TRND (Therapies for Rare and Neglected Diseases) and the NIH team met with FDA representatives on December 13th to discuss the progressive plans to bring Cyclodextrin to a clinical trial in 2012. For TRND, Niemann-Pick Type C is one of six pilot projects that were selected.
The NNPDF does a wonderful job of keeping families and people interested up to date on how things are progressing in the disease community for Niemann-Pick Type C. You can visit updates on their page here: http://www.nnpdf.org/aboutus_14.html Continue Reading
Karen Quandt is the Board Chair for the NNPDF.org. A few years back she was completing a study about neurodegenerative disease in family members of patients with Niemann-Pick Type C (NPC). There differently is a relationship between NPC and other diseases. At this given time ,the current discoveries that scientists are making to further develop treatments for NPC are providing critical insights into our understanding relating to a few of the most common diseases that have been shown to be debilitating to life.
Thank you Karen for allowing us to share.
This week the FDA has stated formally that they see promise in the use of Cyclodextrin in treating Niemann-Pick Type C (NPC) by granting its “Orphan Drug Designation”.
In January of 1983, the Orphan Drug Act (ODA) was passed into law in the United States with consistent pursuit by the National Organization for Rare Disorders. The ideal behind this is to encourage pharmaceutical companies to look into smaller markets with development of drugs for orphan diseases. Niemann-Pick Type C (NPC) certainly falls into this category (around than 500 diagnosed ever worldwide). The benchmark on how to determine an orphan disease is one that affects less than 200,000 people. Continue Reading
This is an email from Nadine Hill of the NNPDF. This is good news. If the FDA does say lets move forward that means more families might be able to participate because the drug would be FDA approved. All though this isn’t a cure it is one of hopefully several more therapies to come to fight this horrible disease.
Hello NNPDF Families and Friends,
NOTE: Deadline of Monday, December 28th, 2009
We have received word of an important and historic FDA related drug advisory
committee meeting which is to be held in Maryland on January 12th, 2010 ,
which will review Zavesca as a possible treatment and therapy for
Niemann-Pick Disease Type C. An advisory committee panel of medical and
clinical experts will gather together to review information and learn the
data particulars as it relates to the use of Zavesca in NPC patients. Continue Reading
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