FDA Orphan Drug Workshop Makes Filing Applications Easy – Even For Rare Disease Patient Advocates!

Our FDA Orphan Drug Filing For Cyclodextrin To Treat Niemann Pick Type C Disease

At a historic orphan drug workshop held by the U.S. Food and Drug Administration Feb. 25-26 and before World Rare Disease Day 2010, we filed a momentous orphan drug application with the FDA for the treatment of Niemann Pick Type C disease with cyclodextrin. Our filing occurred in Claremont, CA, at the Keck Graduate Institute’s Center for Rare Disease Therapies, at 4pm on Friday in the Founder’s Room.

Typically rare disease drug applications are filed by pharmaceutical or biotech companies but our application was filed as patient advocates on behalf of the entire Niemann Pick Type C disease community worldwide. Children’s Hospital of Oakland Research Institute (CHORI) and Dr. Caroline Hastings, Addi and Cassi’s physician, are the official sponsors of the application.

Over the two day FDA workshop, Dr. Ron Browne (a medical consultant hired to helped compile the application over the past few months) and I met with FDA officials. We had four 30 minute meetings over two days to get advice on our filing and make sure we were following all proper procedures to give us the best chance at obtaining an orphan status designation for cyclodextrin. For those considering attending the FDA Orphan Drug Workshop, I would highly recommend it! The next one will be held at the University of Minnesota August 3-4, 2010.

We filled two binders (one original and one copy) with information on Hydroxy Propel Beta Cyclodextrin (HPBCD) for the treatment of Niemann Pick Type C Disease. Niemann Pick Type C disease is a very rare, relentlessly progressive and eventually fatal neurodegenerative genetic cholesterol disorder that afflicts my six year old twins. We made our official submission in hot pink binders (probably a first for the FDA!) as pink is Addi and Cassi’s favorite color.

Approximately 15 other organizations attending the FDA orphan drug workshop filed applications for new rare disease drugs or biologics. We all now wait 60 days to see if the FDAs review division grants us official orphan drug designations.

I learned at the FDA workshop that approximately 60-70% of the orphan drug applications submitted to the FDA result in the granting of orphan status.  The two main criteria that must be met to receive a designation as an orphan drug are:

1.    Prevalence (less than 200,000 in the US are affected with the rare disease/condition)
2.    Promise (there is a medical rationale for believing that a proposed drug has “promise” for treating the rare disease/condition)

With NPC disease and Cyclodextrin, I believe we easily meet both criteria and we are optimistic that will get a designation.

Only one other orphan drug application in history has been filed with the FDA to treat Niemann Pick Type C disease.  This application was made by a Swiss pharmaceutical company called Actelion Corporation for a drug called Zavesca (Miglustat). Zavesca is currently under review and the FDA will make a decision in early March as to whether it approves Zavesca for the treatment of Niemann Pick Type C disease.

Based upon recent animal studies, Cyclodextrin appears to have far greater promise for the treatment of NPC disease than Zavesca.  The goal of this filing to to move forward with the research in order to verify efficacy and to give kids with Niemann Pick Type C disease a real chance at life.