical study (Drs. Todd Cade and Dominic Reeds)… these three independent groups have come together with BSF to test the first drug specifically identified for treating Barth syndrome individuals.

Please Note: “Kid Hugging a Rainbow” by Marendo Müller, artwork (Own work) [Public domain] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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